[EN] PHARMACEUTICAL COMPOSITIONS AND TREATMENT OF GENETIC DISEASES ASSOCIATED WITH NONSENSE MEDIATED RNA DECAY<br/>[FR] COMPOSITIONS PHARMACEUTIQUE ET TRAITEMENT DE MALADIES GÉNÉTIQUES ASSOCIÉES À UNE DÉSINTÉGRATION D'ARN À MÉDIATION NON-SENS
申请人:UNIV NEW YORK
公开号:WO2014055644A2
公开(公告)日:2014-04-10
The present invention relates to pharmaceutical compositions comprising a compound and a pharmaceutically acceptable carrier. The present invention is also directed to a method of treating a genetic disease caused by premature termination codons, or other conditions that render messenger ribonucleic acid (mRNA) susceptible to nonsense mediated RNA decay, in a subject. Also disclosed is a method of inhibiting nonsense mediated RNA decay and/or induction of autophagy. The present invention also relates to a method of identifying inhibitors of nonsense mediated RNA decay and/or inducing autophagy. The present invention further relates to a method of inhibiting nonsense mediated RNA decay and/or induction of autophagy in a subject.