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.
MeSH terms
-
CRISPR-Cas Systems*
-
Gene Editing*
-
Humans
-
RNA Editing