Promising results from a small clinical trial highlight a growing interest in designing gene-editing treatments for common ...

By reactivating a long-lost gene, researchers were able to lower uric acid levels and stop damaging fat accumulation in human ...

A single infusion of CRISPR Therapeutics' experimental gene therapy was safe and reduced levels of harmful LDL cholesterol ...

The investigational gene therapy is designed to inactivate the TTR gene and thereby dampen production of TTR in the liver. A ...

An experimental gene-editing therapy developed by Crispr Therapeutics is showing promise for treating heart disease.

In a world-first trial, scientists used a one-off CRISPR gene edit to switch off a liver “fat brake” gene, slashing stubborn ...

Scientists at St. Jude Children's Research Hospital and Northwestern University identified a previously unknown treatment ...

Researchers report early but encouraging results on an experimental gene-editing treatment for high cholesterol.

CRISPR Therapeutics' CTX310 trial shows strong, sustained lipid reductions and favorable safety, boosting confidence in its ...

Penn and CHOP researchers want to develop urea cycle disorder treatments using CRISPR gene-editing therapy. Get unlimited access to Inquirer.com and The Inquirer App, plus 5 articles each month to ...

In a 15-patient, Phase 1, first-in-human trial, a one-time CRISPR-Cas9 gene-editing therapy safely reduced LDL cholesterol ...

The approach is “very exciting,” but one expert says a lot more research is needed, especially around safety, before clinical ...