In a medical first, a team at Children’s Hospital of Philadelphia and Penn Medicine has successfully treated an infant diagnosed with a rare genetic disorder by using a customized CRISPR gene editing therapy. The work, led by Penn Medicine’s Kiran Musunuru and CHOP’s Rebecca Ahrens-Nicklas, points to the potential to use bespoke gene editing therapies to treat others with rare genetic diseases for which no available medicines exist. We spoke to P.J. Brooks, deputy director of the Office of Rare Disease Research at the National Institutes of Health’s National Center for Advancing Translational Sciences, about the breakthrough treatment, how the researchers were able to move from diagnosis to treatment with great speed, and what it would take to scale such an approach.
