Experimental gene therapies like AMT‑130 lower toxic huntingtin protein and significantly slow Huntington’s disease, offering ...

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 ...

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, ...

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 ...

Even hearing the phrase “Huntington’s disease” will make a room suddenly somber. So the joy that accompanied a recent announcement of results of an experimental gene therapy for the deadly diseases ...

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 ...

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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 ...

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 ...

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 ...