OK, sorry for the acronyms but I just couldn’t resist. CRISPR stands for clustered regularly interspaces short palindromic repeats, so maybe you’ll forgive me. HANE is hereditary angioneurotic edema (commonly known as Hereditary Angioedema). The New England Journal of Medicine recently published research done at Amsterdam University using the CRISPR gene editing tool to treat/cure hereditary angioedema.  

HAE is a genetic disorder that is autosomal dominant (only requires one defective gene) that affects one in 50,000 people, but it can be a debilitating and sometimes fatal condition that requires lifelong treatment. These treatments are both preventative and crisis management in nature. The work, but they require frequent dosing to achieve control. Because of this the Amsterdam Scientists did a small study (27 patients) to see if gene editing could fix the problem. They used the CRISPR technology to edit the gene which encodes Kallikrein BI (KLKBI) the primary source of PR kallikrein. This editing resulted in a reduction in total plasma kallikrein levels which had been the cause of the angioedema attacks. The levels were reduced by 86% and 73% of the patients ended up attack free. Currently, gene editing therapy costs 3 to 4 million dollars do the cost will need to come down to make it economically viable.