In vivo topical gene therapy for recessive dystrophic epidermolysis bullosa: a phase 1 and 2 trial

Recessive dystrophic epidermolysis bullosa (RDEB) is a lifelong genodermatosis associated with blistering, wounding, and scarring caused by mutations in COL7A1, the gene encoding the anchoring fibril component, collagen VII (C7). Here, we evaluated beremagene geperpavec (B-VEC), an engineered, non-r...

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Detalles Bibliográficos
Autores principales: Gurevich, Irina, Agarwal, Pooja, Zhang, PeiPei, Dolorito, John A., Oliver, Stacie, Liu, Henry, Reitze, Nicholas, Sarma, Nikhil, Bagci, Isin Sinem, Sridhar, Kunju, Kakarla, Visesha, Yenamandra, Vamsi K., O’Malley, Mark, Prisco, Marco, Tufa, Sara F., Keene, Douglas R., South, Andrew P., Krishnan, Suma M., Marinkovich, M. Peter
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Nature Publishing Group US 2022
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9018416/
https://www.ncbi.nlm.nih.gov/pubmed/35347281
http://dx.doi.org/10.1038/s41591-022-01737-y

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