Knock-in editing: it functionally corrects! #MMPMID27231391
Porteus MH
Blood 2016[May]; 127 (21): 2507-9 PMID27231391show ga
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.1