Dr. Stuart Turvey said there have been other treatments for chronic granulomatous disease, involving hematopoietic stem cell ...

Ty Sperle says he felt "insane shock" after learning he'd been cured of a rare genetic disease through a clinical trial using ...

Scientists at Texas A&M are turning an everyday pick-me-up into a high-tech medical switch. By combining caffeine with CRISPR ...

One year after receiving CRISPR gene editing therapy at CHOP, a Delaware County baby is thriving, and doctors want to expand ...

Scientists around the world are hard at work figuring out how to combat the many forces threatening the global food supply.

Vector-borne diseases (VBDs) represent a critical challenge in infectious disease control due to their complex transmission dynamics, diverse vector ...

FDA's Plausible Mechanism Framework streamlines individualized therapy approvals for ultra-rare diseases.

Engineers at the University of Pennsylvania and Rice University have refined a technology for editing individual genetic "base pairs" to a new level of precision, opening the door to safer, more ...

Engineers at the University of Pennsylvania and Rice University have refined a technology for editing individual genetic ...

Researchers have found that a new base-editing gene therapy can help treat a rare neurodevelopmental disorder called Snijders Blok–Campeau syndrome caused by mutations in the CHD3 gene. A specialized ...

A version of this Priestley Medal address will be presented at the American Chemical Society Spring 2026 meeting ...