Full-length dystrophin restoration via targeted exon integration by AAV-CRISPR in a humanized mouse model of Duchenne muscular dystrophy

Targeted gene-editing strategies have emerged as promising therapeutic approaches for the permanent treatment of inherited genetic diseases. However, precise gene correction and insertion approaches using homology-directed repair are still limited by low efficiencies. Consequently, many gene-editing...

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Detalles Bibliográficos
Autores principales: Pickar-Oliver, Adrian, Gough, Veronica, Bohning, Joel D., Liu, Siyan, Robinson-Hamm, Jacqueline N., Daniels, Heather, Majoros, William H., Devlin, Garth, Asokan, Aravind, Gersbach, Charles A.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Gene & Cell Therapy 2021
Materias:
Original Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8571168/
https://www.ncbi.nlm.nih.gov/pubmed/34509668
http://dx.doi.org/10.1016/j.ymthe.2021.09.003

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