Angiodema: Gene therapy blocks painful hereditary disorder
4 Feb 2024
BBC
A single dose of gene therapy was enough to stop the painful swelling attacks caused by angiodema.
In the trial, published in the New England Journal of Medicine, angiodema symptoms were reduced by 95% among a small group of patients from the UK, New Zealand, and the Netherlands given a single infusion - targeted at cells in the liver - to reduce the KLKB1 gene's ability to produce plasma prekallikrein.