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US 2024/0075111 A1Published application
Compositions Comprising Nucleic Acid Molecules and Methods of Treating ATPase-Mediated Diseases
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.
Claims21
-61. (canceled)
A method of treating an ATPase-mediated disease in a subject, the method 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.
A method of treating an ATPase-mediated disease in a subject, the method 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% identity to the 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.
A nucleic acid expression cassette comprising a nucleic acid sequence encoding an ATPase, wherein the encoded ATPase comprises ATP1A1, ATP1A2, ATP1A3, ATP2C1, ATP6A1, ATP6V1B1, ATP6V0A4, ATP7A, ATP7B, or ATP11C.
Dependencies are inferred from the claim text.