In a Phase 1, first-in-human trial, a one-time infusion of an investigational CRISPR-Cas9 therapy targeting angiopoietin-like protein 3 (ANGPTL3) was safe and reduced LDL cholesterol by nearly 50% and ...

Explore CRISPR technology explained, gene editing science, DNA modification, and genetic engineering ethics—discover how ...

A single infusion of CRISPR Therapeutics' experimental gene therapy was safe and reduced levels of harmful LDL cholesterol and triglycerides by half in four people taking the highest dose, raising ...

A company’s plan to edit the genomes of human embryos worries some researchers — but it might reflect the changing attitudes towards the controversial approach.

Scientists at Georgia State University have used CRISPR gene editing to restore an ancient enzyme humans lost millions of years ago potentially reversing the buildup of uric acid that causes gout.

We have written about the promise of gene therapy to correct genetic disease many times. An exciting development happened ...

Technology opens the door to more accurate preclinical testing, which could enable the development of safer and more effective therapies.

The Case Western Reserve University Science and Human Rights Coalition will host Andrea Waksmunski, a PhD candidate in the Department of Genetics and Genome Sciences, for a talk titled “Ethics of ...

CRISPR technology could one day create “designer babies, ” raising major ethical and social inequality concerns.

Although outbreaks of Ebola virus are rare, the disease is severe and often fatal, with few treatment options. Rather than targeting the virus itself, one promising therapeutic approach would be to ...

Turning genes on and off is like flipping a light switch, controlling whether genes in a cell are active. When a gene is turned on, the production of proteins or other substances is promoted; when ...