With funds raised from the AHC Foundation, we will support AAV gene therapy and other research related to the project. We believe a path has been identified to a viable permanent treatment: gene therapy. The gene therapy we propose will involve inserting a functional copy of the ATP1A3 gene into a virus (an adeno-associated virus or AAV) that has been stripped of its harmful payload and its ability to reproduce. The virus will then be injected into the cerebrospinal fluid and will carry the functional gene to the cells in the brain that are failing. By delivering additional functional copies of the gene, we should be able to rescue those cells, and treat the disease permanently.
|Anonymous Friend||04/10/2019||$350.00||Anonymous Friend||04/10/2019||$3,000.00|