Another rare and potentially fatal disease cured by Crispr-Cas9 genetic scissors

A new rare but potentially fatal genetic disease, hereditary angioedema, has been cured by gene therapy. And for transthyretin amyloidosis in 2021, a single injection of treatment using the Crispr-Cas9 genetic scissors technique was enough to free patients from lifelong treatment. This is a pilot study conducted on 10 patients and published in the journal New England Journal of Medicine Who has just announced it.

Hereditary angioedema manifests itself as a sudden, irregular and sometimes painful swelling of certain tissues, especially in the skin and digestive tract. Attacks, which usually begin in childhood, are transient but can lead to death by suffocation if they affect the upper airways. In France, 1,500 people are affected by the disease, of which 800 are already being monitored by the National Reference Center for Angioedema (Criac). is”