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

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, ...

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 ...

It’s now possible to treat inherited blood diseases, such as sickle cell disease, with gene editing. Blood stem cells are extracted from the patient, modified, and infused back into their bone ...

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 ...

Jason Mast is a general assignment reporter at STAT focused on the science behind new medicines and the systems and people that decide whether that science ever reaches patients. You can reach Jason ...

CHICAGO, Nov 8 (Reuters) - A single infusion of CRISPR Therapeutics' (CRSP.BN), opens new tab experimental gene therapy was safe and reduced levels of harmful LDL cholesterol and triglycerides by half ...

Aug 20 (Reuters) - The U.S. health regulator has allowed a study of Rocket Pharmaceuticals' (RCKT.O), opens new tab gene therapy to resume, less than three months after halting it following a ...

Lowering cholesterol is one of the most effective ways to reduce your risk of heart disease, and it may soon be possible to get a one-and-done gene therapy to keep cholesterol and triglyceride levels ...

This scan of a mouse brain shows blood vessels (red), brain cells (green), and amyloid plaques (blue). A new gene therapy treatment developed by UC San Diego works by reprogramming the behavior of ...