The goal of gene therapy is to permanently cure hereditary diseases. One of the most promising technologies for this is the ...

Research Ireland and APC will together fund new research into how advanced gene therapies are created and delivered.

Study results published in The New England Journal of Medicine show the therapy significantly improved hearing in nearly a ...

The search for gene therapies to treat rheumatoid arthritis (RA) has moved in fits and starts over the past two decades, with trials starting and stopping, and their focus shifting to osteoarthritis ...

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 ...

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 ...

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 have developed a DNA-based therapy that targets the PCSK9 gene to lower cholesterol naturally. Using polypurine ...

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, ...

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 ...