William Peranteau, M.D. seeks to transform the lives of infants before they are born, in the hopes of providing a life free of the morbidity and mortality of certain birth defects. He is part of the team at The Children’s Hospital of Philadelphia’s (CHOP) Center for Fetal Diagnosis and Treatment (CFDT), which counsels families with pregnancies complicated by structural birth defects. In select circumstances, the CFDT team surgically repairs anomalies while still in utero. But Peranteau’s goal is to not only treat structural birth defects, but to mitigate genetic diseases in their tracks at the genetic level prior to birth — allowing a child to grow and develop without ever facing their effects.
“Every patient with a genetic disease deserves to have a team try to develop and optimize a therapy that could be life-changing for that patient and their family,” said Peranteau, who is a Professor of Surgery at the University of Pennsylvania and the Adzick-McCausland Distinguished Chair in Fetal and Pediatric Surgery at CHOP.
His work centers on taking advantage of normal developmental biology of the fetus to develop gene therapies that are safer and more effective. The ultimate goal is to provide the therapy before the onset of irreversible disease pathology, focusing on diseases that start before or shortly after birth or those in which postnatal therapies have proven very difficult. Specifically, the Peranteau lab develops and optimizes in utero gene editing therapies using CRISPR technology, including adenine base editing (ABE), which can change single DNA bases without cutting the DNA strands in their entirety. Changing the base allows the strand to replicate the new vocabulary, so to speak — and a genetic abnormality can be, essentially, rewritten and fixed. The team is optimizing non-viral delivery approaches for these therapies for metabolic and genetic muscle diseases in fetuses in experimental models with an eye towards clinical translation.
Peranteau earned his B.A. from Princeton University, M.D. from the Perelman School of Medicine at the University of Pennsylvania, and was concurrently a Howard Hughes Medical Institute Medical Student Fellow conducting research in prenatal cell and gene therapy. He completed his General Surgery training at the Brigham and Women’s Hospital and his Pediatric and Fetal Surgery training at CHOP, and is board-certified in pediatric surgery and general surgery. He won the NIH Director’s New Innovator Award in 2020, and his work has also been supported by the NIH Common Fund’s Somatic Cell Genome Editing Program, the Gerber Foundation, and the American College of Surgeons, among others.
He has co-authored more than 200 articles, reviews, and papers, in publications including Nature Medicine, Nature Biomedical Engineering, Science Translational Medicine, Journal of the American College of Surgeons, the Journal of Pediatric Surgery, Nature Reviews Bioengineering, and Fetal Diagnosis and Therapy.
We’re delighted that Dr. Peranteau is joining the DOC 2026 Faculty, and you can hear more from him when he joins Nicole Gaudelli, Ph.D., to talk about ABE technology during the DOC 2026 session, “Gene Editing 2.0.”