Knock-in editing: it functionally corrects!
Blood, 127(21): 2507-9 PMID 27231391 DOI 10.1182/blood-2016-03-703181
Abstract
In this issue of Blood, Hubbard et al provide important proof-of-concept data on the utility of using genome editing to knock-in a wild-type (WT) complementary DNA (cDNA) to functionally correct disease-causing mutations in a gene in primary human T cells.