Therapeutic potential of highly functional codon-optimized microutrophin for muscle-specific expression

High expectations have been set on gene therapy with an AAV-delivered shortened version of dystrophin (µDys) for Duchenne muscular dystrophy (DMD), with several drug candidates currently undergoing clinical trials. Safety concerns with this therapeutic approach include the immune response to introdu...

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Detalles Bibliográficos
Autores principales: Starikova, Anna V., Skopenkova, Victoria V., Polikarpova, Anna V., Reshetov, Denis A., Vassilieva, Svetlana G., Velyaev, Oleg A., Shmidt, Anna A., Savchenko, Irina M., Soldatov, Vladislav O., Egorova, Tatiana V., Bardina, Maryana V.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Nature Publishing Group UK 2022
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8764061/
https://www.ncbi.nlm.nih.gov/pubmed/35039573
http://dx.doi.org/10.1038/s41598-022-04892-x

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8764061/
https://www.ncbi.nlm.nih.gov/pubmed/35039573
http://dx.doi.org/10.1038/s41598-022-04892-x

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