Pharmaceutical therapies to recode nonsense mutations in inherited diseases

Pharmacology and Therapeutics

Volumn 136, Issue 2, 2012, Pages 227-266

  Lee, Hui Ling Rose ^a   Dougherty, Joseph P ^a  

^a RUTGERS ROBERT WOOD JOHNSON MEDICAL SCHOOL   (United States)

Author keywords

Aminoglycoside; Inducible expression system; Non aminoglycoside; Nonsense codon; Nonsense suppression therapy; Premature termination codon; Readthrough

Indexed keywords

AMIKACIN; AMINO ACID; AMINOGLYCOSIDE DERIVATIVE; ANTIBIOTIC G 418; ATALUREN; CLITOCINE; GENTAMICIN; HYGROMYCIN; HYGROMYCIN B; ISEPAMICIN; JL 016; JL 022; JL 023; JL 024; JL 026; JL 027; JOSAMYCIN; KANAMYCIN A; LIVIDOMYCIN; MESSENGER RNA; NB 30; NB 54; NB 74; NB 84; NEGAMYCIN; NEOMYCIN; NETILMICIN; PAROMOMYCIN; PUROMYCIN; RTC 13; RTC 14; SPIRAMYCIN; STREPTOMYCIN; TC 001; TC 003; TC 009; TC 012; TC 015; TC 016; TC 021; TC 023; TC 028; TC 029; TC 032; TC 033; TC 037; TOBRAMYCIN; TYLOSIN; UNCLASSIFIED DRUG; UNINDEXED DRUG;

ADENOMATOUS POLYP; ATAXIA TELANGIECTASIA; BETA THALASSEMIA; BLOOD CLOTTING FACTOR 7 DEFICIENCY; BRUGADA SYNDROME; CHONDROIDEREMIA; CHOROID CANCER; COLOBOMA; COLORECTAL CANCER; CYSTIC FIBROSIS; DIARRHEA; DIZZINESS; DRUG DOSE COMPARISON; DRUG MECHANISM; DRUG MEGADOSE; DUCHENNE MUSCULAR DYSTROPHY; FAMILIAL CANCER; FRAMESHIFT MUTATION; GENE EXPRESSION REGULATION; GENE THERAPY; GENETIC DISORDER; HEADACHE; HEART ATRIUM FIBRILLATION; HEMOPHILIA; HEMOPHILIA B; HUMAN; KIDNEY POLYCYSTIC DISEASE; KIDNEY TUBULE ACIDOSIS; LONG QT SYNDROME; LOW DRUG DOSE; METHYLMALONIC ACIDURIA; MISSENSE MUTATION; MUCOPOLYSACCHARIDOSIS; MUSCULAR DYSTROPHY; MUTANT; NAUSEA; NEOPLASM; NEPHROGENIC DIABETES INSIPIDUS; NONHUMAN; NONSENSE MUTATION; OBESITY; PRIORITY JOURNAL; PROTEIN SYNTHESIS; RANDOMIZED CONTROLLED TRIAL (TOPIC); RETINITIS PIGMENTOSA; RETT SYNDROME; REVIEW; SPINAL MUSCULAR ATROPHY; STOP CODON; USHER SYNDROME; VOMITING;

AMINOGLYCOSIDES; ANIMALS; CODON, NONSENSE; CYSTIC FIBROSIS; GENE EXPRESSION REGULATION; GENETIC DISEASES, INBORN; HEMOPHILIA A; HUMANS; MUSCULAR DYSTROPHY, DUCHENNE; RETT SYNDROME; RNA, MESSENGER;

EID: 84867328079     PISSN: 01637258     EISSN: 1879016X     Source Type: Journal    
DOI: 10.1016/j.pharmthera.2012.07.007     Document Type: Review

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160. PTC Therapeutics Inc. PTC Therapeutics initiates phase 2 study of PTC124 in cystic fibrosis 2005, Dec 6 PTC Therapeutics Inc. PRESS RELEASE
161. PTC Therapeutics Inc. PTC Therapeutics announces positive phase 2 results for PTC124 in cystic fibrosis 2006, Nov 3 PTC Therapeutics Inc. PRESS RELEASE
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164. PTC Therapeutics Inc. Observational data from one-year follow-up study validate six-minute walk distance as an outcome measure in Duchenne and Becker muscular dystrophy 2010, Dec 1 PTC Therapeutics Inc. PRESS RELEASE
165. PTC Therapeutics Inc. PTC Therapeutics completes enrollment of phase 3 trial of ataluren in patients with cystic fibrosis 2010, Dec 21 PTC Therapeutics Inc PRESS RELEASE
166. PTC Therapeutics Inc. PTC Therapeutics and Genzyme Corporation announce preliminary results from the phase 2B clinical trial of ataluren for nonsense mutation Duchenne/Becker muscular dystrophy 2010, Mar 3 PTC Therapeutic Inc. PRESS RELEASE
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