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US 12,753,411 B2Granted
Gene therapy for neurodegenerative disorders using polynucleotide silencing and replacement
AssigneeThe Brigham and Women's Hospital, Inc.
InventorsKimberley S. Gannon, Neil R. Hackett, Martin Goulet
Life of this patent
Estimating the term…
Abstract
The disclosure relates to nucleic acid expression cassettes and vectors 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.
Claims20
An expression cassette comprising:
- a. a first polynucleotide comprising SEQ ID NO:
- 13, wherein the first polynucleotide is operably linked to one or more first promoters;
- and
- b. a second polynucleotide comprising SEQ ID NO:
- 41, SEQ ID NO:
- 48, or nucleotides 1906-3303 of SEQ ID NO:
- 68;
- and wherein the second polynucleotide is operably linked to a second promoter.
5 more elements
A set of vectors comprising:
- a. a first vector comprising an expression cassette comprising a first polynucleotide encoding SEQ ID NO:
- 13, wherein the first polynucleotide is operably linked to one or more first promoters;
- and
- b. a second vector comprising a second polynucleotide encoding SEQ ID NO:
- 41, SEQ ID NO:
- 48, or nucleotides 1906-3303 of SEQ ID NO:
- 68 and wherein the second polynucleotide is operably linked to a second promoter.
4 more elements
A kit comprising:
- a. one or more antisense oligonucleotides comprising SEQ ID NO:
- 13;
- and
- b. a vector comprising a polynucleotide encoding SEQ ID NO:
- 41, SEQ ID NO:
- 48, or nucleotides 1906-3303 of SEQ ID NO:
- 68;
- and wherein the polynucleotide is operably linked to a promoter in the vector.
5 more elements
An isolated nucleic acid sequence comprising SEQ ID NO:
- 41, SEQ ID NO:
- 48, or nucleotides 1906-3303 of SEQ ID NO:
- 68.
Dependencies are inferred from the claim text.