Safety and early efficacy outcomes for lentiviral fibroblast gene therapy in recessive dystrophic epidermolysis bullosa

BACKGROUND: Recessive dystrophic epidermolysis bullosa (RDEB) is a severe form of skin fragility disorder due to mutations in COL7A1 encoding basement membrane type VII collagen (C7), the main constituent of anchoring fibrils (AFs) in skin. We developed a self-inactivating lentiviral platform encodi...

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發表在:JCI Insight
Main Authors: Lwin, Su M., Syed, Farhatullah, Di, Wei-Li, Kadiyirire, Tendai, Liu, Lu, Guy, Alyson, Petrova, Anastasia, Abdul-Wahab, Alya, Reid, Fiona, Phillips, Rachel, Elstad, Maria, Georgiadis, Christos, Aristodemou, Sophia, Lovell, Patricia A., McMillan, James R., Mee, John, Miskinyte, Snaigune, Titeux, Matthias, Ozoemena, Linda, Pramanik, Rashida, Serrano, Sonia, Rowles, Racheal, Maurin, Clarisse, Orrin, Elizabeth, Martinez-Queipo, Magdalena, Rashidghamat, Ellie, Tziotzios, Christos, Onoufriadis, Alexandros, Chen, Mei, Chan, Lucas, Farzaneh, Farzin, Del Rio, Marcela, Tolar, Jakub, Bauer, Johann W., Larcher, Fernando, Antoniou, Michael N., Hovnanian, Alain, Thrasher, Adrian J., Mellerio, Jemima E., Qasim, Waseem, McGrath, John A.
格式: Artigo
語言:Inglês
出版: American Society for Clinical Investigation 2019
主題:
Clinical Medicine
在線閱讀:https://ncbi.nlm.nih.gov/pmc/articles/PMC6629162/
https://ncbi.nlm.nih.gov/pubmed/31167965
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1172/jci.insight.126243
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