Precise therapeutic gene correction by a simple nuclease-induced double-strand break

Current programmable nuclease-based (e.g. CRISPR-Cas9) methods for precise correction of a disease-causing genetic mutation harness the Homology Directed Repair (HDR) pathway. However, this repair process requires co-delivery of an exogenous DNA donor to recode the sequence and can be inefficient in...

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Udgivet i:Nature
Main Authors: Iyer, Sukanya, Suresh, Sneha, Guo, Dongsheng, Daman, Katelyn, Chen, Jennifer C. J., Liu, Pengpeng, Zieger, Marina, Luk, Kevin, Roscoe, Benjamin P., Mueller, Christian, King, Oliver D., Emerson, Charles P., Wolfe, Scot A.
Format: Artigo
Sprog:Inglês
Udgivet: 2019
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Article
Online adgang:https://ncbi.nlm.nih.gov/pmc/articles/PMC6483862/
https://ncbi.nlm.nih.gov/pubmed/30944467
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/s41586-019-1076-8
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