gene therapy

A set of four, or even three, molecules that could convert glial cells into new neurons has been identified by researchers at Pennsylvania State University. The simple drug cocktail could potentially be used to treat stroke, Alzheimer’s disease, and brain injuries. “The biggest problem for brain repair is that neurons don’t regenerate after brain damage… Read more

Growing up in Devon in the 1980s, brothers James* and Matt* had to be careful with their antics – a cut or bruise could land them in hospital. They have haemophilia, a genetic disease where the blood doesn’t clot efficiently due to a lack of a clotting protein. It’s plagued their family for generations. Their… Read more

An emerging gene therapy for Parkinson’s disease creates new circuits in the brain associated with improved motor movement, a new study indicates. The findings, from Feinstein Institute for Medical Research Professor David Eidelberg, MD, and his team, explain the therapeutic mechanisms involved in the emerging Parkinson’s gene therapy called AAV2-GAD. Parkinson’s disease is the second… Read more

A specific type of cell essential to the ability of olfactory neurons to regenerate may be genetically engineered to deliver anticancer therapy to the dangerous brain tumors called glioblastomas, researchers from Massachusetts General Hospital (MGH) report. The team used olfactory ensheathing cells to deliver an anticancer agent only to tumor cells and how the treatment… Read more

A therapeutic technique to transplant hematopoietic stem cells directly into the brain has been reported in a new study. This could represent a significant breakthrough in our approach to treating central nervous system diseases and neurodegenerative disorders. Researchers, from the Dana-Farber/Boston Children’s Cancer and Blood Disorders Center and the San Raffaele Telethon Institute for Gene… Read more