International collaborations, refined gene-editing protocols, and targeted immunologic maneuvers keep the cancer gene therapy ...

For the last 10 years, the only effective treatment for hypophosphatasia (HPP) has been an enzyme replacement therapy that ...

Fondazione Telethon, an Italian biomedical charity focused on rare genetic diseases, has submitted a Marketing Authorization ...

In two new papers, researchers from The Jackson Laboratory (JAX) report the successful use of two approaches—gene therapy and ...

The California Institute for Regenerative Medicine has awarded a $6 million grant to USC investigators pioneering a new first ...

Gene therapy promises individualised management of disease for each patient. Even if two individuals suffer from the same ...

The majority are in the very early preclinical stage of development, but applications for regulatory approval have been ...

Researchers have developed a gene therapy that may provide a long-term treatment for hypophosphatasia. Unlike current ...

Previous research has shown that voretigene neparvovec administered subretinally early in childhood for RPE65-mediated ...

For the last 10 years, the only effective treatment for hypophosphatasia (HPP) has been an enzyme replacement therapy that ...

The gene therapy Casgevy, which will now be offered to some patients in England, could help people live disease-free, experts ...

A new gene editing treatment has been approved for use in the NHS, which will help treat sickle cell disease patients ...