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US 2026/0308598 A1Published application
Gene therapy
OwnerChi Wai Wonginventor
Life of this patent
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Abstract
The present disclosure provides gene therapies for diseases and conditions including but not limited to Kennedy's disease. Such gene therapies include the design of nucleic acid molecules, siRNA, compositions, plasmids, viruses, as well as related methods and uses.
Claims20
A nucleic acid molecule comprising a promoter module, an expression module, and a regulatory module, wherein:
- the promoter module is configured to control transcription of the expression module and/or the regulatory module,
- the expression module includes a first gene of interest (GOI), which encodes a first target protein that is an exogenous protein to a cell,
- the regulatory module includes one or more nucleotide segments that are configured to regulate a second GOI and/or a second target protein encoded by the second GOI, wherein the second target protein is an endogenous protein of the cell,
- the first target protein at least partially and functionally substitutes the second target protein in the cell, or attenuates the second target protein in the cell, and
- the first GOI and the second GOI are not identical but have a sequence identity of at least 80%, 85%, 90%, 95%, or 98%.
2 more elements
A double stranded ribonucleic acid (dsRNA) agent for inhibiting expression of androgen receptor (AR), comprising a sense strand and an antisense strand, wherein
- the sense strand comprises at least 21 contiguous nucleotides differing by no more than 5 nucleotides from any one of SEQ ID NOs:
- 13, 15, 17, 19, 21, 23, 25, 27, and 29, and the antisense strand comprises a nucleotide sequence that is at least partially complementary to the sense strand.
Dependencies are inferred from the claim text.