RNAi-based Gene Therapy for Dominant Limb Girdle Muscular Dystrophies
Scott Q. Harper.
Limb Girdle Muscular Dystrophy (LGMD) refers to a group of 25 genetic diseases linked by common clinical
features, including wasting of muscles supporting the pelvic and shoulder girdles. Cardiac involvement may also occur.
Like other muscular dystrophies, LGMDs are currently incurable, but prospective gene replacement therapies targeting recessive
forms have shown promise in pre-clinical and clinical studies. In contrast, little attention has been paid to developing
gene therapy approaches for dominant forms of LGMD, which would likely benefit from disease gene silencing. Despite
the lack of focus to date on developing gene therapies for dominant LGMDs, the field is not starting at square one,
since translational studies on recessive LGMDs provided a framework that can be applied to treating dominant forms of
the disease. In this manuscript, we discuss the prospects of treating dominantly inherited forms of LGMD with gene silencing
Keywords: AAV, dominant myopathy, gene therapy, LGMD, limb-girdle muscular dystrophy, miRNA, RNAi, RNA interference
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