Gene therapy trial death unnerves promising area of brain research
Gene therapy researchers were converging on a holy grail. A few years ago, researchers at labs and companies…
‘Giant step forward’ for Huntington’s — the scientist behind the first gene therapy
On a video call in early September, Sarah Tabrizi first saw the data that she and other researchers…
The baby whose life was saved by the first personalized CRISPR therapy
You have full access to this article via your institution. To the research team working to save him,…
US 3-Year-Old Is First to Be Treated for Rare, Fatal Disease
A California boy is defying bleak odds after becoming the first patient to receive a new gene…
California boy, 3, receives first-ever gene therapy for his rare disease: ‘It’s just so exciting’
A California toddler is the first person in the world to receive gene therapy to treat his devastating…
Prime editing-installed suppressor tRNAs for disease-agnostic genome editing
General methods DNA amplification was conducted by PCR using Phusion U Green Multiplex PCR Master Mix (ThermoFisher Scientific)…
can gene editing prevent heart disease?
Crystalline forms of cholesterol (blue) in a liver cell (purple), imaged using a scanning electron microscope.Credit: Steve Gschmeissner/Science…
CRISPR gene editing therapy cuts LDL cholesterol in small study
A CRISPR-based gene editing therapy dramatically reduced levels of low-density lipoprotein, often called “bad cholesterol,” and triglycerides in…
uniQure Provides FDA Update on AMT-130; BLA Timing Unclear
uniQure (NASDAQ: QURE) on Nov. 3, 2025 announced FDA feedback…
New gene-editing method can correct many disease-causing mutations in mammalian cells
Some genetic disorders-such as cystic fibrosis, hemophilia and Tay Sachs disease-involve many mutations in a person’s genome, often…
fixing them is about to get a lot easier
CRISPR gene editing has made its way into every corner of modern biology, but not into every corner…
U.S. Cell And Gene Therapy Clinical Trials Market Trends
Dublin, Oct. 03, 2025 (GLOBE NEWSWIRE) — The “U.S. Cell And Gene Therapy Clinical Trials Market Size, Share…