Recombinant virus products and methods for inhibiting expression of myotonic dystrophy protein kinase and / or interfering with trunucleotide repeat elongation in the 3 'untranslated region of DMPK gene

摘要

This disclosure relates to an RNA interference based method for suppressing the expression of a myotonic dystrophy protein kinase (DMPK) gene.The recombinant adeno associated virus of the present disclosure delivers the expression of DMPK DNA that encodes inhibitory RNA that interferes with the expression of CTG repeat associated with knockdown or type I myotonic dystrophy (DM1).This method has an application in the treatment of myotonic dystrophy, including DM1 and other disorders associated with abnormal DMPK expression.