Optimized lentiviral vector to restore full-length dystrophin via a cell-mediated approach in a mouse model of Duchenne muscular dystrophy

Optimized lentiviral vector to restore full-length dystrophin via a cell-mediated approach in a mouse model of Duchenne muscular dystrophy

Duchenne muscular dystrophy (DMD) is a muscle wasting disorder caused by mutations in the DMD gene. Restoration of full-length dystrophin protein in skeletal muscle would have therapeutic benefit, but lentivirally mediated delivery of such a large gene in vivo has been hindered by lack of tissue spe...

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Bibliographic Details
Main Authors: Jinhong Meng, Marc Moore, John Counsell, Francesco Muntoni, Linda Popplewell, Jennifer Morgan
Format: Article
Language:English
Published: Elsevier 2022-06-01
Series:Molecular Therapy: Methods & Clinical Development
Subjects:
lentivirus
Duchenne muscular dystrophy
native full-length dystrophin
sequence-optimized full-length dystrophin
myoblasts
muscle-specific promoter
Online Access:http://www.sciencedirect.com/science/article/pii/S2329050122000638

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http://www.sciencedirect.com/science/article/pii/S2329050122000638

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