Gene therapy with CRISPR 'in vivo' opens the way to the cure of hereditary angioedema. The results show high efficacy with a single administration, as well as safety, a short observation period of 16 weeks.
The European Medicines Agency recommends authorizing the first treatment based on CRisPR gene editing technology. For the allergist "the results are promising, although the study includes only ten patients and longer-term studies are needed with more patients," says specialist Alicia Prieto.