Abstract
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 approaches.
Keywords: AAV, dominant myopathy, gene therapy, LGMD, limb-girdle muscular dystrophy, miRNA, RNAi, RNA interference
Current Gene Therapy
Title:RNAi-based Gene Therapy for Dominant Limb Girdle Muscular Dystrophies
Volume: 12 Issue: 4
Author(s): Jian Liu and Scott Q. Harper
Affiliation:
Keywords: AAV, dominant myopathy, gene therapy, LGMD, limb-girdle muscular dystrophy, miRNA, RNAi, RNA interference
Abstract: 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 approaches.
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Cite this article as:
Liu Jian and Harper Q. Scott, RNAi-based Gene Therapy for Dominant Limb Girdle Muscular Dystrophies, Current Gene Therapy 2012; 12 (4) . https://dx.doi.org/10.2174/156652312802083585
DOI https://dx.doi.org/10.2174/156652312802083585 |
Print ISSN 1566-5232 |
Publisher Name Bentham Science Publisher |
Online ISSN 1875-5631 |
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