Researchers have found that a new base-editing gene therapy can help treat a rare neurodevelopmental disorder called Snijders Blok–Campeau syndrome caused by mutations in the CHD3 gene. A specialized ...

Engineers at the University of Pennsylvania and Rice University have refined a technology for editing individual genetic ...

Engineers at the University of Pennsylvania and Rice University have refined a technology for editing individual genetic "base pairs" to a new level of precision, opening the door to safer, more ...

Scientists have developed a new technology called base editing that allows them to rewrite specific DNA letters without ...

After dumping its sole remaining gene therapy asset last year, Pfizer has decided to exercise its option for global rights to Beam Therapeutics’ liver-targeted gene editing candidate.

Patients with T cell acute lymphoblastic leukemia (T-ALL) require allogeneic hematopoietic stem cell transplantation (HSCT) if they relapse or do not respond to standard chemotherapy. But successful ...

Researchers develop a single genome-editing strategy to treat multiple disorders caused by nonsense mutations, promising efficient and cost-effective therapies.

Mayo Clinic researchers have developed a promising gene-editing therapy that directly corrects a genetic mutation responsible for autosomal dominant polycystic kidney disease (ADPKD), the most common ...

Researchers at the Sanford Stem Cell Institute have taken the fight against rare diseases to the lab, to patients’ bedsides ...

A chubby-cheeked baby called KJ made medical history last year. Faced with a life-threatening metabolic disease, KJ’s doctors at Children’s Hospital of Philadelphia sprinted to create a personalized ...

In a world where agriculture is confronted with unprecedented challenges—such as climate variability, emerging pathogens, and ...