Children born with severe-to-profound hearing loss caused by mutations in the OTOF gene now have a treatment option that did ...

In utero gene therapy uses prenatal genetic screening to identify fetuses with lethal mutations and delivers corrective genes ...

Scientists from the Centenary Institute and the University of Sydney have made a landmark discovery that could lead to safer and more effective gene therapies for a range of serious genetic disorders ...

For years, cell and gene therapies were mainly associated with rare and orphan diseases, those that impact small patient groups and lack effective treatment options. However, recent developments in ...

A research team at the University of Osaka has unveiled the molecular mechanism behind genome ejection from adeno-associated virus (AAV) vectors, a crucial delivery vehicle in gene therapy. The study, ...

The idea behind intra-articular gene therapy for treating osteoarthritis (OA) is to deliver the gene-altering vector or cells directly to the precise site of the disease with a single injection — so ...

The rapid progression of genetic engineering, regenerative medicine, and pharmacology has positioned gene, cell, and ...