RNA Editing for Muscular Dystrophy Therapy

Abstract

Due to lack of effective therapies, muscular dystrophies became a focus for gene therapy. Multiple pre-clinical studies have shown successful restoration of dystrofin and dysferlin by RNA editing both in vivo and in vitro, but possibility of a clinical translation is still obscure. A number of new chemicals are being studied, and a search for new techniques is ongoing. This work is intended to give a brief overview of the current state of the RNA editing for treating muscular dystrophies.