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Namespace Prefixes

PrefixIRI
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Statements

Subject Item
n2:DB05016
rdf:type
n3:Drug
n3:description
PTC124 is a novel, orally administered drug that targets nonsense mutations and is being investigated initially as a treatment for Duchenne muscular dystrophy (DMD) and cystic fibrosis (CF), with the potential to treat a number of other genetic disorders caused by nonsense mutations.
n3:generalReferences
# 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: PTC124 targets genetic disorders caused by nonsense mutations. Nature. 2007 May 3;447(7140):87-91. Epub 2007 Apr 22. "Pubmed":http://www.ncbi.nlm.nih.gov/pubmed/17450125 # Hirawat S, Welch EM, Elfring GL, Northcutt VJ, Paushkin S, Hwang S, Leonard EM, Almstead NG, Ju W, Peltz SW, Miller LL: Safety, tolerability, and pharmacokinetics of PTC124, a nonaminoglycoside nonsense mutation suppressor, following single- and multiple-dose administration to healthy male and female adult volunteers. J Clin Pharmacol. 2007 Apr;47(4):430-44. "Pubmed":http://www.ncbi.nlm.nih.gov/pubmed/17389552 # Hamed SA: Drug evaluation: PTC-124--a potential treatment of cystic fibrosis and Duchenne muscular dystrophy. IDrugs. 2006 Nov;9(11):783-9. "Pubmed":http://www.ncbi.nlm.nih.gov/pubmed/17096300
n3:group
investigational
n3:halfLife
3-6 hours
n3:indication
Investigated for use/treatment in cystic fibrosis and muscular dystrophy.
owl:sameAs
n7:DB05016
dcterms:title
PTC124
adms:identifier
n9:DB05016 n10:11219835 n11:9394889 n12:29203630
n3:mechanismOfAction
PTC124 allowed the cellular machinery to bypass the nonsense mutation, continue the translation process, and thereby restore the production of a full-length, functional protein. The research on the effects of PTC124 on the translation and stability of nonsense-containing mRNA in vitor show that PTC124 promoted readthrough at each of the nonsense codons, showing maximal activity with UGA, while having no effect on mRNA levels. Unlike the stable cell line assays, PTC124 did not discriminate significantly between the UAG and UAA mRNAs. PTC124 was a more potent nonsense-suppressing agent than gentamicin, and exhibited 4- to 15-fold stimulation of in vitro readthrough relative to the controls at levels similar to those in the stable cell reporter assays. These results indicate that PTC124 modulates termination efficiency at premature nonsense codons.
n3:synonym
EC-000.2051
n3:IUPAC-Name
n4:271B6277-363D-11E5-9242-09173F13E4C5
n3:InChI
n4:271B627D-363D-11E5-9242-09173F13E4C5
n3:Molecular-Formula
n4:271B627C-363D-11E5-9242-09173F13E4C5
n3:Molecular-Weight
n4:271B6279-363D-11E5-9242-09173F13E4C5
n3:Monoisotopic-Weight
n4:271B627A-363D-11E5-9242-09173F13E4C5
n3:SMILES
n4:271B627B-363D-11E5-9242-09173F13E4C5
n3:Water-Solubility
n4:271B6275-363D-11E5-9242-09173F13E4C5
n3:logP
n4:271B6273-363D-11E5-9242-09173F13E4C5 n4:271B6276-363D-11E5-9242-09173F13E4C5
n3:logS
n4:271B6274-363D-11E5-9242-09173F13E4C5
n3:H-Bond-Acceptor-Count
n4:271B6283-363D-11E5-9242-09173F13E4C5
n3:H-Bond-Donor-Count
n4:271B6284-363D-11E5-9242-09173F13E4C5
n3:InChIKey
n4:271B627E-363D-11E5-9242-09173F13E4C5
n3:Polar-Surface-Area--PSA-
n4:271B627F-363D-11E5-9242-09173F13E4C5
n3:Polarizability
n4:271B6281-363D-11E5-9242-09173F13E4C5
n3:Refractivity
n4:271B6280-363D-11E5-9242-09173F13E4C5
n3:Rotatable-Bond-Count
n4:271B6282-363D-11E5-9242-09173F13E4C5
n3:Bioavailability
n4:271B6289-363D-11E5-9242-09173F13E4C5
n3:Ghose-Filter
n4:271B628B-363D-11E5-9242-09173F13E4C5
n3:MDDR-Like-Rule
n4:271B628C-363D-11E5-9242-09173F13E4C5
n3:Number-of-Rings
n4:271B6288-363D-11E5-9242-09173F13E4C5
n3:Physiological-Charge
n4:271B6287-363D-11E5-9242-09173F13E4C5
n3:Rule-of-Five
n4:271B628A-363D-11E5-9242-09173F13E4C5
n3:Traditional-IUPAC-Name
n4:271B6278-363D-11E5-9242-09173F13E4C5
n3:pKa--strongest-acidic-
n4:271B6285-363D-11E5-9242-09173F13E4C5
n3:pKa--strongest-basic-
n4:271B6286-363D-11E5-9242-09173F13E4C5