Currently, hereditary angioedema is estimated to affect one in 50,000 people around the world. However, due to its rarity, it is frequently misdiagnosed.
Recently, researchers from the University of Auckland, Amsterdam University Medical Center, and Cambridge University Hospitals achieved notable success in treating a group of patients with this hereditary disorder, marking a significant advancement in the field of medicine.
Targeted gene-editing therapy
The treatment, NTLA-2002, is a pioneering gene-editing therapy that employs CRISPR/Cas9 technology, aiming to treat hereditary angioedema–a genetic disorder characterized by severe, painful, and unpredictable swelling attacks that can interfere with daily life and potentially prove fatal.
The revolutionary aspect of this treatment lies in its ability to provide a permanent cure for the disabling symptoms associated with hereditary angioedema, including reducing the levels of total plasma kallikrein, a statement by the researchers noted.