CRISPR-Cas9 correction in the DMD mouse model is accompanied by upregulation of Dp71f protein

Duchenne muscular dystrophy (DMD) is a severe hereditary disease caused by a deficiency in the dystrophin protein. The most frequent types of disease-causing mutations in the DMD gene are frameshift deletions of one or more exons. Precision genome editing systems such as CRISPR-Cas9 have shown poten...

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Detalles Bibliográficos
Autores principales: Egorova, Tatiana V., Polikarpova, Anna V., Vassilieva, Svetlana G., Dzhenkova, Marina A., Savchenko, Irina M., Velyaev, Oleg A., Shmidt, Anna A., Soldatov, Vladislav O., Pokrovskii, Mikhail V., Deykin, Alexey V., Bardina, Maryana V.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Gene & Cell Therapy 2023
Materias:
Original Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10339130/
https://www.ncbi.nlm.nih.gov/pubmed/37457303
http://dx.doi.org/10.1016/j.omtm.2023.06.006

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