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SRC-c940adf2333d2ac1 · Source record

Patient-Specific In Vivo Gene Editing to Treat a Rare Genetic Disease

New England Journal of Medicine · 1 connected claims

Canonical route

https://www.nejm.org/doi/full/10.1056/NEJMoa2504747

Claims connected to this source

  1. MWITA-FT-2026-001A customized lipid-nanoparticle CRISPR base-editing therapy was designed, authorized and administered to one infant with CPS1 deficiency within roughly six months; the patient tolerated two infusions and showed improved protein tolerance and reduced ammonia-control medication.Evidence B

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