Therapeutic base editing and prime editing of COL7A1 mutations in recessive dystrophic epidermolysis bullosa

Recessive dystrophic epidermolysis bullosa (RDEB) is a severe skin fragility disorder caused by loss-of-function mutations in the COL7A1 gene, which encodes type VII collagen (C7), a protein that functions in skin adherence. From 36 Korean RDEB patients, we identified a total of 69 pathogenic mutati...

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Detalles Bibliográficos
Autores principales: Hong, Sung-Ah, Kim, Song-Ee, Lee, A-Young, Hwang, Gue-Ho, Kim, Jong Hoon, Iwata, Hiroaki, Kim, Soo-Chan, Bae, Sangsu, Lee, Sang Eun
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Gene & Cell Therapy 2022
Materias:
Original Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9372317/
https://www.ncbi.nlm.nih.gov/pubmed/35690907
http://dx.doi.org/10.1016/j.ymthe.2022.06.005

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