Scientific Publications
The following list scientific articles and research papers regarding Sodium Phenylbuterate, published since 2005. For articles published prior to 2005 please inquire by email to info@ampolgen.com
Cancer Research
Cancer Treatment
Bladder Cancer
1. Mortazavi, A; Hoot, DR; Carlton, PS; Wang, S; Degroff, VL.; Lu, Q; Kulp, S; Chen, C-S; Clinton, SK. Inhibition of cell growth and induction of apoptosis in bladder cancer cell lines by a novel histone deacctylase inhibitor derived from phenylbutyrate. Proceedings of the American Association for Cancer Research Annual Meeting 2005;46(Supplement S):422.
2. Picard, V; Bergeron, A; Larue, H; Fradet, Y. MAGE-A9 mRNA and protein expression in bladder cancer. Int J Cancer 2007;120(10):2170-2177.
Brain Tumors
1. Phuphanich, S; Baker, SD; Grossman, SA; Carson, KA; Gilbert, MR; Fisher, JD; Carducci, MA. Oral sodium phenylbutyrate in patients with recurrent malignant gliomas: a dose escalation and pharmacologic study. Neuro-oncol 2005;7(2):177-182.
2. Vila-Carriles, WH; Kovacs, GG; Jovov, B; Zhou, Z-H; Pahwa, AK; Colby, G; Esimai, O; Gillespie, GY; Mapstone, TB; Markert, JM; Fuller, CM; Bubien, JK; Benos, DJ. Surface expression of ASIC2 inhibits the amiloride-sensitive current and migration of glioma cells. J Biol Chem 2006;281(28):19220-19232.
3. Vila-Carriles, WH; Kovacs, GG; Bubien, JK; Gillespie, GY; Fuller, CM; Benos, DJ. Cellular localization of acid sensing ion channels in human astrocytes and gliomas. FASEB Journal 2006;20(4, Part 1):A325.
4. Vila-Carriles, WH; Zhou, Z-H; Bubien, JK; Fuller, CM; Benos, DJ. Participation of the chaperone Hsc70 in the trafficking and functional expression of ASIC2 in glioma cells. J Biol Chem 2007;282(47):34381-34391.
5. Svechnikova, I; Almqvist, PM; Ekstroem, TJ. HDAC inhibitors effectively induce cell type-specific differentiation in human glioblastoma cell lines of different origin. Int J Oncol 2008;32(4):821-827.
6. Entin-Meer, M; Rephaeli, A; Yang, X; Nudelman, A; Nudelman, A; Haas-Kogan, DA. AN-113, a novel prodrug of 4-phenylbutyrate with increased anti-neoplastic activity in glioma cell lines. Cancer Lett 2007;253(2):205-214.
Breast Cancer
1. Christov, K; Grubbs, C; Lubet, R. Altered cell proliferation and apoptosis as biomarkers for identifying preventive/therapeutic agents against chemically induced mammary cancers. Breast Cancer Research and Treatment 2006;100(Suppl. 1):S58.
2. Christov, Konstantin; Grubbs, Clinton J; Shilkaitis, Anne; Juliana, M Margaret; Lubet, Ronald A. Short-term modulation of cell proliferation and apoptosis and preventive/therapeutic efficacy of various agents in a mammary cancer model. Clin Cancer Res 2007;13(18, Part 1):5488-5496.
3. Christov, K; Grubbs, C; Juliana, M; Luber, R. Correlation of the preventive/therapeutic efficacy of agents in the methylnitrosourea (MNU) mammary cancer model with changes in cell proliferation and apoptosis following short-term treatment. Proceedings of the American Association for Cancer Research Annual Meeting 2007;48:997.
Colon Cancer
1. Lea, MA; Ibeh, C; Shah, N; Moyer, MP. Enhanced differentiation of colon cancer cells induced by combinations of inhibitors of kinases and of historic deacerylases. Proceedings of the American Association for Cancer Research Annual Meeting 2006;47:1184.
2. Sung, MW; Waxman, S. Combination of cytotoxic-differentiation therapy with 5-fluorouracil and phenylbutyrate in patients with advanced colorectal cancer. Anticancer Res 2007;27(2):995-1001.
Combination Treatment
1. Hao, C-l; Tang, K-j; Chen, S; Xing, H-y; Wang, M; Wang, J-x. 5-Aza-2'-deoxycytidine enhances differentiation and apoptosis induced by phenylbutyrate in Kasumi-1 cells. Zhonghua Zhong Liu Za Zhi 2005;27(3):148-151.
2. Rudek, MA; Zhao, M; He, P; Hartke, C; Gilbert, J; Gore, SD; Carducci, MA; Baker, SD. Pharmacokinetics of 5-azacitidine administered with phenylbutyrate in patients with refractory solid tumors or hematologic malignancies. J Clin Oncol 2005;23(17):3906-3911.
3. Gore, SD.; Jiemjit, A; Silverman, LB.; Aucott, T; Baylin, S; Carraway, H; Douses, T; Fandy, T; Herman, J; Karp, JE.; Licht, JD.; Murgo, AJ.; Odchimar-Reissig, R; Smith, BD; Zwiebel, JA.; Sugar, E. Combined Methyltransferase/Histone deacetylase inhibition with 5Azacitidine and MS-275 in patients with MDS, CMMoL and AML: Clinical response, Histone Acetylation and DNA damage. Blood 2006;108(11, part 1):156A-157A.
4. Schniewind, B; Heintz, K; Kurdow, R; Ammerpohl, O; Trauzold, A; Emme, D; Dohrmann, P; Kalthoff, H. Combination phenylbutyrate/gemcitabine therapy effectively inhibits in vitro and in vivo growth of NSCLC by intrinsic apoptotic pathways. J Carcinog 2006;5:25.
5. Gore, SD; Baylin, S; Sugar, E; Carraway, H; Miller, CB; Carducci, M; Grever, M; Galm, O; Dauses, T; Karp, JE; Rudek, MA; Zhao, M; Smith, BD; Manning, J; Jiemjit, A; Dover, G; Mays, A; Zwiebel, J; Murgo, A; Weng, L-J; Herman, JG. Combined DNA methyltransferase and histone deacetylase inhibition in the treatment of myeloid neoplasms. Cancer Res 2006;66(12):6361-6369.
6. Carraway, HE; Gore, SD. Addition of histone deacetylase inhibitors in combination therapy. J Clin Oncol 2007;25(15):1955-1956.
7. Verheul, HMW; Qian, DZ; Carducci, MA; Pili, R. Sequence-dependent antitumor effects of differentiation agents in combination with cell cycle-dependent cytotoxic drugs. Cancer Chemother Pharmacol 2007;60(3):329-339.
8. Ammerpohl, O; Trauzold, A; Schniewind, B; Griep, U; Pilarsky, C; Grutzmann, R; Saeger, H-D; Janssen, O; Sipos, B; Kloppel, G; Kalthoff, H. Complementary effects of HDAC inhibitor 4-PB on gap junction communication and cellular export mechanisms support restoration of chemosensitivity of PDAC cells. Br J Cancer 2007;96(1):73-81.
9. Perron, ME; Plourde, F; Guerard, S; Huynh, L; Leroux, JC; et al. An investigation on the use of tributyrin nanoemulsions for docetaxel delivery. J Drug Deliv Sci Technol 2008;18:189-195.
10.Su, J; Zhang, NN; Ho, PC. Evaluation of the pharmacokinetics of all-trans-retinoic acid (ATRA) in Wistar rats after intravenous administration of ATRA loaded into tributyrin submicron emulsion and its cellular activity on Caco-2 and HepG2 cell lines. J Pharm Sci 2008;97:2844-2853.
11.Huynh, L; Grant, J; Leroux, JC; Delmas, P; Allen, C. Predicting the solubility of the anti-cancer agent docetaxel in small molecule excipients using computational methods. Pharm Res 2008;25:147-157.
12.Jiemjit, A; Fandy, TE; Baylin, SB; Carraway, H; Herman, JG; Gore, SD. Sequential administration of DNA methyltransferase inhibitors (DNMTi) and histone deacetylase inhibitors (HDACi) induces apoptosis with caspase and reactive oxygen species (ROS)-dependent synergy. Proceedings of the American Association for Cancer Research Annual Meeting 2007;48:249.
Esophageal Cancer
1. Brock, MV. Clinical aspects of molecular biology for the diagnosis and treatment of esophageal cancer. Esophagus 2006;3(3):91-94.
2. Beyer, SJ; Kulp, SK; Baird, M; Auer, H; Kornacker, K; Chen, C-S; Beer, DG; Kresty, LA. The effects of a phenylbutyrate-derived histone deacetylase inhibitor (HDAC-42) on acid-induced gene expression patterns of SEG-1 human esophageal adenocarcinoma cells. Proceedings of the American Association for Cancer Research Annual Meeting 2006;47:1128-1129.
Ewing Sarcoma
1. Hurtubise, A; Bernstein, ML; Momparler, RL. Preclinical evaluation of the antineoplastic action of 5-aza-2'-deoxycytidine and different histone deacetylase inhibitors on human Ewing's sarcoma cells. Cancer Cell Int 2008;8:16.
General
1. Garcia-Manero, G; Issa, J-P. Histone deacetylase inhibitors: A review of their clinical status as antineoplastic agents. Cancer Investigation 2005;23(7):635-642.
2. Mack, GS. Epigenetic cancer therapy makes headway. Journal of the National Cancer Institute 2006;98(20):1443-1444.
3. Fouladi, M. Histone deacetylase inhibitors in cancer therapy. Cancer Investigation 2006;24(5):521-527.
4. Galm, O; Herman, JG; Baylin, SB. The fundamental role of epigenetics in hematopoietic malignancies. Blood Rev 2006;20(1):1-13.
5. Lubbert, M.; Claus, R. Epigenetic inactivation of gene expression. New therapeutic targets in hematooncology. Onkologe 2007;13(1):46-55.
6. Cortez, CC; Jones, PA. Chromatin, cancer and drug therapies. Mutat Res - Fundam Mol Mech Mutagen 2008;647(1-2):44-51.
7. Monneret, C. Histone deacetylase inhibitors. Eur J Med Chem 2005;40(1):1-13.
8. Holm, E; Schade, I. Effects of short-chain and saturated long-chain fatty acids on tumor growth - in vitro and experimental in vivo effects. Aktuelle Ernahrungsmedizin 2008;33(5):225-230.
Head & Neck Cancer
1. Gao J; Luan X-y; Xu F-l; Liu D-y; Lei D-p. Effects of sodium phenylbutyrate alone or in combination with fluorouracil or cisplatin on laryngeal carcinoma Hep-2 cell line. Zhongguo Xinyao yu Linchuang Zazhi 2005;24(10):778-781.
2. Gao, J; Ruan, X; Pan, X; Xu, F; Lei, D; Liu, D. The effect of sodium phenylbutyrate to agents used in induction chemotherapy on laryngeal carcinoma cells Hep-2 in vitro. Lin Chuang Er Bi Yan Hou Ke Za Zhi 2005;19(15):680-682.
3. Hattori, Y; Fukushima, M; Maitani, Y. Non-viral delivery of the connexin 43 gene with histone deacetylase inhibitor to human nasopharyngeal tumor cells enhances gene expression and inhibits in vivo tumor growth. Int J Oncol 2007; 30(6):1427-1439.
4. Burkitt, K; Ljungman, M. Phenylbutyrate interferes with the Fanconi anemia and BRCA pathway and sensitizes head and neck cancer cells to cisplatin. Mol Cancer 2008;7:24.
Leukemia
1. Sun, J; Liu, S; Yu, J; Wei, M; Mao, C; Ding, H; Kearney, J; Huynh, L; Paschka, P; Wang, D; Klisovic, RB.; Perrotti, D; Chen, C-S; Blum, WG; Marcucci, G. Characterization of HDACI OSU42 as a novel histone deacetylase inhibitor in AML cell lines. Blood 2006;108(11 part 1):563A.
2. Luebbert, M. Combined targeting of the epigenetic silence in leukemia: Cooperating activities of DNA methylation and histone deacetylation inhibitors. Leukemia Research 2005;29(7):727-728.
3. Maslak, P; Chanel, S; Camacho, LH; Soignet, S; Pandolfi, PP; Guernah, I; Warrell, R; Nimer, S. Pilot study of combination transcriptional modulation therapy with sodium phenylbutyrate and 5-azacytidine in patients with acute myeloid leukemia or myelodysplastic syndrome. Leukemia 2006;20(2):212-217.
4. West, DA; Lucas, DM; Davis, ME; De lay, MD; Johnson, AJ; Guster, SE; Freitas, MA; Parthun, MR; Wang, D; Kulp, SK; Grever, MR; Chen, C-S; Byrd, JC. The novel histone deacetylase inhibitor OSU-HDAC42 has class I and II histone deacetylase (HDAC) inhibitory activity and represents a novel therapy for chronic lymphocytic leukemia. Blood 2006;108(11, Part 1):794A-795A.
5. Cunha De Santis, G; de Barros Tamarozzi, M; Sousa, RB; Moreno, SE; Secco, D; Garcia, AB; Lima, ASG; Faccioli, LH; Falcoao, RP; Cunha, FQ; Rego, EM. Adhesion molecules and Differentiation Syndrome: phenotypic and functional analysis of the effect of ATRA, As2O3, phenylbutyrate, and G-CSF in acute promyelocytic leukemia. Haematologica 2007;92(12):1615-1622.
6. Koutsourea, AI; Fousteris, MA; Arsenou, ES; Papageorgiou, A; Pairas, GN; Nikolaropoulos, SS. Rational design, synthesis, and in vivo evaluation of the antileukemic activity of six new alkylating steroidal esters. Bioorg Med Chem 2008;16(9):5207-5215.
7. Estey, E. New drugs in acute myeloid leukemia. Seminars in Oncology 2008;35(4):439-448.
Liver Cancer
1. Meng, M; Wang, C-T; Jiang, J-M; Zhang, J-C; Jiang, J-J; Jin, C-J. Expression of histone deacetyase 4 in human liver carcinoma cell line Bel-7402 and its significance. Chinese Journal of Cancer Biotherapy Chin. J. Cancer Biother 2007;14(2):153-157.
2. Lu, Y-S; Kashida, Y; Kulp, SK; Wang, Y-C; Wang, D; Hung, J-H; Tang, M; Lin, Z-Z; Chen, T-J; Cheng, A-L; Chen, C-S. Efficacy of a novel histone deacetylase inhibitor in murine models of hepatocellular carcinoma. Hepatology 2007;46(4):1119-1130.
3. Svechnikova, I; Ammerpohl, O; Ekstroem, TJ. p21waf1/Cip1 partially mediates apoptosis in hepatocellular carcinoma cells. Biochem Biophys Res Commun 2007;354(2):466-471.
4. Lu, Y-S; Kashida, Y; Hung, J-H; Chen, C-S; Wang, Y-C; Cheng, A-L; Chen, C-S. In vivo efficacy of OSU-HDAC42, a novel phenylbutyrate-based histone deacetylase inhibitor, in human hepatocellular carcinoma animal models. Proceedings of the American Association for Cancer Research Annual Meeting 2007;48:169.
5. Wang, H-E; Wu, H-C; Kao, S-J; Tseng, F-W; Wang, Y-S; Yu, H-M; Chou, S-L; Yen, S-H; Chi, K-H. Modulation of 5-fluorouracil cytotoxicity through thymidylate synthase and NF-kappaB down-regulation and its application on the radiolabelled iododeoxyuridine therapy on human hepatoma cell. Biochem Pharmacol 2005;69(4):617-626.
6. Meng, M; Jiang, JM.; Liu, H; In, CY; Zhu, JR. Effects of sodium phenylbutyrate on differentiation and induction of the P<sup>21WAF1/CIP1</sup> anti-oncogene in human liver carcinoma cell lines. Chin J Dig Dis 2005;6(4):189-192.
Lung Cancer
1. Lyon, CM; Klinge, D; Liechty, KC; Belinsky, SA. DNA demethylating agents and a PPA-gamma agonist cooperate to induce apoptosis in lung cancer cell lines. Proceedings of the American Association for Cancer Research Annual Meeting 2006;47:10.2. Lyon, CM; Klinge, D; Liechty, KC; Grimes, MJ; Grimes, J; Thomas, C; March, T; Stidley, C; Keith, R; Belinsky, SA. Rosiglitazone prevents the progression of pre-invasive lung cancer in a murine model. Proceedings of the American Association for Cancer Research Annual Meeting 2007;48:395.
Myelodysplastic Syndrome
1. Meletis, J; Viniou, N; Terpos, E. Novel agents for the management of myelodysplastic syndromes. Med Sci Monit 2006;12(9):RA194-RA206.
2. Claus, R; Router, B; Loubbert, M. Targets of epigenetic therapy - Gene reactivation as a novel approach in MDS treatment. Cancer Treat Rev 2007;33(Suppl. 1):S47-S52.
3. Luebbert, M. Which myelodysplastic syndromes patients are candidates for treatment with decitabine. Leukemia Research 2007;31(Suppl. 1):S20.
4. Gore SD. Do histone deacetylase inhibitors have a place in the treatment of myelodysplastic syndromes? Leukemia Research 2007;31(Suppl. 1):S21.
Non-Hodgkin’s Lymphoma
1. Phillips, JA; Griffin, BE. Pilot study of sodium phenylbutyrate as adjuvant in cyclophosphamide-resistant endemic Burkitt's lymphoma. Trans R Soc Trop Med Hyg 2007;101(12):1265-1269.
Osteosarcoma
1. Murahari, S; Jalkanen, AL.; Kulp, SK.; Chen, C-S; Jubala, CM; Fosmire, SP; Modiano, JF; Fossey, SL; London, CA; Kisseberth, WC. OSU-HDAC42, a novel histone deacetylase inhibitor with potent antitumor effects on human and canine osteosarcoma cells. Proceedings of the American Association for Cancer Research Annual Meeting 2008;49:578.
Prostate Cancer
1. Milkevitch, M; Shim, H; Pilatus, U; Pickup, S; Wehrle, JP; Samid, D; Poptani, H; Glickson, JD; Delikatny, EJ. Increases in NMR-visible lipid and glycerophosphocholine during phenylbutyrate-induced apoptosis in human prostate cancer cells. Biochim Biophys Acta 2005; 1734(1):1-12.
2. Kulp, SK; Chen, C-S; Wang, D-S; Chen, C-Y; Chen, C-S. Antitumor effects of a novel phenylbutyrate-based histone deacetylase inhibitor, (S)-HDAC-42, in prostate cancer. Clin Cancer Res 2006;12(17):5199-5206.
3. Milkevitch, M; Jeitner, TM; Beardsley, NJ; Delikatny, EJ. Lovastatin enhances phenylbutyrate-induced MR-visible glycerophosphocholine but not apoptosis in DU145 prostate cells. Biochim Biophys Acta 2007;1771(9):1166-1176.
Cardioprotection
1. Daosukho, C; Chen, Y; Noel, T; Sompol, P; Nithipongvanitch, R; Velez, JM; Oberley, TD; St Clair, DK. Phenylbutyrate, a histone deacetylase inhibitor, protects against Adriamycin-induced cardiac injury. Free Radic Biol Med 2007;42(12):1818-1825.
Phase I Study
1. Camacho, LH; Olson, J; Tong, WP; Young, CW; Spriggs, DR; Malkin, MG. Phase I dose escalation clinical trial of phenylbutyrate sodium administered twice daily to patients with advanced solid tumors. Invest New Drugs 2007;25(2):131-138.
Phenylbutyrate Derivatives
1. Sampathkumar, S.-G; Jones, MB.; Meledeo, MA; Hida, K; Sheh, T; Gomatputra, P; Yarema, KJ. Carbohydrate-based small molecules as anti-cancer drugs: Short chain fatty acid-hexosamine hybrids. Glycobiology 2005;15(11):1249-1250.
2. Lu, Q; Wang, D-S; Chen, C-S; Hu, Y-D; Chen, C-S. Structure-based optimization of phenylbutyrate-derived histone deacetylase inhibitors. J Med Chem 2005;48(17):5530-5535.
Radiosensitization
1. Munshi, A; Kurland, JF; Nishikawa, T; Tanaka, T; Hobbs, ML; Tucker, SL; Ismail, S; Stevens, C; Meyn, RE. Histone deacetylase inhibitors radiosensitize human melanoma cells by suppressing DNA repair activity. Clin Cancer Res 2005;11(13):4912-4922.
2. Lopez, CA; Feng, FY; Herman, JM; Nyati, MK; Lawrence, TS; Ljungman, M. Phenylbutyrate sensitizes human glioblastoma cells lacking wild-type p53 function to ionizing radiation. Int J Radiat Oncol Biol Phys 2007;69(1):214-220.
Resistance to Treatment
1. Fantin, VR; Richon, VM. Mechanisms of resistance to histone deacetylase inhibitors and their therapeutic implications. Clin Cancer Res 2007;13(24):7237-7242.
Addiction
1. Romieu, P; Host, L; Gobaille, S; Sandner, G; Aunis, D; Zwiller, J. Histone deacetylase inhibitors decrease cocaine but not sucrose self-administration in rats. J Neurosci 2008;28(38):9342-9348.
Adrenoleukodystrophy
1. Gondcaille, C; Depreter, M; Fourcade, SU; Lecca, MR; Leclercq, S; Martin, PGP; Pineau, T; Cadepond, F; ElEtr, M; Bertrand, N; Beley, A; Duclos, S; De Craemer, D; Roels, F; Savary, SU; Bugaut, M. Phenylbutyrate up-regulates the adrenoleukodystrophy-related gene as a nonclassical peroxisome proliferator. J Cell Biol 2005;169(1):93-104.
Aging
1. Wagner, G. Towards a life prolonging pill? Small molecules with anti-ageing properties. Current Drug Targets 2006;7(11):1531-1537.
2. Burzynski SR. Aging: gene silencing or gene activation? Med Hypotheses 2005;64(1):201-208.
Alpha-Galactosidase Mutation
1. Yam, GH-F; Roth, J; Zuber, C. 4-Phenylbutyrate rescues trafficking incompetent mutant alpha-galactosidase A without restoring its functionality. Biochem Biophys Res Commun 2007;360(2):375-380.
ALS
1. Targets in ALS: designing multidrug therapies. Carri, MT; Grignaschi, G; Bendotti, C. Trends Pharmacol Sci 2006;27(5):267-273.
2. Additive neuroprotective effects of a histone deacetylase inhibitor and a catalytic antioxidant in a transgenic mouse model of amyotrophic lateral sclerosis. Petri, S; Kiaei, M; Kipiani, K; Chen, J; Calingasan, NY; Crow, JP; Beal, MF. Neurobiol Dis 2006;22(1):40-49.
3. Neuroprotective agents for clinical trials in ALS: a systematic assessment. Traynor, BJ; Bruijn, L; Conwit, R; Beal, F; O'Neill, G; Fagan, SC; Cudkowicz, ME. Neurology 2006;67(1):20-27.
4. Sodium phenylbutyrate prolongs survival and regulates expression of anti-apoptotic genes in transgenic amyotrophic lateral sclerosis mice. Ryu, H; Smith, K; Camelo, SI; Carreras, I; Lee, J; Iglesias, AH; Dangond, F; Cormier, KA; Cudkowicz, ME; Brown, RH; Ferrante, RJ. J Neurochem 2005;93(5):1087-1098.
5. Erratum: Sodium phenylbutyrate prolongs survival and regulates expression of anti-apoptotic genes in transgenic amyotrophic lateral sclerosis mice (Journal of Neurochemistry (2005) 93 (1087-1098)). Ryu, H; Smith, K; Camelo, SI; Carreras, I; Lee, J; Iglesias, AH; Dangond, F; Cormier, KA; Cudkowicz, ME; Brown, Jr RH; Ferrante, RJ. J Neurochem 2006;96(3):908.
6. Therapeutic targets for amyotrophic lateral sclerosis: Current treatments and prospects for more effective therapies. Bruijn, LI.; Cudkowicz, M. Expert Rev Neurother 2006;6(3):417-428.
7. Translating preclinical insights into effective human trials in ALS. DiBernardo, AB; Cudkowicz, ME. Biochim Biophys Acta - Mol Basis Dis 2006;1762(11-12):1139-1149.
8. Design, power, and interpretation of studies in the standard murine model of ALS. Scott, S; Kranz, JE; Cole, J; Lincecum, JM; Thompson, K; Kelly, N; Bostrom, A; Theodoss, J; Al-Nakhala, BM; Vieira, FG; Ramasubbu, J; Heywood, JA. Amyotroph Lateral Scler 2008;9(1):4-15.
9. Safety and does escalating study of oral sodium phenylbutyrate in subjects with ALS. Cudkowicz, ME; Andres, PL; Choudry, R; MacDonald, SA; Zhang, H; Schoenfeld, D; Ferrante, RJ. Neurology 2007;68(12, Suppl. 1):A90.
Anemia
1. A new generation of gamma globin gene inducers act on novel molecular and cellular targets. Perrine, S; Trudel, M; Castaneda, SA.; Mankidy, R; Boosalis, MS.; Mpollo, M-SEM; White, GL; Bohacek, R; Faller, DV. Blood 2006;108(11, Part 1):453A.
2. Advances in the management of sickle cell disease. Thompson, AA. Pediatr Blood Cancer 2006;46(5):533-539.
3. Fetal globin stimulant therapies in the beta-hemoglobinopathies: principles and current potential. Perrine, SP. Pediatr Ann 2008;37(5):339-346.
4. Cucurbitacin D upregulates fetal hemoglobin expression in K562 cells and human erythroid progenitors. Xing, H; Zhang, S; Koeffler, HP; Fung, MC. Blood 2007;110(11, Part 2):26B-27B.
5. Sodium phenyl butyrate downregulates endothelin-1 expression in cultured human endothelial cells: Relevance to sickle-cell disease. Odievre, M-H; Brun, M; Krishnamoorthy, R; Lapoumeroulie, C; Elion, J. American Journal of Hematology 2007;82(5):357-362.
6. Pulsed-dosing with oral sodium phenylbutyrate increases hemoglobin F in a patient with sickle cell anemia. Hines, P; Dover, GJ; Resar, LMS. Pediatr Blood Cancer 2008;50(2):357-359.
Anti-Parasite Activity
1. Scriptaid and suberoylanilide hydroxamic acid are histone deacetylase inhibitors with potent anti-Toxoplasma gondii activity in vitro. Strobl, JS; Cassell, M; Mitchell, SM; Reilly, CM; Lindsay, DS. J Parasitol 2007;93(3):694-700.
Arginase Induction
1. Arginase induction by sodium phenylbutyrate in mouse tissues and human cell lines. Kern, RM; Yang, Z; Kim, PS; Grody, WW; Iyer, RK; Cederbaum, SD. Mol Genet Metab 2007;90(1):37-41.
Argininosuccinate Lyase Deficiency
1. Potassium retention in patients treated for argininosuccinate lyase deficiency. Singh, RH; Acosta, PB; Kennedy, MP; Longo, N; Elsas, LJ. Journal of Inherited Metabolic Disease 2006;29(Suppl. 1):48.
2. Sustained Engraftment and Tissue Enzyme Activity After Liver Cell Transplantation for Argininosuccinate Lyase Deficiency. Stephenne, X; Najimi, M; Sibille, C; Nassogne, M; Smets, F; Sokal, EM. Gastroenterology 2006;130(4):1317-1323.
Bladder Dysfunction
1. A novel target for boo-induced bladder dysfunction. Sawada, N; Yao, J; Hiramatsu, N; Hayakawa, K; Zakoji, H; Kobayashi, H; Mochizuki, T; Araki, I; Takeda, M; Kitamura, M. European Urology Supplements 2008;7(3):265.
Chemical Analysis
1. Separation and determination of optical isomers of ethyl small alpha -hydroxyphenylbutyrate by chiral HPLC. Zhang, YY; Chen, GC. Fenxi Ceshi Xuebao 2005;24(1):109-110.
2. Metabolite identification using a nanoelectrospray LC-EC-array-MS integrated system. Schiavo, S; Ebbel, E; Sharma, S; Matson, W; Kristal, BS; Hersch, S; Vouros, P. Anal Chem 2008;80(15):5912-5923.
3. A two-site ELISA can quantify upregulation of SMN protein by drugs for spinal muscular atrophy. Nguyen, M; Humphrey, E; Lam, LT; Fuller, HR; Lynch, TA; Sewry, CA; Goodwin, PR; Mackenzie, AE; Morris, GE. Neurology 2008;71(22):1757-1763.
4. Detection of triglyceride using an iridium nano-particle catalyst based amperometric biosensor. Liao, W-Y; Liu, C-C; Chou, T-C. Analyst 2008;133(12):1757-1763.
5. Multiple-layer substrate zymography for detection of several enzymes in a single sodium dodecyl sulfate gel. Choi, N-S; Kim, B-H; Park, C-S; Han, YJ; Lee, HW; Choi, JH; Lee, S-G; Song, JJ. Anal Biochem 2009;386(1):121-122.
Cholestatic Diseases
1. Levels of plasma membrane expression in progressive and benign mutations of the bile salt export pump (Bsep/Abcb11) correlate with severity of cholestatic diseases. Lam, P; Pearson, CL; Soroka, CJ; Xu, S; Mennone, A; Boyer, JL. Am J Physiol Cell Physiol 2007;293(5):C1709-1716.
2. Chemical chaperones partially reverse the mis-processing of a BRIC2 mutant of the bile salt export pump, ABCB11. Lam, P; Soroka, CJ.; Boyer, JL. Hepatology 2007;46(4, Suppl. S):330A.
3. 4-phenylbutyrate enhances the cell surface expression and the transport capacity of wild-type and mutated bile salt export pumps. Hayashi, H; Sugiyama, Y. Hepatology 2007;45(6):1506-1516.
4. Short-chain ubiquitination is associated with the degradation rate of a cell-surface-resident bile salt export pump (BSEP/ABCB11). Hayashi, H; Sugiyama, Y. Mol Pharmacol 2009;75(1):143-150.
Citrulline Metabolism
1. Manipulation of citrulline availability in humans. Rougoe, C; Des Robert, C; Robins, A; Le Bacquer, O; Volteau, C; De La Cochetioere, M-F; Darmaun, D. Am J Physiol Gastrointest Liver Physiol 2007;293(5):G1061-1067.
Computer Modeling
1. Toward the computational design of diastereomeric resolving agents: an experimental and computational study of 1-phenylethylammonium-2-phenylacetate derivatives. Karamertzanis, PG; Anandamanoharan, PR; Fernandes, P; Cains, PW; Vickers, M; Tocher, DA; Florence, AJ; Price, SL. J Phys Chem B 2007;111(19):5326-5336.
Cystic Fibrosis
1. Pharmacological induction of CFTR function in patients with cystic fibrosis: mutation-specific therapy. Kerem, E. Pediatr Pulmonol 2005;40(3):183-196.
2. Novel short chain fatty acids restore chloride secretion in cystic fibrosis. Nguyen, TD; Kim, U-S; Perrine, SP. Biochem Biophys Res Commun 2006;342(1):245-252.
3. Selective inhibition of endoplasmic reticulum-associated degradation rescues DeltaF508-cystic fibrosis transmembrane regulator and suppresses interleukin-8 levels: therapeutic implications. Vij, N; Fang, S; Zeitlin, PL. J Biol Chem 2006;281(25):17369-17378.
4. Mutation specific therapy in CF. Kerem, E. Pediatr Respir Rev 2006;7(Suppl. 1):S166-S169.
5. Pharmacoproteomics of 4-phenylbutyrate-treated IB3-1 cystic fibrosis bronchial epithelial cells. Singh, OV; Vij, N; Mogayzel, PJ; Jozwik, C; Pollard, HB; Zeitlin, PL. J Proteome Res 2006;5(3):562-571.
6. Cystic fibrosis transmembrane regulator protein mutations: 'Class' opportunity for novel drug innovation. MacDonald, KD; McKenzie, KR; Zeitlin, PL. Pediatr Drugs 2007;9(1):1-10.
7. Chemical rescue of deltaF508-CFTR mimics genetic repair in cystic fibrosis bronchial epithelial cells. Singh, OV; Pollard, HB; Zeitlin, PL. Mol Cell Proteomics 2008;7(6):1099-1110.
8. Proinflammatory effect of sodium 4-phenylbutyrate in deltaF508-cystic fibrosis transmembrane conductance regulator lung epithelial cells: involvement of extracellular signal-regulated protein kinase 1/2 and c-Jun-NH2-terminal kinase signaling. Roque, T; Boncoeur, E; Saint-Criq, V; Bonvin, E; Clement, A; Tabary, O; Jacquot, J. J Pharmacol Exp Ther 2008;326(3):949-956.
Denervating Diseases
1. New treatments for denervating diseases. Pleasure D. J Child Neurol 2005;20(3):258-262.
Endoplasmic Reticulum Stress
1. Mutant tamm-horsfall glycoprotein accumulation in endoplasmic reticulum induces apoptosis reversed by colchicine and sodium 4-phenylbutyrate. Choi, SW; Ryu, OH; Choi, SJ; Song, IS; Bleyer, AJ; Hart, TC. J Am Soc Nephrol 2005;16(10):3006-3014.
2. Suppressive effects of 4-phenylbutyrate on the aggregation of Pael receptors and endoplasmic reticulum stress. Kubota, K; Niinuma, Y; Kaneko, M; Okuma, Y; Sugai, M; Omura, T; Uesugi, M; Uehara, T; Hosoi, T; Nomura, Y. J Neurochem 2006;97(5):1259-1268.
3. Chemical chaperones reduce endoplasmic reticulum stress and prevent mutant HFE aggregate formation. de Almeida, SF; Picarote, G; Fleming, JV; Carmo-Fonseca, M; Azevedo, JE; de Sousa, M. J Biol Chem 2007;282(38):27905-27912.
4. KLF5 plays an important role in endoplasmic reticulum stress response in both cardiovascular and metabolic system. Eguchi, K; Manabe, I; Shen, H; Ohishi, Y; Fujiu, K; Takeda, N; Nagai, R. Circulation 2007;116(16, Suppl. S):44.
5. Involvement of endoplasmic reticulum stress in palmitate-induced endothelial cell apoptosis. Jang, HJ; Jung, SH; Park, TJ; Jo, YE; Kim, YK; Jung, JG; Choi, S-E; Kang, Y; Kim, HJ; Kim, DJ; Lee, KW. Diabetes 2007;56(Suppl. 1):A508-A509.
6. Involvement of hypoxia-triggered endoplasmic reticulum stress in outlet obstruction-induced apoptosis in the urinary bladder. Sawada, N; Yao, J; Hiramatsu, N; Hayakawa, K; Araki, I; Takeda, M; Kitamura, M. Lab Invest 2008;88(5):553-563.7. BAX inhibitor-1 modulates endoplasmic reticulum stress-mediated programmed cell death in Arabidopsis. Watanabe, N; Lam, E. J Biol Chem 2008;283(6):3200-3210.
8. Sodium 4-phenylbutyrate acts as a chemical chaperone on misfolded myocilin to rescue cells from endoplasmic reticulum stress and apoptosis. Yam, GH-F; Gaplovska-Kysela, K; Zuber, C; Roth, J. Invest Ophthalmol Vis Sci 2007;48(4):1683-1690.
9. 4-PBA(4-phenylbutyrate) reduced palmitate-induced endoplasmic reticulum stress and apoptosis in HUVEC. Jang, H-J; Jung, J; Ha, E-S; Choi, S-E; Jung, S; Kim, H; Kim, D; Kang, Y; Lee, K-W. Diabetologia 2007;50(Suppl. 1):S278.
10.Oxidative stress induces the endoplasmic reticulum stress and facilitates inclusion formation in cultured cells. Hanada, S; Harada, M; Kumemura, H; Bishr Omary, M; Koga, H; Kawaguchi, T; Taniguchi, E; Yoshida, T; Hisamoto, T; Yanagimoto, C; Maeyama, M; Ueno, T; Sata, M. J Hepatol 2007;47(1):93-102.
General
1. Activity of the CHMP. Throm, S. Pharm Ind 2005;67(1):57-63.
2. Activity of digestive enzymes in Scophthalmus maximus. Fu XH; Sun M; Sun SC. Journal of Fishery Sciences of China 2005;12(1):26-32.
3. Novel strategy for protein exploration: high-throughput screening assisted with fuzzy neural network. Kato, R; Nakano, H; Konishi, H; Kato, K; Koga, Y; Yamane, T; Kobayashi, T; Honda, H. J Mol Biol 2005;351(3):683-692.
4. Molecular cloning of enantioselective ester hydrolase from Bacillus pumilus DBRL-191. Rasool, S; Johri, S; Riyaz-ul-Hassan, S; Maqbool, Q-u-A; Verma, V; Koul, S; Taneja, SC; Qazi, GN. FEMS Microbiol Lett 2005;249(1):113-120.
5. Modular assembly using sequential palladium coupling gives easy access to the SMoC class of cellular transporters. Rebstock, A-S; Visintin, C; Leo, E; Posada, CG; Kingsbury, SR; Williams, GH; Stoeber, K; Selwood, DL. ChemBioChem 2008;9(11):1787-1796.
6. Waste frying oil as substrate for lipase production by Geotrichum candidum strains. Rywinska, A; Witkowska, D; Juszczyk, P; Rymowicz, W; Kita, A. Polish Journal of Environmental Studies 2008;17(6):925-931.
7. The application of calcined natural dolomitic rock as a solid base catalyst in triglyceride transesterification for biodiesel synthesis. Wilson, K; Hardacre, C; Lee, AF; Montero, JM; Shellard, L. Green Chemistry 2008;10(6):654-659.
Glaucoma
1. Russell body formation and apoptosis in myocilin-caused primary open-angle glaucoma: rescue by the chemical chaperone sodium 4-phenylbutyrate. Roth, J; Yam, GH-F; Gaplovska-Kysela, K; Zuber, C. FASEB Journal 2007;21(5):A182.
Glycosylphosphatidyl Inositol Deficiency
1. Targeted molecular therapy for inherited glycosylphosphatidylinositol deficiency. Almeida, AM.; Murakami, Y; Baker, A; Maeda, Y; Roberts, I; Kinoshita, T; Layton, DM; Karadimitris, A. Blood 2006;108(11, Part 1):148A.
2. Targeted therapy for inherited GPI deficiency. Almeida, AM; Murakami, Y; Baker, A; Maeda, Y; Roberts, IAG; Kinoshita, T; Layton, DM; Karadimitris, A. New Engl J Med 2007;356(16):1641-1647.
Huntington’s Disease
1. Genome-wide expression profiling of human blood reveals biomarkers for Huntington's disease. Borovecki, F; Lovrecic, L; Zhou, J; Jeong, H; Then, F; Rosas, HD; Hersch, SM; Hogarth, P; Bouzou, B; Jensen, RV; Krainc, D. Proc Natl Acad Sci U S A 2005;102(31):11023-11028.
2. Neuroprotective effects of phenylbutyrate in the N171-82Q transgenic mouse model of Huntington's disease. Gardian, G; Browne, SE; Choi, D-K; Klivenyi, P; Gregorio, J; Kubilus, JK; Ryu, H; Langley, B; Ratan, RR; Ferrante, RJ; Beal, MF. J Biol Chem 2005;280(1):556-563.
3. A transcriptional report card from the peripheral blood: Can it measure disease progression in Huntington's disease? Tabchy, A; Housman, D. European Journal of Human Genetics 2006;14(6):649-650.
4. Sodium phenylbutyrate in Huntington's disease: a dose-finding study. Hogarth, P; Lovrecic, L; Krainc, D. Mov Disord 2007;22(13):1962-1964.
Inborn Errors in Metabolism
1. Inborn errors of metabolism: The flux from Mendelian to complex diseases. Lanpher, B.; Brunetti-Pierri, N.; Lee, B. Nat Rev Gen 2006;7(6):449-460.
2. Functional and trafficking defects in ATP binding cassette A3 mutants associated with respiratory distress syndrome. Cheong, N; Madesh, M; Gonzales, LW; Zhao, M; Yu, K; Ballard, PL; Shuman, H. J Biol Chem 2006;281(14):9791-9800.
3. Pharmacological treatment of congenital metabolic disorders. Campino VA. Aten Farm 2006;8(1):39-46.
4. A newborn infant with hyperventilation. Lindemann, R; Myhre, MC; Bakken, M; Fugelseth, D; Rustad, CF; Woldseth, B. Tidsskr Nor Laegeforen 2008;128(13):1535-1536.
5. Failure of bone morphogenetic protein receptor trafficking in pulmonary arterial hypertension: potential for rescue. Sobolewski, A; Rudarakanchana, N; Upton, PD; Yang, J; Crilley, TK; Trembath, RC; Morrell, NW. Hum Mol Genet 2008;17(20):3180-3190.
Klinefelter Syndrome
1. An OTC deficiency 'phenocopy' in association with Klinefelter syndrome. Swarts, L; Leisegang, F; Owen, EP; Henderson, HE. J Inherit Metab Dis 2007;30(1):101.
Leucine Metabolism
1. Acute depletion of plasma glutamine increases leucine oxidation in prednisone-treated humans. Le Bacquer, O; Mauras, N; Welch, S; Haymond, M; Darmaun, D. Clin Nutr 2007;26(2):231-238.
2. Phenylbutyrate reduces plasma leucine concentrations without affecting the flux of leucine. Marini, JC; Lanpher, B; Scaglia, F; Carter, S; Garlick, PJ; Jahoor, F; Lee, B. FASEB Journal 2007;21(5):A335.
Lipoprotein Receptor
1. Rescue of the low density lipoprotein receptor 2A mutant G544V by 4-phenylbutyrate. Tveten, K; Holla, OL; Ranheim, T; Berge, KE; Leren, TP; Kulseth, MA. Molecular chaperones and the heat shock response 2006;187.
2. 4-Phenylbutyrate restores the functionality of a misfolded mutant low-density lipoprotein receptor. Tveten, K; Holla, OL; Ranheim, T; Berge, KE; Leren, TP; Kulseth, MA. FEBS J 2007;274(8):1881-1893.
Lysinuric Protein Intolerance
1. Nutrient intake in lysinuric protein intolerance. Tanner, LM; Noantoe-Salonen, K; Venetoklis, J; Kotilainen, S; Niinikoski, H; Huoponen, K; Simell, O. J Inherit Metab Dis 2007;30(5):716-721.
Mechanism of Action
1. Does acute glutamine depletion enhance the response of glutamine synthesis to fasting in muscle in adult and old rats? Meynial-Denis, D; Verdier, L; Mignon, M; Leclerc, J-NA; Bayle, GU; Darmaun, D. Clin Nutr 2005;24(3):398-406.
2. Activation of STAT-3 in CF epithelial cell by Sodium 4-Phenylbutyrate. Suaud, L; Rubenstein, RC. FASEB Journal 2005;19(4, Suppl. S, Part 1):A658.
3. Cellular and molecular responses to cerebral hypoxia: Involvement of ER in neuronal death. Nomura, Y. Journal of Pharmacological Sciences 2005;97(Suppl. 1):6P.
4. DNA methyltransferase (DNMT)-independent effects of DNMT inhibitors on gene expression. Jiemjit, A; Baylin, S; Herman, J; Gore, S. Proceedings of the American Association for Cancer Research Annual Meeting 2005;46(Suppl. S):427.
5. Mutational analysis of 6-aminohexanoate-dimer hydrolase: Relationship between nylon oligomer hydrolytic and esterolytic activities. Ohki, T; Wakitani, Y; Takeo, M; Yasuhira, K; Shibata, N; Higuchi, Y; Negoro, S. FEBS Lett 2006;580(21):5054-5058.
6. Anti-inflammatory effects of short chain fatty acids in IFN-gamma-stimulated RAW 264.7 murine macrophage cells: involvement of NF-kappaB and ERK signaling pathways. Park, J-S; Lee, E-J; Lee, J-C; Kim, W-K; Kim, H-S. Int Immunopharmacol 2007;7(1):70-77.
7. Multiple molecular chaperone-mediated pharmacologic rescue of Delta F508-CFTR from ERAD. Singh, OV; Zeitlin, PL. FASEB Journal 2007;21(5):A420.
8. Optimum immobilization of Candida rugosa lipase on Celite by RSM. Chang SF; Chang SW; Yen YH; Shieh CJ. Applied Clay Science 2007;37(1/2):67-73.
9. Manipulating the quality control pathway in transfected cells: low temperature allows rescue of secretion-defective fibrinogen mutants. Vu, D; Di Sanza, C; Neerman-Arbez, M. Haematologica 2008;93(2):224-231.
10.Pro-inflammatory effect of sodium 4-phenylbutyrate in DeltaF508-CFTR lung cells: Involvement of ERK1/2 and JNK signaling. Roque, T; Boncoeur, E; Saint-Criq, V; Bonvin, E; Clement, A; Tabary, O; Jacquot, J. Rev Mal Respir 2008;25(9):1186.
Metabolism
1. Phenylacetate catabolism in Rhodococcus sp. strain RHA1: a central pathway for degradation of aromatic compounds. Navarro-Llorens, JM; Patrauchan, MA; Stewart, GR; Davies, JE; Eltis, LD; Mohn, WW. J Bacteriol 2005;187(13):4497-4504.
2. Microbial reduction of ethyl 2-oxo-4-phenylbutyrate. Searching for R-enantioselectivity. New access to the enalapril like ACE inhibitors. de, Lacerda PSB; Ribeiro, JB; Leite, SGF; Ferrara, MA; Coelho, RB; Bon, EPS; da, Silva Lima EL; Antunes, OAC. Tetrahedron Asymmetry 2006;17(8):1186-1188.
3. Regio- and enantio selective reduction of methyleneketoesters mediated by Saccharomyces cerevisiae. Clososki, GC; Milagre, CDF; Moran, PJS; Rodrigues, JAR. Journal of Molecular Catalysis B Enzymatic 2007;48(3-4):70-76.
4. Modulation of the activity of newly synthesized human phenylalanine hydroxylase mutant proteins by low-molecular-weight compounds. Nascimento, C; Leandro, J; Tavares de Almeida, I; Leandro, P. Protein J 2008;27(6):392-400.
5. Effect of ionic liquid BMIM PF6 on asymmetric reduction of ethyl 2-oxo-4-phenylbutyrate by Saccharomyces cerevisiae. Shi, Y-G; Fang, Y; Ren, Y-P; Wu, H-P; Guan, H-L. J Ind Microbiol Biotechnol 2008;35(11):1419-1424.
N-acetylglutamate Synthase Deficiency
1. Misleading diagnosis of partial N-acetylglutamate synthase deficiency based on enzyme measurement corrected by mutation analysis. Heckmann, M; Wermuth, B; Heberle, J; Koch, HG; Gortner, L; Kreuder, JG. Acta Paediatr 2005;94(1):121-124.
Neuroprotection
1. Synergistic neuroprotective effects of lithium and valproic acid or other histone deacetylase inhibitors in neurons: roles of glycogen synthase kinase-3 inhibition. Leng, Y; Liang, M-H; Ren, M; Marinova, Z; Leeds, P; Chuang, D-M. J Neurosci 2008;28(10):2576-2588.
2. HDAC inhibition amplifies gap junction communication in neural progenitors: potential for cell-mediated enzyme prodrug therapy. Khan, Z; Akhtar, M; Asklund, T; Juliusson, B; Almqvist, PM; Ekstroem, TJ. Exp Cell Res 2007;313(13):2958-2967.
3. Endogenous alpha-synuclein is induced by valproic acid through histone deacetylase inhibition and participates in neuroprotection against glutamate-induced excitotoxicity. Leng, Y; Chuang, D-M. J Neurosci 2006;26(28):7502-7512.
4. Phenylbutyrate modulates multiple neuronal survival gene expression in SMA cells. Ganta, M; Grzeschik, SM; Heavlin, WD; Wang, CH. Annals of Neurology 2006;60(Suppl. 10):S140.
Parkinson’s Disease
1. Neurodegeneration of mouse nigrostriatal dopaminergic system induced by repeated oral administration of rotenone is prevented by 4-phenylbutyrate, a chemical chaperone. Inden, M; Kitamura, Y; Takeuchi, H; Yanagida, T; Takata, K; Kobayashi, Y; Taniguchi, T; Yoshimoto, K; Kaneko, M; Okuma, Y; Taira, T; Ariga, H; Shimohama, S. J Neurochem 2007;101(6):1491-1504.
2. Neuroprotective effect of chemical chaperone 4-PBA on rotenone-induced dopaminagic neurodegeneration. Tamaki, A; Yanagida, T; Kitamura, Y; Takata, K; Taniguchi, T. Journal of Pharmacological Sciences 2008;106(suppl. 1):233P.
3. Translational research of parkinsonian therapeutics. Kitamura, Y; Taniguchi, T; Ariga, H. J Pharm Soc Jap 2008;128:53-56.
Pharmacy
1. Of orphan children and other rare medications. Part 2. Klement A. Chin J Dig Dis 2005;6(4):189-192.
2. Dispensing ultra rare essential medication. Dooms, MM; Deckx, HP; Derijdt, TF. ASHP Midyear Clinical Meeting 2007;42.
3. The advantage of polymer addition to a non-ionic oil in water microemulsion for the dermal delivery of progesterone. Biruss, B; Valenta, C. Int J Pharm 2008;349:269-273.
Primary Carnitine Deficiency
1. Pharmacological rescue of carnitine transport in primary carnitine deficiency. Amat di San Filippo, C; Pasquali, M; Longo, N. Hum Mutat 2006;27(6):513-523.
2. Deficiency of the carnitine transporter (OCTN2) with partial N-acetylglutamate synthase (NAGS) deficiency. Hwu, W-L; Chien, Y-H; Tang, NLS; Law, L-K; Lin, C-Y; Lee, N-C. J Inherit Metab Dis 2007;30(5):816.
Retinal Injury
1. Retinal ischemic injury rescued by sodium 4-phenylbutyrate in a rat model. Jeng, Y-Y; Lin, N-T; Chang, P-H; Huang, Y-P; Pang, VF; Liu, C-H; Lin, C-T. Exp Eye Res 2007;84(3):486-492.
Sodium Absorption
1. Sodium 4-phenylbutyrate upregulates ENaC and sodium absorption in T84 cells. Iordache, C; Duszyk, M. Exp Cell Res 2007;313(2):305-311.
2. Modulation of epithelial sodium channel trafficking and function by sodium 4-phenylbutyrate in human nasal epithelial cells. Prulioere-Escabasse, V; Planoes, C; Escudier, E; Fanen, P; Coste, A; Clerici, C. J Biol Chem 2007;282(47):34048-34057.
Spinal Muscular Atrophy
1. The molecular-genetic background of spinal muscular atrophy: Diagnosis, prognosis and future treatments. Schwartz, M. Ugeskr Laeg 2005;167(7):745-748.
2. Mildly affected patients with spinal muscular atrophy are partially protected by an increased SMN2 copy number. Wirth, B; Brichta, L; Schrank, B; Lochmouller, H; Blick, S; Baasner, A; Heller, R. Hum Genet 2006;119(4):422-428.
3. Spinal muscular atrophy: from gene to therapy. Wirth, B; Brichta, L; Hahnen, E. Semin Pediatr Neurol 2006;13(2):121-131.
4. Spinal muscular atrophy and therapeutic prospects. Wirth, B; Brichta, L; Hahnen, E. Prog Mol Subcell Biol 2006;44:109-132.
5. Phenylbutyrate increases SMN gene expression in spinal muscular atrophy patients. Brahe, C; Vitali, T; Tiziano, FD; Angelozzi, C; Pinto, AM; Borgo, F; Moscato, U; Bertini, E; Mercuri, E; Neri, G. Eur J Hum Genet 2005;13(2):256-259.
6. Clinical trials in spinal muscular atrophy. Darras, BT; Kang, PB. Curr Opin Pediatr 2007;19(6):675-679.
7. Randomized, double-blind, placebo-controlled trial of phenylbutyrate in spinal muscular atrophy. Mercuri, E; Bertini, E; Messina, S; Solari, A; D'Amico, A; Angelozzi, C; Battini, R; Berardinelli, A; Boffi, P; Bruno, C; Cini, C; Colitto, F; Kinali, M; Minetti, C; Mongini, T; Morandi, L; Neri, G; Orcesi, S; Pane, M; Pelliccioni, M; Pini, A; Tiziano, FD; Villanova, M; Vita, G; Brahe, C. Neurology 2007;68(1):51-55.
8. Prospective evaluation of pre-symptomatic infants with spinal muscular atrophy: Implications for early therapeutic intervention. Swoboda, KJ. Annals of Neurology 2007;62(Suppl. 11):S2.
9. Survival motor neuron gene 2 silencing by DNA methylation correlates with spinal muscular atrophy disease severity and can be bypassed by histone deacetylase inhibition. Hauke, J; Riessland, M; Lunke, S; Eyoupoglu, IY; Bloumcke, I; El-Osta, A; Wirth, B; Hahnen, E. Hum Mol Genet 2009;18(2):304-317.
10.Drug treatment for spinal muscular atrophy types II and III. Bosboom, WM; Vrancken, AF; van den Berg, LH; Wokke, JH; Iannaccone, St. Cochrane Database Syst Rev 2009;1: CD006282.
11.A preliminary report on spinal muscular atrophy lymphoblastoid cell lines: are they an appropriate tool for drug screening? Dayangaoc-Erden, D; TopaloAlu, H; Erdem-Yurter, H. Adv Ther 2008;25(3):274-279.
Stability
1. Stability of sodium phenylbutyrate in two oral liquid vehicles. Caruthers RL. ASHP Midyear Clinical Meeting 2006;41.
2. Stability of extemporaneously prepared sodium phenylbutyrate oral suspensions. Caruthers, RL; Johnson, CE. Am J Health Syst Pharm 2007;64(14):1513-1515.
Synthesis
1. Asymmetric synthesis of unnatural L-amino acids using thermophilic aromatic L-amino acid transaminase. Cho, B-K; Seo, J-H; Kim, J; Lee, C-S; Kim, B-G. Biotechnology and Bioprocess Engineering 2006;11(4):299-305.
2. Preparation of enantiomerically pure (R)- and (S)-3-amino-3-phenyl-1- propanol via resolution with immobilized penicillin G acylase. Fadnavis, NW; Radhika, KR; Vedamayee, Devi A. Tetrahedron Asymmetry 2006;17(2):240-244.
3. Formulation and characterization of biodegradable chitosan films for topical application of terbinafine HC1. Ahmed, SM; Ibrahim, MA; Sarhan, HA; Amin, MA. Bull Pharm Sci 2007;30:111-129.
4. Creation of novel enantioselective lipases by SIMPLEX. Koga, Y; Yamane, T; Nakano, H. Methods Mol Biol 2007;375:165-181.
5. Highly enantioselective sequential hydrogenation of ethyl 2-oxo-4-arylbut-3-enoate to ethyl 2-hydroxy-4-arylbutyrate. Meng, Q; Zhu, L; Zhang, Z. J Org Chem 2008;73(18):7209-7212.
6. Synthesis of γ-amino acid esters by 1,4-addition of deprotonated α-aminonitriles and α-(alkylideneamino)nitriles to α,β-unsaturated esters. Bergner, I; Opatz, T. Synthesis 2007;918-928.
Transplantation
1. Attenuation of primary nonfunction for syngeneic islet graft using sodium 4-phenylbutyrate. Fu, S-H; Chen, S-T; Hsu, B R-S. Transplant Proc 2005;37(4):1830-1831.
2. The vast majority of bone-marrow-derived cells integrated into mdx muscle fibers are silent despite long-term engraftment. Wernig, G; Janzen, V; Schefer, R; Zweyer, M; Knauf, U; Hoegemeier, O; Mundegar, RR; Garbe, S; Stier, S; Franz, T; Wernig, M; Wernig, A. Proc Natl Acad Sci U S A 2005;102(33):11852-11857.
3. Neurological outcome in a patient with a null ornithine transcarbamylase genotype following liver transplantation at 6 weeks of age. Orfanelli, L; Greene, C; Glass, P; Gaillard, WD; Vezina, LG; Issacs, J; Higgs, J; Tuchman, M; Dunn, S; Berry, GT. Journal of Inherited Metabolic Disease 2006;29(Suppl. 1):31.
4. Enhancing engraftment of islets using perioperative sodium 4-phenylbutyrate. Hsu, BR-S; Chen, S-T; Fu, S-H. Int Immunopharmacol 2006;6(13-14):1952-1959.
Urea Cycle Disorders
1. Orphan drug: Carglumic acid for the treatment of urea cycle disorders. Dtsch Apoth Ztg 2005;145(1):44-46.
2. Sodium 4-phenylbutyrate protects against liver ischemia reperfusion injury by inhibition of endoplasmic reticulum-stress mediated apoptosis. Vilatoba, M; Eckstein, C; Bilbao, G; Smyth, CA; Jenkins, S; Thompson, JA; Eckhoff, DE; Contreras, JL. Surgery 2005;138(2):342-351.
3. Long-term outcome of urea cycle disorders. Bachmann, C. Acta Gastroenterol Belg 2005;68(4):466-468.
4. The pattern of intravenous drug administration during the transfer of critically ill children by a specialist transport team. Saha, AS; Langridge, P; Playfor, SD. Paediatr Anaesth 2006;16(10):1063-1067.
5. Successful extracorporeal treatment of a male with hyperammonaemic coma. Haller, M; Boulanger, AH-L; Sass, JO; Brandis, M; Zimmerhackl, LB. Nephrology Dialysis Transplantation 2005;20(2):453-455.
6. Outcomes among male OTC patients treated with ammonaps (R). Wuebbels, BH. Journal of Inherited Metabolic Disease 2006;29(Suppl. 1):129.
7. Urea cycle disorders in adult patients. Maillot, F; Crenn, P. Revue Neurologique (Paris) 2007;163(10):897-903.
8. A review of biochemical outcomes of adults with OTC deficiency. Bhattacharya, K; Briddon, A; Lee, PJ. Journal of Inherited Metabolic Disease 2007;30(Suppl. 1):82.
9. Cross-sectional multicenter study of patients with urea cycle disorders in the United States. Tuchman, M; Lee, B; Lichter-Konecki, U; Summar, ML; Yudkoff, M; Cederbaum, SD; Kerr, DS; Diaz, GA; Seashore, MR; Lee, H-S; McCarter, RJ; Krischer, JP; Batshaw, ML; Urea Cycle Disorders Consortium of the Rare Diseases Clinical Research Network. Mol Genet Metab 2008;94(4):397-402.
10.Hypothesis: proposals for the management of a neonate at risk of hyperammonaemia due to a urea cycle disorder. Leonard, JV; Ward, P; Martin, P; Morris, AAM. Eur J Pediatr 2008;167(3):305-309.
11.L-ornithine phenylacetate reduces arterial ammonia, improves brain osmolytes and reduces brain water in a bile duct ligated pat model of cirrhosis. Wright, GA; Davies, NA; Ytrebo, LM; Stadlbauer, V; Fuskevag, O-M; Zwingmann, C; Hodges, S; Jalan, R. Hepatology 2007;46(4, Suppl. S):604A-605A.
12.Preliminary data on adult patients with urea cycle disorders (UCD) in an open-label, switch-over, dose-escalation study comparing a new ammonia scavenger, glyceryl tri(4-phenylbutyrate) (HPN-100), to buphenyl (sodium phenylbutyrate (PBA). Lee, B; Garovoy, MR; Gargosky, SE; Berry, SA. Journal of Inherited Metabolic Disease 2008;31(Suppl. 1):91.
13.Sodium phenylbutyrate (Ammonaps) in the treatment of patients with urea cycle disorders - European post-approval data. Dobbelaere, DD. Journal of Inherited Metabolic Disease 2008;31(Suppl. 1):92.
1. Fantin, VR; Richon, VM. Mechanisms of resistance to histone deacetylase inhibitors and their therapeutic implications. Clin Cancer Res 2007;13(24):7237-7242.
Addiction
1. Romieu, P; Host, L; Gobaille, S; Sandner, G; Aunis, D; Zwiller, J. Histone deacetylase inhibitors decrease cocaine but not sucrose self-administration in rats. J Neurosci 2008;28(38):9342-9348.
Adrenoleukodystrophy
1. Gondcaille, C; Depreter, M; Fourcade, SU; Lecca, MR; Leclercq, S; Martin, PGP; Pineau, T; Cadepond, F; ElEtr, M; Bertrand, N; Beley, A; Duclos, S; De Craemer, D; Roels, F; Savary, SU; Bugaut, M. Phenylbutyrate up-regulates the adrenoleukodystrophy-related gene as a nonclassical peroxisome proliferator. J Cell Biol 2005;169(1):93-104.
Aging
1. Wagner, G. Towards a life prolonging pill? Small molecules with anti-ageing properties. Current Drug Targets 2006;7(11):1531-1537.
2. Burzynski SR. Aging: gene silencing or gene activation? Med Hypotheses 2005;64(1):201-208.
Alpha-Galactosidase Mutation
1. Yam, GH-F; Roth, J; Zuber, C. 4-Phenylbutyrate rescues trafficking incompetent mutant alpha-galactosidase A without restoring its functionality. Biochem Biophys Res Commun 2007;360(2):375-380.
ALS
1. Targets in ALS: designing multidrug therapies. Carri, MT; Grignaschi, G; Bendotti, C. Trends Pharmacol Sci 2006;27(5):267-273.
2. Additive neuroprotective effects of a histone deacetylase inhibitor and a catalytic antioxidant in a transgenic mouse model of amyotrophic lateral sclerosis. Petri, S; Kiaei, M; Kipiani, K; Chen, J; Calingasan, NY; Crow, JP; Beal, MF. Neurobiol Dis 2006;22(1):40-49.
3. Neuroprotective agents for clinical trials in ALS: a systematic assessment. Traynor, BJ; Bruijn, L; Conwit, R; Beal, F; O'Neill, G; Fagan, SC; Cudkowicz, ME. Neurology 2006;67(1):20-27.
4. Sodium phenylbutyrate prolongs survival and regulates expression of anti-apoptotic genes in transgenic amyotrophic lateral sclerosis mice. Ryu, H; Smith, K; Camelo, SI; Carreras, I; Lee, J; Iglesias, AH; Dangond, F; Cormier, KA; Cudkowicz, ME; Brown, RH; Ferrante, RJ. J Neurochem 2005;93(5):1087-1098.
5. Erratum: Sodium phenylbutyrate prolongs survival and regulates expression of anti-apoptotic genes in transgenic amyotrophic lateral sclerosis mice (Journal of Neurochemistry (2005) 93 (1087-1098)). Ryu, H; Smith, K; Camelo, SI; Carreras, I; Lee, J; Iglesias, AH; Dangond, F; Cormier, KA; Cudkowicz, ME; Brown, Jr RH; Ferrante, RJ. J Neurochem 2006;96(3):908.
6. Therapeutic targets for amyotrophic lateral sclerosis: Current treatments and prospects for more effective therapies. Bruijn, LI.; Cudkowicz, M. Expert Rev Neurother 2006;6(3):417-428.
7. Translating preclinical insights into effective human trials in ALS. DiBernardo, AB; Cudkowicz, ME. Biochim Biophys Acta - Mol Basis Dis 2006;1762(11-12):1139-1149.
8. Design, power, and interpretation of studies in the standard murine model of ALS. Scott, S; Kranz, JE; Cole, J; Lincecum, JM; Thompson, K; Kelly, N; Bostrom, A; Theodoss, J; Al-Nakhala, BM; Vieira, FG; Ramasubbu, J; Heywood, JA. Amyotroph Lateral Scler 2008;9(1):4-15.
9. Safety and does escalating study of oral sodium phenylbutyrate in subjects with ALS. Cudkowicz, ME; Andres, PL; Choudry, R; MacDonald, SA; Zhang, H; Schoenfeld, D; Ferrante, RJ. Neurology 2007;68(12, Suppl. 1):A90.
Anemia
1. A new generation of gamma globin gene inducers act on novel molecular and cellular targets. Perrine, S; Trudel, M; Castaneda, SA.; Mankidy, R; Boosalis, MS.; Mpollo, M-SEM; White, GL; Bohacek, R; Faller, DV. Blood 2006;108(11, Part 1):453A.
2. Advances in the management of sickle cell disease. Thompson, AA. Pediatr Blood Cancer 2006;46(5):533-539.
3. Fetal globin stimulant therapies in the beta-hemoglobinopathies: principles and current potential. Perrine, SP. Pediatr Ann 2008;37(5):339-346.
4. Cucurbitacin D upregulates fetal hemoglobin expression in K562 cells and human erythroid progenitors. Xing, H; Zhang, S; Koeffler, HP; Fung, MC. Blood 2007;110(11, Part 2):26B-27B.
5. Sodium phenyl butyrate downregulates endothelin-1 expression in cultured human endothelial cells: Relevance to sickle-cell disease. Odievre, M-H; Brun, M; Krishnamoorthy, R; Lapoumeroulie, C; Elion, J. American Journal of Hematology 2007;82(5):357-362.
6. Pulsed-dosing with oral sodium phenylbutyrate increases hemoglobin F in a patient with sickle cell anemia. Hines, P; Dover, GJ; Resar, LMS. Pediatr Blood Cancer 2008;50(2):357-359.
Anti-Parasite Activity
1. Scriptaid and suberoylanilide hydroxamic acid are histone deacetylase inhibitors with potent anti-Toxoplasma gondii activity in vitro. Strobl, JS; Cassell, M; Mitchell, SM; Reilly, CM; Lindsay, DS. J Parasitol 2007;93(3):694-700.
Arginase Induction
1. Arginase induction by sodium phenylbutyrate in mouse tissues and human cell lines. Kern, RM; Yang, Z; Kim, PS; Grody, WW; Iyer, RK; Cederbaum, SD. Mol Genet Metab 2007;90(1):37-41.
Argininosuccinate Lyase Deficiency
1. Potassium retention in patients treated for argininosuccinate lyase deficiency. Singh, RH; Acosta, PB; Kennedy, MP; Longo, N; Elsas, LJ. Journal of Inherited Metabolic Disease 2006;29(Suppl. 1):48.
2. Sustained Engraftment and Tissue Enzyme Activity After Liver Cell Transplantation for Argininosuccinate Lyase Deficiency. Stephenne, X; Najimi, M; Sibille, C; Nassogne, M; Smets, F; Sokal, EM. Gastroenterology 2006;130(4):1317-1323.
Bladder Dysfunction
1. A novel target for boo-induced bladder dysfunction. Sawada, N; Yao, J; Hiramatsu, N; Hayakawa, K; Zakoji, H; Kobayashi, H; Mochizuki, T; Araki, I; Takeda, M; Kitamura, M. European Urology Supplements 2008;7(3):265.
Chemical Analysis
1. Separation and determination of optical isomers of ethyl small alpha -hydroxyphenylbutyrate by chiral HPLC. Zhang, YY; Chen, GC. Fenxi Ceshi Xuebao 2005;24(1):109-110.
2. Metabolite identification using a nanoelectrospray LC-EC-array-MS integrated system. Schiavo, S; Ebbel, E; Sharma, S; Matson, W; Kristal, BS; Hersch, S; Vouros, P. Anal Chem 2008;80(15):5912-5923.
3. A two-site ELISA can quantify upregulation of SMN protein by drugs for spinal muscular atrophy. Nguyen, M; Humphrey, E; Lam, LT; Fuller, HR; Lynch, TA; Sewry, CA; Goodwin, PR; Mackenzie, AE; Morris, GE. Neurology 2008;71(22):1757-1763.
4. Detection of triglyceride using an iridium nano-particle catalyst based amperometric biosensor. Liao, W-Y; Liu, C-C; Chou, T-C. Analyst 2008;133(12):1757-1763.
5. Multiple-layer substrate zymography for detection of several enzymes in a single sodium dodecyl sulfate gel. Choi, N-S; Kim, B-H; Park, C-S; Han, YJ; Lee, HW; Choi, JH; Lee, S-G; Song, JJ. Anal Biochem 2009;386(1):121-122.
Cholestatic Diseases
1. Levels of plasma membrane expression in progressive and benign mutations of the bile salt export pump (Bsep/Abcb11) correlate with severity of cholestatic diseases. Lam, P; Pearson, CL; Soroka, CJ; Xu, S; Mennone, A; Boyer, JL. Am J Physiol Cell Physiol 2007;293(5):C1709-1716.
2. Chemical chaperones partially reverse the mis-processing of a BRIC2 mutant of the bile salt export pump, ABCB11. Lam, P; Soroka, CJ.; Boyer, JL. Hepatology 2007;46(4, Suppl. S):330A.
3. 4-phenylbutyrate enhances the cell surface expression and the transport capacity of wild-type and mutated bile salt export pumps. Hayashi, H; Sugiyama, Y. Hepatology 2007;45(6):1506-1516.
4. Short-chain ubiquitination is associated with the degradation rate of a cell-surface-resident bile salt export pump (BSEP/ABCB11). Hayashi, H; Sugiyama, Y. Mol Pharmacol 2009;75(1):143-150.
Citrulline Metabolism
1. Manipulation of citrulline availability in humans. Rougoe, C; Des Robert, C; Robins, A; Le Bacquer, O; Volteau, C; De La Cochetioere, M-F; Darmaun, D. Am J Physiol Gastrointest Liver Physiol 2007;293(5):G1061-1067.
Computer Modeling
1. Toward the computational design of diastereomeric resolving agents: an experimental and computational study of 1-phenylethylammonium-2-phenylacetate derivatives. Karamertzanis, PG; Anandamanoharan, PR; Fernandes, P; Cains, PW; Vickers, M; Tocher, DA; Florence, AJ; Price, SL. J Phys Chem B 2007;111(19):5326-5336.
Cystic Fibrosis
1. Pharmacological induction of CFTR function in patients with cystic fibrosis: mutation-specific therapy. Kerem, E. Pediatr Pulmonol 2005;40(3):183-196.
2. Novel short chain fatty acids restore chloride secretion in cystic fibrosis. Nguyen, TD; Kim, U-S; Perrine, SP. Biochem Biophys Res Commun 2006;342(1):245-252.
3. Selective inhibition of endoplasmic reticulum-associated degradation rescues DeltaF508-cystic fibrosis transmembrane regulator and suppresses interleukin-8 levels: therapeutic implications. Vij, N; Fang, S; Zeitlin, PL. J Biol Chem 2006;281(25):17369-17378.
4. Mutation specific therapy in CF. Kerem, E. Pediatr Respir Rev 2006;7(Suppl. 1):S166-S169.
5. Pharmacoproteomics of 4-phenylbutyrate-treated IB3-1 cystic fibrosis bronchial epithelial cells. Singh, OV; Vij, N; Mogayzel, PJ; Jozwik, C; Pollard, HB; Zeitlin, PL. J Proteome Res 2006;5(3):562-571.
6. Cystic fibrosis transmembrane regulator protein mutations: 'Class' opportunity for novel drug innovation. MacDonald, KD; McKenzie, KR; Zeitlin, PL. Pediatr Drugs 2007;9(1):1-10.
7. Chemical rescue of deltaF508-CFTR mimics genetic repair in cystic fibrosis bronchial epithelial cells. Singh, OV; Pollard, HB; Zeitlin, PL. Mol Cell Proteomics 2008;7(6):1099-1110.
8. Proinflammatory effect of sodium 4-phenylbutyrate in deltaF508-cystic fibrosis transmembrane conductance regulator lung epithelial cells: involvement of extracellular signal-regulated protein kinase 1/2 and c-Jun-NH2-terminal kinase signaling. Roque, T; Boncoeur, E; Saint-Criq, V; Bonvin, E; Clement, A; Tabary, O; Jacquot, J. J Pharmacol Exp Ther 2008;326(3):949-956.
Denervating Diseases
1. New treatments for denervating diseases. Pleasure D. J Child Neurol 2005;20(3):258-262.
Endoplasmic Reticulum Stress
1. Mutant tamm-horsfall glycoprotein accumulation in endoplasmic reticulum induces apoptosis reversed by colchicine and sodium 4-phenylbutyrate. Choi, SW; Ryu, OH; Choi, SJ; Song, IS; Bleyer, AJ; Hart, TC. J Am Soc Nephrol 2005;16(10):3006-3014.
2. Suppressive effects of 4-phenylbutyrate on the aggregation of Pael receptors and endoplasmic reticulum stress. Kubota, K; Niinuma, Y; Kaneko, M; Okuma, Y; Sugai, M; Omura, T; Uesugi, M; Uehara, T; Hosoi, T; Nomura, Y. J Neurochem 2006;97(5):1259-1268.
3. Chemical chaperones reduce endoplasmic reticulum stress and prevent mutant HFE aggregate formation. de Almeida, SF; Picarote, G; Fleming, JV; Carmo-Fonseca, M; Azevedo, JE; de Sousa, M. J Biol Chem 2007;282(38):27905-27912.
4. KLF5 plays an important role in endoplasmic reticulum stress response in both cardiovascular and metabolic system. Eguchi, K; Manabe, I; Shen, H; Ohishi, Y; Fujiu, K; Takeda, N; Nagai, R. Circulation 2007;116(16, Suppl. S):44.
5. Involvement of endoplasmic reticulum stress in palmitate-induced endothelial cell apoptosis. Jang, HJ; Jung, SH; Park, TJ; Jo, YE; Kim, YK; Jung, JG; Choi, S-E; Kang, Y; Kim, HJ; Kim, DJ; Lee, KW. Diabetes 2007;56(Suppl. 1):A508-A509.
6. Involvement of hypoxia-triggered endoplasmic reticulum stress in outlet obstruction-induced apoptosis in the urinary bladder. Sawada, N; Yao, J; Hiramatsu, N; Hayakawa, K; Araki, I; Takeda, M; Kitamura, M. Lab Invest 2008;88(5):553-563.7. BAX inhibitor-1 modulates endoplasmic reticulum stress-mediated programmed cell death in Arabidopsis. Watanabe, N; Lam, E. J Biol Chem 2008;283(6):3200-3210.
8. Sodium 4-phenylbutyrate acts as a chemical chaperone on misfolded myocilin to rescue cells from endoplasmic reticulum stress and apoptosis. Yam, GH-F; Gaplovska-Kysela, K; Zuber, C; Roth, J. Invest Ophthalmol Vis Sci 2007;48(4):1683-1690.
9. 4-PBA(4-phenylbutyrate) reduced palmitate-induced endoplasmic reticulum stress and apoptosis in HUVEC. Jang, H-J; Jung, J; Ha, E-S; Choi, S-E; Jung, S; Kim, H; Kim, D; Kang, Y; Lee, K-W. Diabetologia 2007;50(Suppl. 1):S278.
10.Oxidative stress induces the endoplasmic reticulum stress and facilitates inclusion formation in cultured cells. Hanada, S; Harada, M; Kumemura, H; Bishr Omary, M; Koga, H; Kawaguchi, T; Taniguchi, E; Yoshida, T; Hisamoto, T; Yanagimoto, C; Maeyama, M; Ueno, T; Sata, M. J Hepatol 2007;47(1):93-102.
General
1. Activity of the CHMP. Throm, S. Pharm Ind 2005;67(1):57-63.
2. Activity of digestive enzymes in Scophthalmus maximus. Fu XH; Sun M; Sun SC. Journal of Fishery Sciences of China 2005;12(1):26-32.
3. Novel strategy for protein exploration: high-throughput screening assisted with fuzzy neural network. Kato, R; Nakano, H; Konishi, H; Kato, K; Koga, Y; Yamane, T; Kobayashi, T; Honda, H. J Mol Biol 2005;351(3):683-692.
4. Molecular cloning of enantioselective ester hydrolase from Bacillus pumilus DBRL-191. Rasool, S; Johri, S; Riyaz-ul-Hassan, S; Maqbool, Q-u-A; Verma, V; Koul, S; Taneja, SC; Qazi, GN. FEMS Microbiol Lett 2005;249(1):113-120.
5. Modular assembly using sequential palladium coupling gives easy access to the SMoC class of cellular transporters. Rebstock, A-S; Visintin, C; Leo, E; Posada, CG; Kingsbury, SR; Williams, GH; Stoeber, K; Selwood, DL. ChemBioChem 2008;9(11):1787-1796.
6. Waste frying oil as substrate for lipase production by Geotrichum candidum strains. Rywinska, A; Witkowska, D; Juszczyk, P; Rymowicz, W; Kita, A. Polish Journal of Environmental Studies 2008;17(6):925-931.
7. The application of calcined natural dolomitic rock as a solid base catalyst in triglyceride transesterification for biodiesel synthesis. Wilson, K; Hardacre, C; Lee, AF; Montero, JM; Shellard, L. Green Chemistry 2008;10(6):654-659.
Glaucoma
1. Russell body formation and apoptosis in myocilin-caused primary open-angle glaucoma: rescue by the chemical chaperone sodium 4-phenylbutyrate. Roth, J; Yam, GH-F; Gaplovska-Kysela, K; Zuber, C. FASEB Journal 2007;21(5):A182.
Glycosylphosphatidyl Inositol Deficiency
1. Targeted molecular therapy for inherited glycosylphosphatidylinositol deficiency. Almeida, AM.; Murakami, Y; Baker, A; Maeda, Y; Roberts, I; Kinoshita, T; Layton, DM; Karadimitris, A. Blood 2006;108(11, Part 1):148A.
2. Targeted therapy for inherited GPI deficiency. Almeida, AM; Murakami, Y; Baker, A; Maeda, Y; Roberts, IAG; Kinoshita, T; Layton, DM; Karadimitris, A. New Engl J Med 2007;356(16):1641-1647.
Huntington’s Disease
1. Genome-wide expression profiling of human blood reveals biomarkers for Huntington's disease. Borovecki, F; Lovrecic, L; Zhou, J; Jeong, H; Then, F; Rosas, HD; Hersch, SM; Hogarth, P; Bouzou, B; Jensen, RV; Krainc, D. Proc Natl Acad Sci U S A 2005;102(31):11023-11028.
2. Neuroprotective effects of phenylbutyrate in the N171-82Q transgenic mouse model of Huntington's disease. Gardian, G; Browne, SE; Choi, D-K; Klivenyi, P; Gregorio, J; Kubilus, JK; Ryu, H; Langley, B; Ratan, RR; Ferrante, RJ; Beal, MF. J Biol Chem 2005;280(1):556-563.
3. A transcriptional report card from the peripheral blood: Can it measure disease progression in Huntington's disease? Tabchy, A; Housman, D. European Journal of Human Genetics 2006;14(6):649-650.
4. Sodium phenylbutyrate in Huntington's disease: a dose-finding study. Hogarth, P; Lovrecic, L; Krainc, D. Mov Disord 2007;22(13):1962-1964.
Inborn Errors in Metabolism
1. Inborn errors of metabolism: The flux from Mendelian to complex diseases. Lanpher, B.; Brunetti-Pierri, N.; Lee, B. Nat Rev Gen 2006;7(6):449-460.
2. Functional and trafficking defects in ATP binding cassette A3 mutants associated with respiratory distress syndrome. Cheong, N; Madesh, M; Gonzales, LW; Zhao, M; Yu, K; Ballard, PL; Shuman, H. J Biol Chem 2006;281(14):9791-9800.
3. Pharmacological treatment of congenital metabolic disorders. Campino VA. Aten Farm 2006;8(1):39-46.
4. A newborn infant with hyperventilation. Lindemann, R; Myhre, MC; Bakken, M; Fugelseth, D; Rustad, CF; Woldseth, B. Tidsskr Nor Laegeforen 2008;128(13):1535-1536.
5. Failure of bone morphogenetic protein receptor trafficking in pulmonary arterial hypertension: potential for rescue. Sobolewski, A; Rudarakanchana, N; Upton, PD; Yang, J; Crilley, TK; Trembath, RC; Morrell, NW. Hum Mol Genet 2008;17(20):3180-3190.
Klinefelter Syndrome
1. An OTC deficiency 'phenocopy' in association with Klinefelter syndrome. Swarts, L; Leisegang, F; Owen, EP; Henderson, HE. J Inherit Metab Dis 2007;30(1):101.
Leucine Metabolism
1. Acute depletion of plasma glutamine increases leucine oxidation in prednisone-treated humans. Le Bacquer, O; Mauras, N; Welch, S; Haymond, M; Darmaun, D. Clin Nutr 2007;26(2):231-238.
2. Phenylbutyrate reduces plasma leucine concentrations without affecting the flux of leucine. Marini, JC; Lanpher, B; Scaglia, F; Carter, S; Garlick, PJ; Jahoor, F; Lee, B. FASEB Journal 2007;21(5):A335.
Lipoprotein Receptor
1. Rescue of the low density lipoprotein receptor 2A mutant G544V by 4-phenylbutyrate. Tveten, K; Holla, OL; Ranheim, T; Berge, KE; Leren, TP; Kulseth, MA. Molecular chaperones and the heat shock response 2006;187.
2. 4-Phenylbutyrate restores the functionality of a misfolded mutant low-density lipoprotein receptor. Tveten, K; Holla, OL; Ranheim, T; Berge, KE; Leren, TP; Kulseth, MA. FEBS J 2007;274(8):1881-1893.
Lysinuric Protein Intolerance
1. Nutrient intake in lysinuric protein intolerance. Tanner, LM; Noantoe-Salonen, K; Venetoklis, J; Kotilainen, S; Niinikoski, H; Huoponen, K; Simell, O. J Inherit Metab Dis 2007;30(5):716-721.
Mechanism of Action
1. Does acute glutamine depletion enhance the response of glutamine synthesis to fasting in muscle in adult and old rats? Meynial-Denis, D; Verdier, L; Mignon, M; Leclerc, J-NA; Bayle, GU; Darmaun, D. Clin Nutr 2005;24(3):398-406.
2. Activation of STAT-3 in CF epithelial cell by Sodium 4-Phenylbutyrate. Suaud, L; Rubenstein, RC. FASEB Journal 2005;19(4, Suppl. S, Part 1):A658.
3. Cellular and molecular responses to cerebral hypoxia: Involvement of ER in neuronal death. Nomura, Y. Journal of Pharmacological Sciences 2005;97(Suppl. 1):6P.
4. DNA methyltransferase (DNMT)-independent effects of DNMT inhibitors on gene expression. Jiemjit, A; Baylin, S; Herman, J; Gore, S. Proceedings of the American Association for Cancer Research Annual Meeting 2005;46(Suppl. S):427.
5. Mutational analysis of 6-aminohexanoate-dimer hydrolase: Relationship between nylon oligomer hydrolytic and esterolytic activities. Ohki, T; Wakitani, Y; Takeo, M; Yasuhira, K; Shibata, N; Higuchi, Y; Negoro, S. FEBS Lett 2006;580(21):5054-5058.
6. Anti-inflammatory effects of short chain fatty acids in IFN-gamma-stimulated RAW 264.7 murine macrophage cells: involvement of NF-kappaB and ERK signaling pathways. Park, J-S; Lee, E-J; Lee, J-C; Kim, W-K; Kim, H-S. Int Immunopharmacol 2007;7(1):70-77.
7. Multiple molecular chaperone-mediated pharmacologic rescue of Delta F508-CFTR from ERAD. Singh, OV; Zeitlin, PL. FASEB Journal 2007;21(5):A420.
8. Optimum immobilization of Candida rugosa lipase on Celite by RSM. Chang SF; Chang SW; Yen YH; Shieh CJ. Applied Clay Science 2007;37(1/2):67-73.
9. Manipulating the quality control pathway in transfected cells: low temperature allows rescue of secretion-defective fibrinogen mutants. Vu, D; Di Sanza, C; Neerman-Arbez, M. Haematologica 2008;93(2):224-231.
10.Pro-inflammatory effect of sodium 4-phenylbutyrate in DeltaF508-CFTR lung cells: Involvement of ERK1/2 and JNK signaling. Roque, T; Boncoeur, E; Saint-Criq, V; Bonvin, E; Clement, A; Tabary, O; Jacquot, J. Rev Mal Respir 2008;25(9):1186.
Metabolism
1. Phenylacetate catabolism in Rhodococcus sp. strain RHA1: a central pathway for degradation of aromatic compounds. Navarro-Llorens, JM; Patrauchan, MA; Stewart, GR; Davies, JE; Eltis, LD; Mohn, WW. J Bacteriol 2005;187(13):4497-4504.
2. Microbial reduction of ethyl 2-oxo-4-phenylbutyrate. Searching for R-enantioselectivity. New access to the enalapril like ACE inhibitors. de, Lacerda PSB; Ribeiro, JB; Leite, SGF; Ferrara, MA; Coelho, RB; Bon, EPS; da, Silva Lima EL; Antunes, OAC. Tetrahedron Asymmetry 2006;17(8):1186-1188.
3. Regio- and enantio selective reduction of methyleneketoesters mediated by Saccharomyces cerevisiae. Clososki, GC; Milagre, CDF; Moran, PJS; Rodrigues, JAR. Journal of Molecular Catalysis B Enzymatic 2007;48(3-4):70-76.
4. Modulation of the activity of newly synthesized human phenylalanine hydroxylase mutant proteins by low-molecular-weight compounds. Nascimento, C; Leandro, J; Tavares de Almeida, I; Leandro, P. Protein J 2008;27(6):392-400.
5. Effect of ionic liquid BMIM PF6 on asymmetric reduction of ethyl 2-oxo-4-phenylbutyrate by Saccharomyces cerevisiae. Shi, Y-G; Fang, Y; Ren, Y-P; Wu, H-P; Guan, H-L. J Ind Microbiol Biotechnol 2008;35(11):1419-1424.
N-acetylglutamate Synthase Deficiency
1. Misleading diagnosis of partial N-acetylglutamate synthase deficiency based on enzyme measurement corrected by mutation analysis. Heckmann, M; Wermuth, B; Heberle, J; Koch, HG; Gortner, L; Kreuder, JG. Acta Paediatr 2005;94(1):121-124.
Neuroprotection
1. Synergistic neuroprotective effects of lithium and valproic acid or other histone deacetylase inhibitors in neurons: roles of glycogen synthase kinase-3 inhibition. Leng, Y; Liang, M-H; Ren, M; Marinova, Z; Leeds, P; Chuang, D-M. J Neurosci 2008;28(10):2576-2588.
2. HDAC inhibition amplifies gap junction communication in neural progenitors: potential for cell-mediated enzyme prodrug therapy. Khan, Z; Akhtar, M; Asklund, T; Juliusson, B; Almqvist, PM; Ekstroem, TJ. Exp Cell Res 2007;313(13):2958-2967.
3. Endogenous alpha-synuclein is induced by valproic acid through histone deacetylase inhibition and participates in neuroprotection against glutamate-induced excitotoxicity. Leng, Y; Chuang, D-M. J Neurosci 2006;26(28):7502-7512.
4. Phenylbutyrate modulates multiple neuronal survival gene expression in SMA cells. Ganta, M; Grzeschik, SM; Heavlin, WD; Wang, CH. Annals of Neurology 2006;60(Suppl. 10):S140.
Parkinson’s Disease
1. Neurodegeneration of mouse nigrostriatal dopaminergic system induced by repeated oral administration of rotenone is prevented by 4-phenylbutyrate, a chemical chaperone. Inden, M; Kitamura, Y; Takeuchi, H; Yanagida, T; Takata, K; Kobayashi, Y; Taniguchi, T; Yoshimoto, K; Kaneko, M; Okuma, Y; Taira, T; Ariga, H; Shimohama, S. J Neurochem 2007;101(6):1491-1504.
2. Neuroprotective effect of chemical chaperone 4-PBA on rotenone-induced dopaminagic neurodegeneration. Tamaki, A; Yanagida, T; Kitamura, Y; Takata, K; Taniguchi, T. Journal of Pharmacological Sciences 2008;106(suppl. 1):233P.
3. Translational research of parkinsonian therapeutics. Kitamura, Y; Taniguchi, T; Ariga, H. J Pharm Soc Jap 2008;128:53-56.
Pharmacy
1. Of orphan children and other rare medications. Part 2. Klement A. Chin J Dig Dis 2005;6(4):189-192.
2. Dispensing ultra rare essential medication. Dooms, MM; Deckx, HP; Derijdt, TF. ASHP Midyear Clinical Meeting 2007;42.
3. The advantage of polymer addition to a non-ionic oil in water microemulsion for the dermal delivery of progesterone. Biruss, B; Valenta, C. Int J Pharm 2008;349:269-273.
Primary Carnitine Deficiency
1. Pharmacological rescue of carnitine transport in primary carnitine deficiency. Amat di San Filippo, C; Pasquali, M; Longo, N. Hum Mutat 2006;27(6):513-523.
2. Deficiency of the carnitine transporter (OCTN2) with partial N-acetylglutamate synthase (NAGS) deficiency. Hwu, W-L; Chien, Y-H; Tang, NLS; Law, L-K; Lin, C-Y; Lee, N-C. J Inherit Metab Dis 2007;30(5):816.
Retinal Injury
1. Retinal ischemic injury rescued by sodium 4-phenylbutyrate in a rat model. Jeng, Y-Y; Lin, N-T; Chang, P-H; Huang, Y-P; Pang, VF; Liu, C-H; Lin, C-T. Exp Eye Res 2007;84(3):486-492.
Sodium Absorption
1. Sodium 4-phenylbutyrate upregulates ENaC and sodium absorption in T84 cells. Iordache, C; Duszyk, M. Exp Cell Res 2007;313(2):305-311.
2. Modulation of epithelial sodium channel trafficking and function by sodium 4-phenylbutyrate in human nasal epithelial cells. Prulioere-Escabasse, V; Planoes, C; Escudier, E; Fanen, P; Coste, A; Clerici, C. J Biol Chem 2007;282(47):34048-34057.
Spinal Muscular Atrophy
1. The molecular-genetic background of spinal muscular atrophy: Diagnosis, prognosis and future treatments. Schwartz, M. Ugeskr Laeg 2005;167(7):745-748.
2. Mildly affected patients with spinal muscular atrophy are partially protected by an increased SMN2 copy number. Wirth, B; Brichta, L; Schrank, B; Lochmouller, H; Blick, S; Baasner, A; Heller, R. Hum Genet 2006;119(4):422-428.
3. Spinal muscular atrophy: from gene to therapy. Wirth, B; Brichta, L; Hahnen, E. Semin Pediatr Neurol 2006;13(2):121-131.
4. Spinal muscular atrophy and therapeutic prospects. Wirth, B; Brichta, L; Hahnen, E. Prog Mol Subcell Biol 2006;44:109-132.
5. Phenylbutyrate increases SMN gene expression in spinal muscular atrophy patients. Brahe, C; Vitali, T; Tiziano, FD; Angelozzi, C; Pinto, AM; Borgo, F; Moscato, U; Bertini, E; Mercuri, E; Neri, G. Eur J Hum Genet 2005;13(2):256-259.
6. Clinical trials in spinal muscular atrophy. Darras, BT; Kang, PB. Curr Opin Pediatr 2007;19(6):675-679.
7. Randomized, double-blind, placebo-controlled trial of phenylbutyrate in spinal muscular atrophy. Mercuri, E; Bertini, E; Messina, S; Solari, A; D'Amico, A; Angelozzi, C; Battini, R; Berardinelli, A; Boffi, P; Bruno, C; Cini, C; Colitto, F; Kinali, M; Minetti, C; Mongini, T; Morandi, L; Neri, G; Orcesi, S; Pane, M; Pelliccioni, M; Pini, A; Tiziano, FD; Villanova, M; Vita, G; Brahe, C. Neurology 2007;68(1):51-55.
8. Prospective evaluation of pre-symptomatic infants with spinal muscular atrophy: Implications for early therapeutic intervention. Swoboda, KJ. Annals of Neurology 2007;62(Suppl. 11):S2.
9. Survival motor neuron gene 2 silencing by DNA methylation correlates with spinal muscular atrophy disease severity and can be bypassed by histone deacetylase inhibition. Hauke, J; Riessland, M; Lunke, S; Eyoupoglu, IY; Bloumcke, I; El-Osta, A; Wirth, B; Hahnen, E. Hum Mol Genet 2009;18(2):304-317.
10.Drug treatment for spinal muscular atrophy types II and III. Bosboom, WM; Vrancken, AF; van den Berg, LH; Wokke, JH; Iannaccone, St. Cochrane Database Syst Rev 2009;1: CD006282.
11.A preliminary report on spinal muscular atrophy lymphoblastoid cell lines: are they an appropriate tool for drug screening? Dayangaoc-Erden, D; TopaloAlu, H; Erdem-Yurter, H. Adv Ther 2008;25(3):274-279.
Stability
1. Stability of sodium phenylbutyrate in two oral liquid vehicles. Caruthers RL. ASHP Midyear Clinical Meeting 2006;41.
2. Stability of extemporaneously prepared sodium phenylbutyrate oral suspensions. Caruthers, RL; Johnson, CE. Am J Health Syst Pharm 2007;64(14):1513-1515.
Synthesis
1. Asymmetric synthesis of unnatural L-amino acids using thermophilic aromatic L-amino acid transaminase. Cho, B-K; Seo, J-H; Kim, J; Lee, C-S; Kim, B-G. Biotechnology and Bioprocess Engineering 2006;11(4):299-305.
2. Preparation of enantiomerically pure (R)- and (S)-3-amino-3-phenyl-1- propanol via resolution with immobilized penicillin G acylase. Fadnavis, NW; Radhika, KR; Vedamayee, Devi A. Tetrahedron Asymmetry 2006;17(2):240-244.
3. Formulation and characterization of biodegradable chitosan films for topical application of terbinafine HC1. Ahmed, SM; Ibrahim, MA; Sarhan, HA; Amin, MA. Bull Pharm Sci 2007;30:111-129.
4. Creation of novel enantioselective lipases by SIMPLEX. Koga, Y; Yamane, T; Nakano, H. Methods Mol Biol 2007;375:165-181.
5. Highly enantioselective sequential hydrogenation of ethyl 2-oxo-4-arylbut-3-enoate to ethyl 2-hydroxy-4-arylbutyrate. Meng, Q; Zhu, L; Zhang, Z. J Org Chem 2008;73(18):7209-7212.
6. Synthesis of γ-amino acid esters by 1,4-addition of deprotonated α-aminonitriles and α-(alkylideneamino)nitriles to α,β-unsaturated esters. Bergner, I; Opatz, T. Synthesis 2007;918-928.
Transplantation
1. Attenuation of primary nonfunction for syngeneic islet graft using sodium 4-phenylbutyrate. Fu, S-H; Chen, S-T; Hsu, B R-S. Transplant Proc 2005;37(4):1830-1831.
2. The vast majority of bone-marrow-derived cells integrated into mdx muscle fibers are silent despite long-term engraftment. Wernig, G; Janzen, V; Schefer, R; Zweyer, M; Knauf, U; Hoegemeier, O; Mundegar, RR; Garbe, S; Stier, S; Franz, T; Wernig, M; Wernig, A. Proc Natl Acad Sci U S A 2005;102(33):11852-11857.
3. Neurological outcome in a patient with a null ornithine transcarbamylase genotype following liver transplantation at 6 weeks of age. Orfanelli, L; Greene, C; Glass, P; Gaillard, WD; Vezina, LG; Issacs, J; Higgs, J; Tuchman, M; Dunn, S; Berry, GT. Journal of Inherited Metabolic Disease 2006;29(Suppl. 1):31.
4. Enhancing engraftment of islets using perioperative sodium 4-phenylbutyrate. Hsu, BR-S; Chen, S-T; Fu, S-H. Int Immunopharmacol 2006;6(13-14):1952-1959.
Urea Cycle Disorders
1. Orphan drug: Carglumic acid for the treatment of urea cycle disorders. Dtsch Apoth Ztg 2005;145(1):44-46.
2. Sodium 4-phenylbutyrate protects against liver ischemia reperfusion injury by inhibition of endoplasmic reticulum-stress mediated apoptosis. Vilatoba, M; Eckstein, C; Bilbao, G; Smyth, CA; Jenkins, S; Thompson, JA; Eckhoff, DE; Contreras, JL. Surgery 2005;138(2):342-351.
3. Long-term outcome of urea cycle disorders. Bachmann, C. Acta Gastroenterol Belg 2005;68(4):466-468.
4. The pattern of intravenous drug administration during the transfer of critically ill children by a specialist transport team. Saha, AS; Langridge, P; Playfor, SD. Paediatr Anaesth 2006;16(10):1063-1067.
5. Successful extracorporeal treatment of a male with hyperammonaemic coma. Haller, M; Boulanger, AH-L; Sass, JO; Brandis, M; Zimmerhackl, LB. Nephrology Dialysis Transplantation 2005;20(2):453-455.
6. Outcomes among male OTC patients treated with ammonaps (R). Wuebbels, BH. Journal of Inherited Metabolic Disease 2006;29(Suppl. 1):129.
7. Urea cycle disorders in adult patients. Maillot, F; Crenn, P. Revue Neurologique (Paris) 2007;163(10):897-903.
8. A review of biochemical outcomes of adults with OTC deficiency. Bhattacharya, K; Briddon, A; Lee, PJ. Journal of Inherited Metabolic Disease 2007;30(Suppl. 1):82.
9. Cross-sectional multicenter study of patients with urea cycle disorders in the United States. Tuchman, M; Lee, B; Lichter-Konecki, U; Summar, ML; Yudkoff, M; Cederbaum, SD; Kerr, DS; Diaz, GA; Seashore, MR; Lee, H-S; McCarter, RJ; Krischer, JP; Batshaw, ML; Urea Cycle Disorders Consortium of the Rare Diseases Clinical Research Network. Mol Genet Metab 2008;94(4):397-402.
10.Hypothesis: proposals for the management of a neonate at risk of hyperammonaemia due to a urea cycle disorder. Leonard, JV; Ward, P; Martin, P; Morris, AAM. Eur J Pediatr 2008;167(3):305-309.
11.L-ornithine phenylacetate reduces arterial ammonia, improves brain osmolytes and reduces brain water in a bile duct ligated pat model of cirrhosis. Wright, GA; Davies, NA; Ytrebo, LM; Stadlbauer, V; Fuskevag, O-M; Zwingmann, C; Hodges, S; Jalan, R. Hepatology 2007;46(4, Suppl. S):604A-605A.
12.Preliminary data on adult patients with urea cycle disorders (UCD) in an open-label, switch-over, dose-escalation study comparing a new ammonia scavenger, glyceryl tri(4-phenylbutyrate) (HPN-100), to buphenyl (sodium phenylbutyrate (PBA). Lee, B; Garovoy, MR; Gargosky, SE; Berry, SA. Journal of Inherited Metabolic Disease 2008;31(Suppl. 1):91.
13.Sodium phenylbutyrate (Ammonaps) in the treatment of patients with urea cycle disorders - European post-approval data. Dobbelaere, DD. Journal of Inherited Metabolic Disease 2008;31(Suppl. 1):92.