While views outside our window tell me we may need to wait, I am attempting with all my might to channel the sunshine and blue skies into existence. With that hopeful spirit and with Rare Disease Day approaching in a matter of days, I am asking for your help in meeting our fundraising deadline for gene therapy. Our hope is to develop a treatment and ultimately a cure for all AHC families impacted by this terrible disorder. Like never before, the families, foundations, and research groups have come together as a collective to move forward with this specific research.
ANYTHING you can spare will help us all tremendously - $2, $10, $25 or more will be significant in reaching our goal and giving a real chance to end AHC.
We are beyond grateful for your continued support as we move forward with sunshine and hope.
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.
|David Diaz||02/18/2019||$20.00||Happy to be an Ally against AHC. Love to Alaina!||Lee Dittmer||02/18/2019||$25.00||Anything for Alaina.||Ginger Crabtree||02/17/2019||$35.00||Hoping for a cure!!|