Gene therapy for sickle cell and β-thalassemia works by disrupting three-dimensional genome structure

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

Gene Therapy Shows Lipid Improvement but Raises Flags

A CRISPR-based gene therapy showed improvement in lipids in difficult-to-treat patients, but one study raised flags after a ...

CRISPR gene therapy slashes 'bad' cholesterol, triglycerides by half in small study

A single infusion of CRISPR Therapeutics' experimental gene therapy was safe and reduced levels of harmful LDL cholesterol and triglycerides by half in four people taking the highest dose, raising ...
STAT

Critics call on Sarepta to show how it’s measuring gene therapy’s performance

Jason Mast is a general assignment reporter at STAT focused on the science behind new medicines and the systems and people that decide whether that science ever reaches patients. You can reach Jason ...
Time

A Huntington’s Disease Treatment Is Closer Than Ever

Gene therapy is becoming a powerful way to treat challenging diseases that don’t respond to traditional treatments, and researchers now report the first success in modifying genes to slow Huntington’s ...
Scientific American

Gene Editing Helped One Baby—Could It Help Thousands?

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