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Gene editing toward the use of autologous therapies in recessive dystrophic epidermolysis bullosa

Recessive dystrophic epidermolysis bullosa (RDEB) is a disease caused by mutations in the COL7A1 gene that result in absent or dysfunctional type VII collagen protein production. Clinically, RDEB manifests as early and severe chronic cutaneous blistering, damage to internal epithelium, an elevated r...

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Detalhes bibliográficos
Publicado no:Transl Res
Main Authors: Perdoni, Christopher, Osborn, Mark J., Tolar, Jakub
Formato: Artigo
Idioma:Inglês
Publicado em: 2015
Assuntos:
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC4662628/
https://ncbi.nlm.nih.gov/pubmed/26073463
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.trsl.2015.05.008
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