Scientists push fetal CRISPR editing to halt rare genetic disease before birth
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Progress grows in using CRISPR plus prenatal diagnostics to treat rare genetic disorders in the womb. Kiran Musunuru at the University of Pennsylvania, who in 2025 helped create the first personalized gene-editing treatment for an infant with a rare genetic disorder, works with William Peranteau at Children’s Hospital of Philadelphia; David Stitelman at Yale School of Medicine, Tippi MacKenzie at UCSF and Aijun Wang at UC Davis also pursue fetal editing. Musunuru said the worst disease damage must be prevented in utero, not after birth; after slow progress, he and Peranteau reported an unexpected finding.
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