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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Dades bibliogràfiques
Publicat a:JCI Insight
Autors principals: 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.
Format: Artigo
Idioma:Inglês
Publicat: American Society for Clinical Investigation 2019
Matèries:
Clinical Medicine
Accés en línia: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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