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 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 ...

A large genetic screen has revealed how stem cells transform into brain cells, exposing hundreds of genes that make this ...

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 ...