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Systemic AAV Micro-dystrophin Gene Therapy for Duchenne Muscular Dystrophy

17 Jul 2018

Abstract excerpt

Duchenne muscular dystrophy (DMD) is a lethal muscle disease caused by dystrophin gene mutation. Conceptually, replacing the mutated gene with a normal one would cure the disease. However, this task has encountered significant challenges due to the enormous size of the gene and the distribution of muscle throughout the body. The former creates a hurdle for viral vector packaging and the latter begs for whole-body...

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PMID: 30093306
DOI: 10.1016/j.ymthe.2018.07.011

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