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US 12,491,267 B2Granted
Gene therapy for neurodegenerative disorders
AssigneeThe Brigham and Women's Hospital, Inc.
InventorsKimberley S. Gannon, Martin Goulet, Neil R. Hackett
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Abstract
The disclosure relates to nucleic acid expression cassettes for the treatment of neurodegenerative disorders. Methods of treating neurodegenerative disorders such as Alzheimer's disease, frontotemporal dementia, frontotemporal lobar degeneration, Pick's disease, Lewy body dementia, memory loss, cognitive impairment, and mild cognitive impairment are also provided.
Claims29
An isolated polynucleotide set forth in SEQ ID NO:
- 37 or SEQ ID NO:
- 39, wherein the polynucleotide is not naturally occurring.
A nucleic acid expression cassette comprising:
- (I) a polynucleotide encoding presenilin 1, wherein the polynucleotide comprises:
- (a) a polynucleotide set forth in SEQ ID NO:
- 37 or SEQ ID NO:
- 39;
- and
- (II) one or more regulatory elements operably linked to the polynucleotide encoding presenilin 1.
3 more elements
A method of producing presenilin 1 protein comprising:
- transforming a host cell with a vector comprising an optimized polynucleotide set forth in SEQ ID NO:
- 37 or SEQ ID NO:
- 39;
- and
- culturing the cell under conditions and for a time that allow expression of the presenilin 1 protein, wherein the expression of the presenilin 1 protein encoded by the optimized polynucleotide in the host cell is greater than a level of expression of presenilin 1 protein encoded by a wilt-type polynucleotide in a host cell, thereby producing presenilin 1 protein.
2 more elements
Dependencies are inferred from the claim text.