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Ex Vivo COL7A1 Correction for Recessive Dystrophic Epidermolysis Bullosa Using CRISPR/Cas9 and Homology-Directed Repair

Recessive dystrophic epidermolysis bullosa is a rare and severe genetic skin disease resulting in blistering of the skin and mucosa. Recessive dystrophic epidermolysis bullosa (RDEB) is caused by a wide variety of mutations in COL7A1-encoding type VII collagen, which is essential for dermal-epiderma...

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Detalles Bibliográficos
Autores principales: Izmiryan, Araksya, Ganier, Clarisse, Bovolenta, Matteo, Schmitt, Alain, Mavilio, Fulvio, Hovnanian, Alain
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Gene & Cell Therapy 2018
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6077132/
https://www.ncbi.nlm.nih.gov/pubmed/30195791
http://dx.doi.org/10.1016/j.omtn.2018.06.008