Scientists are testing an entirely new way to fight heart disease: whether gene editing might offer a one-time fix for high ...

A research team has successfully demonstrated the world's first gene-editing treatment for Leber's hereditary optic ...

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 ...

Researchers reprogrammed bacterial bridge recombinases to edit large genomic regions in mammalian cells, revealing a ...

Faced with a life-threatening metabolic disease, KJ’s doctors at Children’s Hospital of Philadelphia sprinted to create a ...

A new skin-applied gene therapy uses lipid nanoparticles to deliver gene editing into skin stem cells, correcting ...

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 is now reaching the mainstream, ushering in a new era of genetic manipulation. Traditionally, inserting or deleting entire genes, regulating their expression, and altering specific ...

Scientists achieved a genetic breakthrough by removing an extra human chromosome using gene-editing technology. This landmark study successfully deleted the surplus chromosome 21 responsible for Down ...

A multidisciplinary committee of experts studied the scientific underpinnings of human gene-editing technologies, their potential use in biomedical research and medicine -- including human germline ...