The gene editing treatment targets the relatively common 1717-1G>A splicing mutation, which does not respond to many current CF therapies.

The U.S. Food and Drug Administration on Thursday approved the first gene therapy for inherited hearing loss, a one-time ...

And the places we have called home in more welcoming parts of the world have helped shape our species — from our genes to our ...

Ataxia is more than just clumsiness—it’s a neurological condition that affects coordination, balance, speech, and even vision ...

Dr Aaron Wenger reveals how improvements in long-read sequencing technology is enabling the elucidation of complex disease ...

SYNGAP1 encephalopathy is a rare genetic disorder for which there is no treatment. It causes epilepsy, intellectual ...

The firm closed an oversubscribed Series B financing round to fund a pivotal trial of RTx-015 and advance a clinical trial in ...

They present a compelling story of radiation, mutation and survival against the odds. But the underlying science didn’t ...

First Ascent Biomedical's Functional Precision Oncology bridges that gap by testing living tumor cells to generate actionable data in about 10 days, helping physicians choose the right treatment ...

A gene expression score may be able to predict outcomes of adoptive cell therapy and prevent unnecessary surgeries in patients with uveal melanoma, researchers say. A gene expression score may be able ...