Custom gene edit treats baby with rare disease

CHOP and Penn Medicine reported a baby with severe CPS1 deficiency received a gene editing therapy designed just for his mutation, published in the New England Journal of Medicine.

WHERE THIS STANDS

Claim
Verified
Usable
In use

The case was peer reviewed and published in NEJM.

What moves it next: Moves to Usable when people can try it: a product, service, code or model becomes publicly available. How we decide

Personalized base editing · CHOP and Penn Medicine

WHY THIS MATTERS

It shows a path to fast custom cures for rare genetic diseases that have no treatment today.

Our interpretation of possible significance, not a promised outcome.

What happened?

CHOP and Penn Medicine reported a baby with severe CPS1 deficiency received a gene editing therapy designed just for his mutation, published in the New England Journal of Medicine.

What changed?

It was the first personalized CRISPR base editing therapy, designed and made in about six months.

What has been checked?

Peer reviewed paper

It is one patient, and longer follow up is needed to know how well and how long it works.

Can I use it today?

This entry covers a research result. Consult the original publication for released code, data, access conditions and experimental limitations.

See the evidence and full research details

Read the original sources

Source materials checked Oct 7, 2026. Leapscope has not independently replicated this result.

Original announcement or research