An experimental gene therapy seems to slow the progression of Huntington’s disease by about 75 per cent, and researchers are ...

Now, Musunuru and colleagues are preparing a master clinical protocol to study a CRISPR therapy that's mostly the same as the one they used to treat baby KJ, except they plan to swap out the gene ...

X-linked retinoschisis gene replacement therapy, aiming to improve retinal architecture and visual function in young male ...

A small group of volunteers will receive multiple injections of the experimental treatments next month, says Unlimited Bio.

Using X-ray diffraction data, James Watson (1928-2025) made the seminal discovery of the double-helical structure of DNA ...

From rare diseases to chronic conditions, epigenetic editing unlocks new ways to control gene activity without altering the genetic code.

Renowned molecular biologist Yunsun Nam, Ph.D., Professor in the Departments of Biochemistry and Biophysics and the Doris and ...

Researchers discovered that a tiny structural feature of the enzyme GPX4 helps keep neurons safe. A rare mutation removes this protection, allowing harmful molecules to damage cell membranes and ...

“Bubble boy disease” was once a death sentence. A scientific breakthrough changed that. By Simar Bajaj A common cold was enough to kill Cora Oakley. Born in Morristown, N.J., with virtually no immune ...

A California toddler is the first person in the world to receive gene therapy to treat his devastating disease. Three-year-old Oliver Chu was born with a rare, genetic condition called Hunter syndrome ...

When Tim Hunt was invited to a meeting with Robert F. Kennedy Jr. in early March, the Department of Health and Human Services (HHS) secretary had only been sworn in a few weeks earlier. But RFK Jr.

In a world-first trial, scientists used a one-off CRISPR gene edit to switch off a liver “fat brake” gene, slashing stubborn LDL cholesterol and triglycerides in patients whose levels refused to budge ...