Preclinical model for phenotypic correction of dystrophic epidermolysis bullosa by in vivo CRISPR-Cas9 delivery using adenoviral vectors

Recessive dystrophic epidermolysis bullosa, a devastating skin fragility disease characterized by recurrent skin blistering, scarring, and a high risk of developing squamous cell carcinoma is caused by mutations in COL7A1, the gene encoding type VII collagen, which is the major component of the anch...

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Detalles Bibliográficos
Autores principales: García, Marta, Bonafont, Jose, Martínez-Palacios, Jesús, Xu, Rudan, Turchiano, Giandomenico, Svensson, Stina, Thrasher, Adrian J., Larcher, Fernando, Del Rio, Marcela, Hernández-Alcoceba, Rubén, Garín, Marina I., Mencía, Ángeles, Murillas, Rodolfo
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/PMC9531050/
https://www.ncbi.nlm.nih.gov/pubmed/36212909
http://dx.doi.org/10.1016/j.omtm.2022.09.005

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