Understanding how cells turn genes on and off is one of biology's most enduring mysteries. Now, a new technology developed by ...

The national feature highlighted the story of Lindsey Rambo, the second participant in Opus Genetics’ ongoing Phase 1/2 trial of gene therapy OPGx-LCA5, aimed at restoring vision for people living ...

Imagine if you can get your $100 million athlete back before the end of the season,” said Dr. Paul Liu, executive chairman of Pax Therapeutics.

A naturally occurring gene called Cyclin A2 (CCNA2), which turns off after birth in humans, can actually make new, functioning heart cells and help the heart repair itself from injury, including a ...

There are a number of considerations that can help streamline the pathway to the clinic. One of the first is which vector system will be used, with AAV and lentivirus being two of the most commonly ...

According to Bloomberg News, Vinay Prasad, who oversees gene therapies at the FDA, said the plans will be published in early November. He foresees the approach sparking curiosity for the next wave of ...

Most current approaches rely on adding nuclear localization signal (NLS) motifs to the ends of Cas9 to facilitate nuclear entry. However, this method is inefficient, and much of the Cas9 that is ...

In a world first, a bespoke gene-editing therapy benefitted one child. Now reseachers plan to launch a clinical trial of the ...

A top US regulator plans to unveil a faster approach to approving custom gene-editing treatments, a move designed to unleash a wave of industry investment that will yield cures for patients with rare ...

Patients are less likely to drop out of messaged-based therapy, but they develop stronger relationships with their therapists ...

Seven years after the first gene-edited babies were revealed, biotech startup Manhattan Genomics is reviving the idea of ...

In 1962, a pediatric neurology resident at Columbia's Neurological Institute and his colleagues in the College of Physicians ...