First-in-Human In Vivo Base Editing for CPS1 Deficiency Demonstrates Feasibility of Patient-Customized CRISPR Therapy.
A single-patient expanded-access study showcases the rapid development and safe delivery of lipid nanoparticle–based adenine base editing targeting a rare metabolic disorder.
TribeMD
4 min read
July 23, 2025
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Rare Diseases
Sources
- Musunuru K, Grandinette SA, Wang X, et al. Patient-specific in vivo gene editing to treat a rare genetic disease. N Engl J Med. 2025;390(20):1–9. doi:10.1056/NEJMoa2504747
Highlights
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SnackableHealth® |Non-cardiac effects in HELIOS-B trial: gastrointestinal symptoms and quality-of-life signals discussed by Drs. Mike Gibson and Tony Urey
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