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Institution · Hospital

Children's Hospital of Philadelphia

Where the first personalised gene-editing medicine was administered to an infant.

Simple explanation

This is the hospital that, with the University of Pennsylvania, designed and gave a gene-editing treatment built for one baby's specific mutation. It is also a long-standing centre for gene therapy in children.

Go deeper

Children's Hospital of Philadelphia, with the University of Pennsylvania, developed and administered the first patient-specific in vivo base-editing therapy, for an infant with severe CPS1 deficiency, in 2025. It has a long history in paediatric gene therapy including the development pathway of voretigene neparvovec for inherited retinal dystrophy.

Sources

Written by The CRISPR Atlas editorial team Last updated Aug 25, 2026

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Treatments

Bespoke base editor for CPS1 deficiency (the KJ Muldoon case)

Scientists

Kiran Musunuru
Educational information only This page is a reference, not medical advice. Research and regulatory status change; check the last-updated date above and confirm anything important against the primary sources listed.