Key points from article :
A new CRISPR gene therapy seems to have cured 9 out of 10 people with a potentially deadly swelling condition.
Nine out of 10 individuals with hereditary angioedema, a rare genetic inflammatory condition, participated in the first trial of this new CRISPR-based gene therapy.
The treatment involves making cuts in the gene for kallikrein, a protein associated with inflammation, and was administered directly into the body, a method known as "in vivo" delivery.
Participants experienced a 95% reduction in swelling attacks within six months of treatment, with subsequent long-term remission observed in most cases.
The therapy targets mutations in the gene encoding C1-inhibitor, a protein involved in inflammation regulation.
Administered by Intellia Therapeutics, the therapy utilizes lipid nanoparticles to deliver genetic material into liver cells where kallikrein is produced.
Published in NEJM, the success of the trial offers promise for treating various genetic conditions using CRISPR-based therapies.
