The Aims of this project are twofold: Aim 1 – “To determine the effects of various approved drugs and experimental compounds on AHC features in D801Y mice”. The goal of this project will be to complete the ongoing investigation to determine if any specific drugs or experimental compounds can prevent cold-induced attacks of weakness and dystonia in D801Y mice. Aim 2 – “To measure pump current in neurons from D801Y mice” In previous experiments AHC neurons exhibited impaired sodium-potassium pump activity that could be directly measured as ‘pump current’ and provided the first direct evidence of ATP1A3 loss of function in a human neuron. The goal of this aim is to make similar measurements in neurons from D801Y mice then compare ATP1A3 function between mouse and human neurons. The resulting findings will contribute to the discovery of better pharmacological treatments for AHC and reveal new disease mechanisms that could be exploited for novel therapeutic strategies.
|Effective start/end date||7/1/18 → 8/31/19|
- Alternating Hemiplegia of Childhood Foundation (AGMT 12/3/18)
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