RECOMBINANT VIRUS PRODUCTS AND METHODS FOR INHIBITING EXPRESSION OF DYSTROPHIA MYOTONICA PROTEIN KINASE AND/OR INTERFERING WITH A TRINUCLEOTIDE REPEAT EXPANSION IN THE 3' UNTRANSLATED REGION OF THE DMPK GENE

摘要

The present disclosure relates to RNA interference-based methods for inhibiting the expression of the dystrophia myotonia protein kinase (DMPK) gene. Recombinant adeno-associated viruses of the disclosure deliver DNAs encoding inhibitory RNAs that knock down the expression of DMPK or interfere with the expression of the CTG repeat associated with myotonic dystrophy type-1 (DM1). The methods have application in the treatment of myotonic dystrophies, including DM1, and other disorders associated with aberrant DMPK expression.