Researchers at University of California San Diego School of Medicine have developed a gene therapy for Alzheimer’s disease that could help protect the brain from damage and preserve cognitive function ...

Cell and gene therapies use the body's building blocks - cells and genetic material - to boost the immune system or correct ...

A teenage girl is alive thanks to an experimental gene therapy treatment created at UCLA. She was born with a rare genetic disorder, so any common infection could've killed her. But instead of living ...

Gene therapy has been effective for young children with genetic hearing loss, but this is the first study to show promising results in older patients. Reading time 3 minutes In recent years, gene ...

An experimental gene therapy was found to slow the progression of Huntington’s disease in a “pivotal” Phase 1/2 study, according to drugmaker uniQure, marking a major step toward a potential first ...

Viral vectors dominate gene therapy, with lentivirus, adenovirus, and AAV being key players, each with unique advantages and limitations. Non-viral vectors, such as lipid nanoparticles and GalNAc, ...

ELRIG, a not-for-profit, volunteer-led organisation dedicated to the global drug discovery community, today announced the ...

Columbus, Ohio is becoming a global hub for gene therapy, but its contributions often go unmentioned in national media. Two of the first eight FDA-approved gene therapies were invented and developed ...

LEANDER, Texas — Andrew Revell leaned into his father as he pulled up from his wheelchair to stand, his legs trembling with effort. It had been more than a year since he joined a clinical trial for a ...

Forbes contributors publish independent expert analyses and insights. A patient in a late-stage gene editing trial for a rare heart condition died from fatal liver complications after receiving an ...