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 ...

Can a One-Time Gene Therapy Lower Cholesterol?

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

First-in-human trial of CRISPR gene-editing therapy safely lowers cholesterol and triglycerides

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

Gene therapy delivery system successfully targets CD90+ hematopoietic stem cells in vivo

Researchers in the lab of Hans-Peter Kiem, MD, PhD, at Fred Hutch Cancer Center have devised a method that could one day treat genetic hematologic disorders by correcting how the body makes blood ...
Morningstar

Biologic Competition, Payer Pressures, and Gene Therapy Momentum Define a Pivotal Moment in nAMD and DME Management, According to Spherix Global Insights

Exton, Pennsylvania, July 10, 2025 (GLOBE NEWSWIRE) -- Recently released research from Spherix Global Insights provides a timely look at the increasingly competitive and complex treatment landscape ...
News Medical

AAN releases Evidence in Focus Article on new gene therapy for Duchenne muscular dystrophy

To help neurologists, clinicians and families understand the current evidence for a new gene therapy for Duchenne muscular dystrophy called delandistrogene moxeparvovec, the American Academy of ...
Medscape

Mutation-Agnostic Gene Therapy May Restore Vision in RP

A new approach to gene therapy for retinitis pigmentosa may help patients with advanced disease regain vision regardless of which genetic mutation is causing the condition. An optogenetic therapy ...
The Bakersfield Californian

Biologic Competition, Payer Pressures, and Gene Therapy Momentum Define a Pivotal Moment in ...

Exton, Pennsylvania, July 10, 2025 (GLOBE NEWSWIRE) -- Recently released research from Spherix Global Insights provides a timely look at the increasingly competitive and complex treatment landscape ...