Rescuing Neuronal Function by Gene Therapy in Alternating Hemiplegia of Childhood

Project: Research project

Project Details


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 date3/1/216/30/22


  • Hope for Annabel, Inc. (AGMT 3/16/21 AMEND)


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