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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Vydáno v:J Invest Dermatol
Hlavní autoři: 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
Médium: Artigo
Jazyk:Inglês
Vydáno: 2019
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Article
On-line přístup: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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