Adeno-Associated Virus (AAV)-Mediated Gene Therapy for Duchenne Muscular Dystrophy: The Issue of Transgene Persistence

Adeno-Associated Virus (AAV)-Mediated Gene Therapy for Duchenne Muscular Dystrophy: The Issue of Transgene Persistence

Duchenne muscular dystrophy (DMD) is an X-linked recessive, infancy-onset neuromuscular disorder characterized by progressive muscle weakness and atrophy, leading to delay of motor milestones, loss of autonomous ambulation, respiratory failure, cardiomyopathy, and premature death. DMD originates fro...

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Bibliographic Details
Main Authors: Arianna Manini, Elena Abati, Andi Nuredini, Stefania Corti, Giacomo Pietro Comi
Format: Article
Language:English
Published: Frontiers Media S.A. 2022-01-01
Series:Frontiers in Neurology
Subjects:
Duchenne muscular dystrophy
adeno-associated virus
gene therapy
persistence
dystrophin
microdystrophin
Online Access:https://www.frontiersin.org/articles/10.3389/fneur.2021.814174/full

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https://www.frontiersin.org/articles/10.3389/fneur.2021.814174/full

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