"CRISPR Gene Therapy Shows Promise in Treating Hereditary Angioedema"

TL;DR Summary
Gene therapy using Crispr-Cas9 has shown promising results in treating hereditary angiodema, a genetic disorder causing painful and unpredictable swelling attacks. Patients treated with a single dose of the therapy showed little sign of further symptoms, with one patient experiencing only minor symptoms. The treatment has the potential to provide a permanent cure for this debilitating condition and could also be used to treat other genetic disorders. Larger trials and long-term monitoring are planned to assess the therapy's safety and efficacy.
- Angiodema: Gene therapy blocks painful hereditary disorder BBC.com
- 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
- Gene therapy hailed as ‘medical magic wand’ for hereditary swelling disorder The Guardian
- 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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