Correcting hemophilia

A group of Korean researchers has demonstrated that gene editing with CRISPR can correct large chromosomal inversions that account for nearly half of all hemophilia A cases. By creating guide RNAs that induced no off-target editing, the team circumvented one of the main concerns about using CRISPR therapeutically.

The study involved creating induced pluripotent stem (iPS) cells from patients with either the less common 140 kbp inversion in the intron 1 homolog of the Factor VIII gene, which accounts for about 5% of cases, or the more common 600 kbp inversion in the intron 22 homolog, which accounts for about 40% of cases. ...