For the first time, the tools to rewrite human biology are moving from speculative fiction into regulated clinics and ...

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 ...

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

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 ...

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 ...

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 ...

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 ...

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 ...

“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 ...