Micro-laminin gene therapy can function as an inhibitor of muscle disease in the dyW mouse model of MDC1A

Micro-laminin gene therapy can function as an inhibitor of muscle disease in the dyW mouse model of MDC1A

Gene replacement for laminin-α2-deficient congenital muscular dystrophy 1A (MDC1A) is currently not possible using a single adeno-associated virus (AAV) vector due to the large size of the LAMA2 gene. LAMA2 encodes laminin-α2, a subunit of the trimeric laminin-211 extracellular matrix (ECM) protein...

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Bibliographic Details
Main Authors: Davin Packer, Paul T. Martin
Format: Article
Language:English
Published: Elsevier 2021-06-01
Series:Molecular Therapy: Methods & Clinical Development
Subjects:
gene therapy
muscular dystrophy
laminin
AAV
extracellular matrix
Online Access:http://www.sciencedirect.com/science/article/pii/S232905012100019X

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