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Penn and CHOP will test gene therapy for rare diseases with a new FDA trial protocol
Philadelphia Inquirer

Penn and CHOP will test gene therapy for rare diseases with a new FDA trial protocol

A team led by Kiran Musunuru and Rebecca Ahrens-Nicklas of the Perelman School of Medicine and Children’s Hospital of Philadelphia will test the CRISPR framework for treating customized gene therapies for urea cycle disorders related to any one of seven genes.

Nov 13, 2025

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