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US 11,717,560 B2Granted
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.
Claims17
An isolated nucleic acid molecule, comprising from 5′ to 3′:
- a first inverted terminal repeat sequence,
- a human synapsin promoter sequence operably linked to a nucleic acid sequence encoding ATP1A3, wherein the nucleic acid sequence encoding ATP1A3 has at least 90% identity to the sequence set forth in SEQ ID NO:07, and
- a second inverted terminal repeat sequence.
Dependencies are inferred from the claim text.