Personalised in vivo gene-editing therapy
Kiran MusunuruRebecca Ahrens-Nicklas
This page delivers no verdict; it presents claims and evidence with their sources. No theory in science is absolute. Method
For everyone
In 2025 in the US, a baby named KJ, born with a severe rare metabolic disease (CPS1 deficiency), received a base-editing therapy designed and manufactured in six months solely to correct his mutation. The results were published in the New England Journal of Medicine. The long-term effect and safety of this single-patient result are not yet known.
For specialists
The therapy consisted of adenine base-editor mRNA and a patient-specific guide RNA delivered to the liver in lipid nanoparticles. Three doses were given between February and April 2025. Protein intake could be increased and the need for nitrogen-scavenger medication fell; no serious adverse events were reported. The fraction of liver cells edited could not be measured directly.
Claims
- A gene-editing therapy tailored to a single patient's mutation can be designed within months and administered safely.[1]
Evidence
Supporting
After treatment the patient's blood ammonia levels stayed under control and dietary tolerance increased.[1]
Challenging
The result rests on a single patient and short follow-up; because the patient also received standard treatments, how much of the effect is due to the edit cannot be separated with certainty.[1]
Connections
Sources
- Article (2025). Patient-specific in vivo gene editing to treat a rare genetic disease. New England Journal of MedicineDOI 10.1056/NEJMoa2504747archive
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Cite this page
Athena contributors. (2026). Personalised in vivo gene-editing therapy. Athena: The tree of human knowledge. Retrieved November 1, 2026, from https://latifkedi.github.io/athena/en/d/kisiye-ozel-crispr/
@misc{athena_kisiye_ozel_crispr,
title = {Personalised in vivo gene-editing therapy},
author = {{Athena contributors}},
year = {2026},
howpublished = {\url{https://latifkedi.github.io/athena/en/d/kisiye-ozel-crispr/}},
note = {Athena: The tree of human knowledge. Accessed: 2026-11-01}
}Added: September 27, 2026 · Last changed: September 28, 2026 · Page history