"Breakthrough Gene Therapy Offers Hope for Hereditary Angioedema"

TL;DR Summary
A groundbreaking gene therapy has shown promising results in treating hereditary angioedema, a rare disorder causing painful and potentially fatal swelling. Patients in the trial experienced dramatic improvements, with some being able to come off long-term medication and return to normal life. The therapy, using Crispr gene editing, aims to provide a permanent cure by targeting the genetic mutation causing the condition. While larger trials are ongoing, the high cost of one-shot gene therapies may limit accessibility, but the results offer hope for a potential cure for some sufferers.
Topics:health#clinical-trials#gene-therapy#hereditary-angioedema#medical-breakthrough#medicine#nobel-prize
- Gene therapy hailed as ‘medical magic wand’ for hereditary swelling disorder The Guardian
- CRISPR-Cas9 In Vivo Gene Editing of KLKB1 for Hereditary Angioedema | NEJM nejm.org
- CRISPR gene therapy seems to cure dangerous inflammatory condition New Scientist
- Angiodema: Gene therapy blocks painful hereditary disorder BBC.com
- Intellia Therapeutics Announces Publication of Positive Interim Phase 1 Data for NTLA-2002 in Patients with Hereditary Angioedema in the New England Journal of Medicine Yahoo Finance
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