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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Detaylı Bibliyografya
Yayımlandı:JCI Insight
Asıl Yazarlar: 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.
Materyal Türü: Artigo
Dil:Inglês
Baskı/Yayın Bilgisi: American Society for Clinical Investigation 2019
Konular:
Clinical Medicine
Online Erişim: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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