CRISPR/Cas9 gene editing therapy shows promise in treating hereditary angioedema

Researchers from the University of Auckland, Amsterdam University Medical Center, and Cambridge University Hospitals reported that over ten patients with hereditary angioedema (HAE) have been successfully treated with a CRISPR/Cas9 gene editing technology. Preliminary findings were published in The New England Journal of Medicine.

HAE is a rare genetic disease that causes severe, painful, and sudden swelling attacks of the skin, gut lining, and lungs. The swelling symptoms of HAE are caused by dysregulation of the peptide bradykinin. One therapeutic target for angioedema attacks is plasma kallikrein, which activates bradykinin. Treatments for HAE include inhibitors of plasma kallikrein activity, and newer RNA-silencing approaches that reduce total plasma kallikrein levels. However, these treatments require lifelong administration.