Can Gene Editing Treat Angelman Syndrome?
Scientists have determined how to overcome a genetic mutation at the center of this debilitating neurodevelopmental condition.
Category: Gene Therapy
Thanks to a UNC research project, she can walk again
As UNC geneticist Jonathan Berg gears up to lead a $9.7-million renewal of the NCGENES project, we look at a story from the original study and how it changed the fortunes of a woman from Goldsboro, NC.
With ‘new life’ after transplant, cancer survivor renews marriage vows
Recognizing that her husband now had a chance for a new, healthier life thanks to a stem cell transplant, Jackie Hannah secretly arranged for a ceremony for her husband David and her to renew their marriage vows. While he was in the Bone Marrow Transplant Unit in March, just after the transplant, an
Gene therapy cocktail shows promise in long-term clinical trial for...
This form of gene therapy was created and developed at the UNC School of Medicine. The treatment uses a virus (adeno-associated virus, or AAV) as a “viral vector” meticulously tailored to enter the brain and safely switch good genes for bad.