Efficacy and muscle safety assessment of fukutin-related protein gene therapy

Efficacy and muscle safety assessment of fukutin-related protein gene therapy

Limb-girdle muscular dystrophy type R9 (LGMDR9) is a muscle-wasting disease that begins in the hip and shoulder regions of the body. This disease is caused by mutations in fukutin-related protein (FKRP), a glycosyltransferase critical for maintaining muscle cell integrity. Here we investigated poten...

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Bibliographic Details
Main Authors: Halli Benasutti, Joseph W. Maricelli, Jane Seto, John Hall, Christine Halbert, Jacqueline Wicki, Lydia Heusgen, Nicholas Purvis, Michael Regnier, David C. Lin, Buel D. Rodgers, Jeffrey S. Chamberlain
Format: Article
Language:English
Published: Elsevier 2023-09-01
Series:Molecular Therapy: Methods & Clinical Development
Subjects:
gene therapy
fukutin-related protein
FKRP
limb-girdle muscular dystrophy type R9 (LGMDR9)
adeno-associated viral vector (AAV)
physiology
Online Access:http://www.sciencedirect.com/science/article/pii/S2329050123000840

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http://www.sciencedirect.com/science/article/pii/S2329050123000840

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