CRISPR-Cas9-based non-viral gene editing therapy for topical treatment of recessive dystrophic epidermolysis bullosa

Recessive dystrophic epidermolysis bullosa (RDEB) is an autosomal monogenic skin disease caused by mutations in COL7A1 gene and lack of functional type VII collagen (C7). Currently, there is no cure for RDEB, and most of the gene therapies under development have been designed as ex vivo strategies b...

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Detalles Bibliográficos
Autores principales: Wang, Xianqing, Wang, Xi, Li, Yinghao, A, Sigen, Qiu, Bei, Bushmalyova, Albina, He, Zhonglei, Wang, Wenxin, Lara-Sáez, Irene
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/PMC10630779/
https://www.ncbi.nlm.nih.gov/pubmed/38027067
http://dx.doi.org/10.1016/j.omtm.2023.101134

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