Gene therapy for neurodegenerative disorders
AssigneeParos Bio, Inc.
InventorsKimberley S. Gannon, Martin Goulet, Neil R. Hackett
Life of this patent
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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.
Claims31
An isolated polynucleotide set forth in SEQ ID NO:6, SEQ ID NO:7, SEQ ID NO:8, SEQ ID NO:36, SEQ ID NO:37, SEQ ID NO:38, or SEQ ID NO:39;
- or having at least 95% sequence identity to SEQ ID NO:6, SEQ ID NO:7, SEQ ID NO:8, SEQ ID NO:36, SEQ ID NO:37, SEQ ID NO:38, or SEQ ID NO:39, and encoding the same polypeptide encoded by each of the foregoing, wherein the polynucleotide is not naturally occurring.
A nucleic acid expression cassette comprising:
- (I) a polynucleotide encoding presenilin 1, wherein the polynucleotide comprises any one of:
- (a) a polynucleotide set forth in SEQ ID NO:6, SEQ ID NO:7, SEQ ID NO:8, SEQ ID NO:36, SEQ ID NO:37, SEQ ID NO:38, or SEQ ID NO:39;
- or
- (b) a polynucleotide having at least 95% identity to SEQ ID NO:6, SEQ ID NO:7, SEQ ID NO:8, SEQ ID NO:36, SEQ ID NO:37, SEQ ID NO:38, or SEQ ID NO:39;
- and
- (II) one or more regulatory elements operably linked to the polynucleotide encoding presenilin 1.
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:6, SEQ ID NO:7, SEQ ID NO:8, SEQ ID NO:36, SEQ ID NO:37, SEQ ID NO:38, or SEQ ID NO:39;
- or by a vector comprising a polynucleotide having at least 95% identity to SEQ ID NO:6, SEQ ID NO:7, SEQ ID NO:8, SEQ ID NO:36, SEQ ID NO:37, SEQ ID NO:38, or SEQ ID NO:39, and encoding the same polypeptide encoded by each of the foregoing;
- 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 wild-type polynucleotide in a host cell, thereby producing presenilin 1 protein.
Dependencies are inferred from the claim text.