Gene editing in hemophilia: a 'CRISPR' choice?

摘要

In this issue of Blood, Wang et al report correction of the bleeding phenotype in newborn and adult factor IX (FIX) knockout mice through in vivo gene editing mediated by clustered regularly interspaced short palindromic repeats/CRISPR associated protein 9 (CRISPR/Cas9). 1 This technology involves a guide RNA thatmatches the target gene of interest and the endonuclease, Cas9, which introduces a double-stranded DNA-break enabling modifications to the genome.