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US 12,533,397 B2Granted
Methods of treating ATPase-mediated diseases with a nucleic acid encoding ATP1A3 and a neuron-specific promoter
AssigneeDuke University
InventorsMohamad Mikati, Arsen Hunanyan, Boris Kantor, Aravind Asokan, Ram Puranam, Dwight Koeberl
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Abstract
The present disclosure provides nucleic acid expression cassettes, vectors, compositions and methods for the treatment of ATPase-mediated diseases in a subject.
Claims15
A method for treating an ATPase-mediated disease comprising:
- administering to a subject in need thereof a therapeutically effective amount of a vector comprising a nucleic acid sequence encoding ATP1A3 and a neuron-specific promoter, wherein following administration of the vector, one or more symptoms of the ATPase-mediated disease are decreased and/or ameliorated;
- wherein the one or more symptoms of the ATPase-mediated disease comprise abnormalities affecting the subject's balance, gait, memory, and impulsivity and wherein the ATPase-mediated disease is alternating hemiplegia of childhood (AHC);
- wherein the nucleic acid sequence encoding ATP1A3 has at least 90% sequence identity to the nucleotide sequence set forth in SEQ ID NO:01, SEQ ID NO:02, or SEQ ID NO:07.
A method for treating an ATPase-mediated disease comprising:
- administering to a subject in need thereof a therapeutically effective amount of a vector comprising a nucleic acid sequence encoding ATP1A3 operably linked to a human synapsin promoter, wherein the nucleic acid sequence encoding ATP1A3 has at least 90% sequence identity to the nucleotide sequence set forth in SEQ ID NO:07, wherein following administration of the vector, one or more symptoms of the ATPase-mediated disease are decreased and/or ameliorated;
- wherein the one or more symptoms of the ATPase-mediated disease comprise abnormalities affecting the subject's balance, gait, memory, and impulsivity and wherein the ATPase-mediated disease is alternating hemiplegia of childhood (AHC).
Dependencies are inferred from the claim text.