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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Detalles Bibliográficos
Autores principales: Yakovlev, Ivan A., Emelin, Aleksei M., Slesarenko, Yana S., Limaev, Igor S., Vetrova, Iuliia A., Belikova, Liliya D., Grafskaia, Ekaterina N., Bobrovsky, Pavel A., Pokrovsky, Mikhail V., Kuzubova, Elena V., Pokrovsky, Vladimir M., Lebedev, Pyotr A., Bardakov, Sergei N., Isaev, Artur A., Deev, Roman V.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: MDPI 2023
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10488094/
https://www.ncbi.nlm.nih.gov/pubmed/37686363
http://dx.doi.org/10.3390/ijms241713551

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