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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Gepubliceerd in:J Invest Dermatol
Hoofdauteurs: 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
Formaat: Artigo
Taal:Inglês
Gepubliceerd in: 2019
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Article
Online toegang: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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