The purpose of this research study is to determine whether the viral delivery of an ATP1A3 transgene will correct the neurophysiological dysfunction in hiPSC-derived neurons with the ATP1A3-L839P mutation, and thereby demonstrate the efficacy of viral delivery of ATP1A3 cDNA to human neurons derived from a child with alternating hemiplegia of childhood (AHC), and the proof-of-concept for gene therapy in this disorder.
|Effective start/end date||3/1/21 → 6/30/22|
- Hope for Annabel, Inc. (AGMT 3/16/21 AMEND)
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