CRISPR-based therapy shows promise for reducing hereditary angioedema attacks
Hereditary angioedema, a C1 inhibitor deficiency that is caused by an excess of plasma kallikrein. The genetic absence of prekallikrein (Fletcher syndrome) appears to be associated with normal health and life expectancy. Researchers therefore sought to assess safety and efficacy by evaluating NTLA-2002 (Intellia Therapeutics), an experimental CRISPR/Cas9-based therapy that targets KLKB1 – the gene […]