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Article

The progress of AAV-mediated gene therapy in neuromuscular disorders

21 May 2018

Abstract excerpt

INTRODUCTION: The well-defined genetic causes and monogenetic nature of many neuromuscular disorders, including Duchenne muscular dystrophy (DMD) and spinal muscular atrophy (SMA), present gene therapy as a prominent therapeutic approach. The novel variants of adeno-associated virus (AAV) can achieve satisfactory transduction efficiency of exogenous genes through the central nervous system and body-wide in...

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PMID: 29781327
DOI: 10.1080/14712598.2018.1479739

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