FDA rejects Regenxbio’s MPS II rare-disease gene therapy
Adam Feuerstein is a senior writer and biotech columnist, reporting on the crossroads of drug development, business, Wall…
Exclusive: In major test for longevity field, FDA greenlights study on a 'near total reset' of cells – Endpoints News
Exclusive: In major test for longevity field, FDA greenlights study on a ‘near total reset’ of cells Endpoints NewsScientists…
New FDA cell and gene therapy oversight system comes with risks
At a Food and Drug Administration cell and gene therapy (CGT) roundtable last June, most speakers pushed for…
Science in 2026: what to expect this year – Nature
Science in 2026: what to expect this year NatureAll the eclipses, supermoons, meteor showers and planets to spot in…
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…