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

"Gene editing takes centre stage in FDA’s new rare disease approval pathway" was originally created and published by ...

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

UCLA researchers have developed a lipid nanoparticle-based gene-editing approach capable of inserting an entire healthy gene into human airway cells, restoring key biological function in a laboratory ...

UCLA researchers have developed a lipid nanoparticle-based gene-editing approach capable of inserting an entire healthy gene ...

Scientists are testing whether gene editing might offer a one-time fix for high cholesterol, and approaches being developed ...

The Food and Drug Administration aims to evaluate treatments for rare diseases based on plausible evidence that they would ...

TSRA-196 is designed to precisely correct the genetic mutation underlying AATD, with the goal of restoring production of functional alpha-1 antitrypsin (AAT) protein through a one-time, durable ...

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 ...

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