Research
Having recently launched in March 2022, Aubrey’s Hope for a Cure does not currently support its own research projects.
With funds raised from Aubrey’s Hope for a Cure and in collaboration with international AHC organizations, we hope to provide financial support to global research in greatest need of resources to develop effective treatments and a cure for AHC. Aubrey’s Hope for a Cure also welcomes any grant proposal that furthers our mission.
Cutting edge gene therapy is underway at labs in the U.S.A., which are funded largely by the fundraising efforts of AHC families and organizations. Technologies being explored include Adeno Associated Virus (AAV) mediated gene therapy, CRISPR gene editing, and Antisense Oligonucleotides (ASO). You can learn more about these projects at the Alternating Hemiplegia of Childhood Foundation’s website here.
Research in ATP1A3 and AHC goes far beyond AHC patients. Ten different diseases are caused by mutations in the same gene that causes AHC and those diseases are likely to benefit from the treatments developed from AHC. AHC treatments may also rescue other channelopathies, epilepsies, and neurological disorders, with implications for hundreds of thousands of people living with related genetic diseases. The ATP1A3 gene is one of the most important genes in the brain, encoding the sodium potassium pump which uses 50% of the brain’s energy. Knowledge gained from AHC research has the potential to benefit other disorders that result from loss of function of the sodium potassium pump including more common diseases affecting adults such as Parkinson’s, Alzheimer’s, Stroke, and Blood Sugar disorders.
