Base editor correction of COL7A1 in recessive dystrophic epidermolysis bullosa patient-derived fibroblasts and iPSCs

Genome editing represents a promising strategy for therapeutic correction of COL7A1 mutations that cause recessive dystrophic epidermolysis bullosa (RDEB). DNA cleavage followed by homology-directed repair (HDR) using an exogenous template has previously been used to correct COL7A1 mutations. HDR ra...

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Publicat a:J Invest Dermatol
Autors principals: Osborn, Mark J., Newby, Gregory A., McElroy, Amber N., Knipping, Friederike, Nielsen, Sarah C., Riddle, Megan J., Xia, Lily, Chen, Weili, Eide, Cindy R., Webber, Beau R., Wandall, Hans H., Dabelsteen, Sally, Blazar, Bruce R., Liu, David R., Tolar, Jakub
Format: Artigo
Idioma:Inglês
Publicat: 2019
Matèries:
Article
Accés en línia:https://ncbi.nlm.nih.gov/pmc/articles/PMC6983342/
https://ncbi.nlm.nih.gov/pubmed/31437443
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.jid.2019.07.701
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