For the millions of people living with genetic diseases like muscular dystrophy and inherited liver disorders, one of the ...

Urnov is a professor of molecular therapeutics at the University of California, Berkeley, and a director at its Innovative Genomics Institute. In May, news broke of a biomedical first: the on-demand ...

A research team has discovered an enhanced CRISPR gene-editing system that could enable targeted delivery inside the human ...

"Traditional gene-editing technologies work best with single mutations and are expensive to optimize, so gene therapies tend to focus on the mutations that are the most common," Buffington said. "But ...

By Nancy Lapid April 17 (Reuters) - Researchers have developed a modified version of the CRISPR gene-editing tool that in ...

WASHINGTON — Scientists are testing an entirely new way to fight heart disease: a gene-editing treatment that might offer a one-time fix for high cholesterol. It's very early stage research, tried in ...

Advanced biotechnology repurposes two bacterial immune systems to correct large stretches of DNA. Human cells that have been edited with the new retron-based gene editing technology. Orange dots mark ...

Late last year, dozens of researchers spanning thousands of miles banded together in a race to save one baby boy’s life. The result was a world first: a cutting-edge gene-editing therapy fashioned for ...

Editor’s note: On June 3, 2025, KJ Muldoon was discharged from the hospital and is now at home with his family. When a baby born in Philadelphia was announced as the first person to get a gene therapy ...

Gene therapy has long promised to provide a solution for genetic blood disorders, but delivering on that promise has proven complex due to challenges including difficulties delivering the editing ...