On behalf of the Alternating Hemiplegia of Childhood Foundation (AHCF) we are pleased to announce an award of $173,419.00 to the Department of Pharmacology, Northwestern University, Feinberg School of Medicine for the project “Exploring Mechanisms and the Therapeutic Opportunities in AHC”.
Under the direction of Dr Alfred George, the scientific team will use a multidisciplinary approach to investigate cellular mechanisms of AHC using human neurons and a new mouse model ATP1A3-G947R, to establish their clinical features (phenotype) and to investigate aspects of neuronal function that have not been examined in other mouse models.
Secondly, in the recent push for developing viral gene therapy for AHC, a concern has emerged that the presence of a dominant-negative mutant ATP1A3 allele may hinder efficacy of this therapeutic approach. A strategy to eliminate or suppress the mutant allele may be needed, therefore they will test oiligonucleotide (ASO) strategies to suppress ATP1A3 mutations in human neurons.
We are excited to discover additional clues to a cure and supplement our ongoing gene therapy work with this two year grant. The AHC community has made this research possible through their generous and ambitious work. Together we have but one mission: END AHC.
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