Angiodema: Gene therapy blocks painful hereditary disorder

Angiodema: Gene therapy blocks painful hereditary disorder

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. Source link