Article
Correction of a pathogenic gene mutation in human embryos.
Nature - 24 Aug 2017
Ma Hong, Marti-Gutierrez Nuria, Park Sang-Wook, Wu Jun, Lee Yeonmi, Suzuki Keiichiro, Koski Amy, Ji Dongmei, Hayama Tomonari, Ahmed Riffat, Darby Hayley, Van Dyken Crystal, Li Ying, Kang Eunju, Park A-Reum, Kim Daesik, Kim Sang-Tae, Gong Jianhui, Gu Ying, Xu Xun, Battaglia David, Krieg Sacha A, Lee David M, Wu Diana H, Wolf Don P, Heitner Stephen B, Belmonte Juan Carlos Izpisua, Amato Paula, Kim Jin-Soo, Kaul Sanjiv, Mitalipov Shoukhrat
Abstract excerpt
Genome editing has potential for the targeted correction of germline mutations. Here we describe the correction of the heterozygous MYBPC3 mutation in human preimplantation embryos with precise CRISPR-Cas9-based targeting accuracy and high homology-directed repair efficiency by activating an endogenous, germline-specific DNA repair response. Induced double-strand breaks (DSBs) at the mutant paternal allele were...
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