Correction of Dystrophia Myotonica Type 1 Toxic RNA Transcripts Via Trans-Splicing
Friday, November 7, 2014
Plaza Foyer (Sheraton)
Dystrophia myotonica (DM1) a common form of muscular dystrophy is caused by a mutation within the DMPK gene. In this study a type of gene therapy, SMaRT technology, was studied and examined as a potential therapy for DM1.