Help us give Kathryn the childhood she deserves! It isn't too late to improve the quality of life for all AHC kids.
Kathryn loves being outside, camping with the family is a new favorite! Episodes are inevitable, and often difficult to predict. When she has an episode, and is paralyzed on one half of her body, she still tries to play and walk. During episodes, it is as if she is behind a screen and can’t move or talk, but she understands everything. So we are inspired to be hopeful and strong for her. And we know that with the strides researchers and doctors are making every day, there is an answer for her and all AHC kids.
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.
|Rosalie Porter||04/27/2019||$25.00||Rosalie Porter||04/26/2019||$20.00|