PEPTIDE-LINKED MORPHOLINO ANTISENSE OLIGONUCLEOTIDES FOR TREATMENT OF MYOTONIC DYSTROPHY

摘要

Peptide-binding morpholino (PPMO) antisense oligonucleotides targeting the poly CUG repeat region within the 3'untranslated region of the gene encoding dystrophy-protein kinase (DMPK) and type I myotonic dystrophy (DM1) A method of systemic administration of the above for the treatment of) is provided herein.