Phase 1/2a clinical trial of gene-corrected autologous cell therapy for recessive dystrophic epidermolysis bullosa

BACKGROUND: Recessive dystrophic epidermolysis bullosa (RDEB) patients have mutations in the COL7A1 gene and thus lack functional type VII collagen (C7) protein; they have marked skin fragility and blistering. This single-center phase 1/2a open-label study evaluated the long-term efficacy, safety, a...

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Detalhes bibliográficos
Publicado no:JCI Insight
Main Authors: Eichstadt, Shaundra, Barriga, Melissa, Ponakala, Anusha, Teng, Claudia, Nguyen, Ngon T., Siprashvili, Zurab, Nazaroff, Jaron, Gorell, Emily S., Chiou, Albert S., Taylor, Lisa, Khuu, Phuong, Keene, Douglas R., Rieger, Kerri, Khosla, Rohit K., Furukawa, Louise K., Lorenz, H. Peter, Marinkovich, M. Peter, Tang, Jean Y.
Formato: Artigo
Idioma:Inglês
Publicado em: American Society for Clinical Investigation 2019
Assuntos:
Clinical Medicine
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC6795403/
https://ncbi.nlm.nih.gov/pubmed/31578311
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1172/jci.insight.130554
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