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 ...

Researchers at Penn’s School of Veterinary Medicine have developed new mechanisms to treat advanced forms of inherited retinal degeneration, bridging a previous gap in patient treatments. Raghavi ...

A single one-time gene therapy could free patients with α-thalassemia, a rare and debilitating blood disorder, from the burden of lifelong transfusions. A single one-time gene therapy could free ...

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, ...

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 ...

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

After the deaths of three patients, the maker of an investigational gene therapy stopped shipments of the drug after it refused a request from the U.S. Food and Drug Administration (FDA) to pull the ...

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 ...