CRISPR/Cas9-based genetic correction for recessive dystrophic epidermolysis bullosa

Recessive dystrophic epidermolysis bullosa (RDEB) is a severe disorder caused by mutations to the COL7A1 gene that deactivate production of a structural protein essential for skin integrity. Haematopoietic cell transplantation can ameliorate some of the symptoms; however, significant side effects fr...

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Détails bibliographiques
Publié dans:NPJ Regen Med
Auteurs principaux: Webber, Beau R, Osborn, Mark J, McElroy, Amber N, Twaroski, Kirk, Lonetree, Cara-lin, DeFeo, Anthony P, Xia, Lily, Eide, Cindy, Lees, Christopher J, McElmurry, Ron T, Riddle, Megan J, Kim, Chong Jai, Patel, Dharmeshkumar D, Blazar, Bruce R, Tolar, Jakub
Format: Artigo
Langue:Inglês
Publié: Nature Publishing Group 2016
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Article
Accès en ligne:https://ncbi.nlm.nih.gov/pmc/articles/PMC5328670/
https://ncbi.nlm.nih.gov/pubmed/28250968
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/npjregenmed.2016.14
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