Dual Adeno-Associated Virus 9 with Codon-Optimized DYSF Gene Promotes In Vivo Muscle Regeneration and May Decrease Inflammatory Response in Limb Girdle Muscular Dystrophy Type R2

Dual Adeno-Associated Virus 9 with Codon-Optimized DYSF Gene Promotes In Vivo Muscle Regeneration and May Decrease Inflammatory Response in Limb Girdle Muscular Dystrophy Type R2

Dysferlinopathy treatment is an active area of investigation. Gene therapy is one potential approach. We studied muscle regeneration and inflammatory response after injection of an AAV-9 with a codon-optimized DYSF gene. A dual-vector system AAV.DYSF.OVERLAP with overlapping DYSF cDNA sequences was...

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Bibliographic Details
Main Authors: Ivan A. Yakovlev, Aleksei M. Emelin, Yana S. Slesarenko, Igor S. Limaev, Iuliia A. Vetrova, Liliya D. Belikova, Ekaterina N. Grafskaia, Pavel A. Bobrovsky, Mikhail V. Pokrovsky, Elena V. Kuzubova, Vladimir M. Pokrovsky, Pyotr A. Lebedev, Sergei N. Bardakov, Artur A. Isaev, Roman V. Deev
Format: Article
Language:English
Published: MDPI AG 2023-08-01
Series:International Journal of Molecular Sciences
Subjects:
LGMD R2
dysferlin
gene transfer
dual AAV vector
immune response
muscle regeneration
Online Access:https://www.mdpi.com/1422-0067/24/17/13551

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https://www.mdpi.com/1422-0067/24/17/13551

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