Researchers have unveiled a new DNA-based therapy that silences the PCSK9 gene to lower cholesterol without statin side effects. Early results show dramatic reductions in cholesterol levels, offering ...

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 in the lab of Hans-Peter Kiem, MD, PhD, at Fred Hutch Cancer Center have devised a method that could one day treat genetic hematologic disorders by correcting how the body makes blood ...

Genetic medicines could treat certain diseases, however, the current delivery systems available—viral vectors and lipid nanoparticles (LNPs)—pose unique challenges for gene therapy applications. For ...

Smarter design through targeted delivery and human-relevant testing can save the industry from costly safety failures.

The gene therapy for neurological diseases market is poised for growth due to rising neurological disorder prevalence, demand ...

Gene therapy approaches include gene replacement, suppression, and editing, each matched to specific genetic mechanisms in hereditary hearing loss. Preclinical studies in rodent models show promise, ...

Gene editing is already being used to treat a handful of rare genetic diseases; however, most therapies involve breaking or inactivating genes. Precisely fixing disease-causing mutations is far more ...

Shares of Sarepta Therapeutics plunged more than 30% on Friday as the future of its approved gene therapy treatment appeared at risk. The Food and Drug Administration will request that the company ...

A baby born with a rare and dangerous genetic disease is growing and thriving after getting an experimental gene editing treatment made just for him. Researchers described the case in a new study, ...