Treating genetic blood disorders in the era of CRISPR-mediated genome editing.

In the setting of monogenic disease, advances made in genome editing technologies can, in principle, be deployed as a therapeutic strategy to precisely correct a specific gene mutation in an affected cell type and restore functionality. Using the β-hemoglobinopathies and hemophilia as exemplars, we review recent experimental breakthroughs utilizing CRISPR-derived genome editing technology that have translated to significant improvements in the management of inherited hematologic disorders. Yet there are also challenges facing the use of CRISPR mediated genome editing in these patients and we discuss possible ways to obviate those issues for furtherance of clinical benefit.