Redesign of trans-splicing molecules for the correction of dystrophia myotonica type 1 toxic RNA transcripts

  • Eleanor G. Harrison
  • Published 2014

Abstract

Dystrophia myotonica (DM1), one of the most common forms of muscular dystrophy, is caused by a repeated trinucleotide expansion in the DMPK gene. This mutation results in the accumulation of toxic cellular RNA transcripts. Spliceosome-mediated RNA trans-splicing (SMaRT) technology is a form of gene therapy that possesses the potential to correct these toxic… (More)

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