寻药
PTC124药物是由PTC Therapeutics公司生产的一种能够修正遗传突变产生的副作用的药物。它能够“欺骗”病人的细胞使其产生有功能的产物。
有关的最新进展：
1: Mechanism of PTC124 activity in cell-based luciferase assays of nonsense codon suppression.
Auld DS, Thorne N, Maguire WF, Inglese J.
Proc Natl Acad Sci U S A. 2009 Feb 10. [Epub ahead of print]
PMID: *********** [PubMed - as supplied by publisher]
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2: Drug developers aim to treat cystic fibrosis through disease modification.
Thompson CA.
Am J Health Syst Pharm. 2008 Dec 15;65(24):2310-2. No abstract available.
PMID: *********** [PubMed - indexed for MEDLINE]
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3: Gunvalson decision sends shockwaves through industry.
Allison M.
Nat Biotechnol. 2008 Nov;26(11):1201-2. No abstract available.
PMID: *********** [PubMed - indexed for MEDLINE]
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4: New approaches to treatment of primary immunodeficiencies: fixing mutations with chemicals.
Hu H, Gatti RA.
Curr Opin Allergy Clin Immunol. 2008 Dec;8(6):540-6.
PMID: *********** [PubMed - in process]
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5: Ignoring the nonsense: a phase II trial in cystic fibrosis.
Hyde SC, Gill DR.
Lancet. 2008 Aug 30;372(9640):691-2. Epub 2008 Aug 20. No abstract available.
PMID: *********** [PubMed - indexed for MEDLINE]
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6: Effectiveness of PTC124 treatment of cystic fibrosis caused by nonsense mutations: a prospective phase II trial.
Kerem E, Hirawat S, Armoni S, Yaakov Y, Shoseyov D, Cohen M, Nissim-Rafinia M, Blau H, Rivlin J, Aviram M, Elfring GL, Northcutt VJ, Miller LL, Kerem B, Wilschanski M.
Lancet. 2008 Aug 30;372(9640):719-27. Epub 2008 Aug 20.
PMID: *********** [PubMed - indexed for MEDLINE]
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7: Pharmacological probing of type 1 autism.
Manev H, Manev R.
J Autism Dev Disord. 2008 Aug;38(7):1400-1. Epub 2008 May 30. No abstract available.
PMID: *********** [PubMed - indexed for MEDLINE]
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8: What's new in cystic fibrosis? From treating symptoms to correction of the basic defect.
Proesmans M, Vermeulen F, De Boeck K.
Eur J Pediatr. 2008 Aug;167(8):839-49. Epub 2008 Apr 4. Review.
PMID: *********** [PubMed - indexed for MEDLINE]
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9: PTC124 is an orally bioavailable compound that promotes suppression of the human CFTR-G542X nonsense allele in a CF mouse model.
Du M, Liu X, Welch EM, Hirawat S, Peltz SW, Bedwell DM.
Proc Natl Acad Sci U S A. 2008 Feb 12;105(6):2064-9. Epub 2008 Feb 6.
PMID: *********** [PubMed - indexed for MEDLINE]
Related Articles Free article in PMC | at journal site
10: PTC124, nonsense mutations and Duchenne muscular dystrophy.
Wilton S.
Neuromuscul Disord. 2007 Oct;17(9-10):719-20. No abstract available.
PMID: *********** [PubMed - indexed for MEDLINE]
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11: Studies point way to new therapeutic prospects for muscular dystrophy.
Kuehn BM.
JAMA. 2007 Sep 26;298(12):1385-6. No abstract available.
PMID: *********** [PubMed - indexed for MEDLINE]
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12: Emerging drug treatments for cystic fibrosis.
Zeitlin PL.
Expert Opin Emerg Drugs. 2007 May;12(2):329-36. Review.
PMID: *********** [PubMed - indexed for MEDLINE]
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13: Chemical biology: ignore the nonsense.
Schmitz A, Famulok M.
Nature. 2007 May 3;447(7140):42-3. No abstract available.
PMID: *********** [PubMed - indexed for MEDLINE]
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14: PTC124 targets genetic disorders caused by nonsense mutations.
Welch EM, Barton ER, Zhuo J, Tomizawa Y, Friesen WJ, Trifillis P, Paushkin S, Patel M, Trotta CR, Hwang S, Wilde RG, Karp G, Takasugi J, Chen G, Jones S, Ren H, Moon YC, Corson D, Turpoff AA, Campbell JA, Conn MM, Khan A, Almstead NG, Hedrick J, Mollin A, Risher N, Weetall M, Yeh S, Branstrom AA, Colacino JM, Babiak J, Ju WD, Hirawat S, Northcutt VJ, Miller LL, Spatrick P, He F, Kawana M, Feng H, Jacobson A, Peltz SW, Sweeney HL.
Nature. 2007 May 3;447(7140):87-91. Epub 2007 Apr 22.
PMID: *********** [PubMed - indexed for MEDLINE]
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15: Safety, tolerability, and pharmacokinetics of PTC124, a nonaminoglycoside nonsense mutation suppressor, following single- and multiple-dose administration to healthy male and female adult volunteers.
Hirawat S, Welch EM, Elfring GL, Northcutt VJ, Paushkin S, Hwang S, Leonard EM, Almstead NG, Ju W, Peltz SW, Miller LL.
J Clin Pharmacol. 2007 Apr;47(4):430-44.
PMID: *********** [PubMed - indexed for MEDLINE]
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16: Drug evaluation: PTC-124--a potential treatment of cystic fibrosis and Duchenne muscular dystrophy.
Hamed SA.
IDrugs. 2006 Nov;9(11):783-9.
PMID: *********** [PubMed - indexed for MEDLINE]
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17: Readthrough strategies for stop codons in Duchenne muscular dystrophy.
Aurino S, Nigro V.
Acta Myol. 2006 Jun;25(1):5-12. Review.
PMID: *********** [PubMed - indexed for MEDLINE]
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18: Gateways to clinical trials.
Bayes M, Rabasseda X, Prous JR.
Methods Find Exp Clin Pharmacol. 2005 Jul-Aug;27(6):411-61.
PMID: *********** [PubMed - indexed for MEDLINE
