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 ...

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

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

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 ...

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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 ...

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 ...

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 ...

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 ...

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 ...