New one-dose treatment edits a gene in the liver, disabling the ANGPTL3 protein and significantly lowers cholesterol.

Few companies stand better poised than Beam Therapeutics to reap the first fruits of the FDA’s promised flexibility toward cell and gene therapies. | Riding a regulatory win for its base editing ...

As CRISPR moves from lab success to real-world medicine, 2026 will test whether these revolutionary gene-editing therapies can reach the patients who need them most or remain costly, complex ...

“Through CRISPR RNA optimization, we achieved around 59% editing at the TTR locus in our in vitro experiments. In the mouse ...

Gene therapy represents one of medicine’s most ambitious attempts to treat disease at its root cause by altering the genetic code itself. The approach works by recovering the functions of critical ...

A new gene-editing technique enables the correction of multiple genetic mutations simultaneously, transforming the prospects for millions living with complex inherited diseases, such as cystic ...

Gene-editing techniques such as CRISPR-Cas9 have many uses in the area of food and agriculture. They can combat persistent drought and disease, and improve the colour and nutritional content of food ...

A gene-editing delivery system developed by UT Southwestern Medical Center researchers simultaneously targeted the liver and lungs of a preclinical model of a rare genetic disease known as alpha-1 ...

Revolutions in gene therapy are rapidly changing the landscape of modern medicine. Revolutions in gene therapy are rapidly changing the landscape of modern medicine, forcing society and science alike ...

An infant with a rare metabolic disease became the world’s first patient to be treated with a personalized CRISPR gene-editing treatment in a landmark study between Penn Medicine and the Children’s ...

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 ...