JABSOM Cell and Molecular Biology researcher Dr. Jesse Owens has spent the better part of two decades chasing a vision that ...

For decades, scientists have dreamt of a future where genetic diseases, such as the blood clotting disorder hemophilia, could be a thing of the past. Gene therapy, the idea of fixing faulty genes with ...

Scientists from the Gray Faculty of Medical & Health Sciences at Tel Aviv University introduced an innovative gene therapy method to treat impairments in hearing and balance caused by inner ear ...

A novel gene therapy enabled children with congenital deafness due to otoferlin (OTOF) gene variants to hear for the first time, the pivotal phase I/II CHORD study showed. Of 12 such children treated ...

Gene therapy is a revolutionary field in modern medicine, offering transformative potential to treat and potentially cure a wide range of genetic and acquired diseases. By addressing the root cause of ...

A new gene therapy can reverse the effects of heart failure and restore heart function in a large animal model. The therapy increases the amount of blood the heart can pump and dramatically improves ...

For adeno-associated viral gene therapies to scale, scientists must first address safety and manufacturability challenges. Improving payload design and optimizing production systems can help ...

Scientists at St. Jude Children's Research Hospital and Northwestern University identified a previously unknown treatment opportunity for sickle cell disease and β-thalassemia. The discovery, ...