发明公开
- 专利标题: SYNERGISTIC EFFECT OF SMN1 AND MIR-23A IN TREATING SPINAL MUSCULAR ATROPHY
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申请号: US18274496申请日: 2022-01-28
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公开(公告)号: US20240091383A1公开(公告)日: 2024-03-21
- 发明人: Dmitriy Aleksandrovich MADERA , Anna Sergeevna VESELOVA , Aleksei Sergeevich SIUTKIN , Pavel Mikhailovich GERSHOVICH , Dmitry Valentinovich MOROZOV
- 申请人: JOINT STOCK COMPANY «BIOCAD»
- 申请人地址: RU Saint Petersburg
- 专利权人: JOINT STOCK COMPANY «BIOCAD»
- 当前专利权人: JOINT STOCK COMPANY «BIOCAD»
- 当前专利权人地址: RU Saint Petersburg
- 优先权: RU 21102051 2021.01.29
- 国际申请: PCT/RU2022/000025 2022.01.28
- 进入国家日期: 2023-07-27
- 主分类号: A61K48/00
- IPC分类号: A61K48/00 ; C12N15/86
摘要:
The present application relates to the fields of biotechnology, virology, genetics, and molecular biology. More specifically, the present invention relates to an isolated nucleic acid for producing a gene therapy viral product, said isolated nucleic acid comprising a nucleic acid that encodes the SMN1 protein having the amino acid sequence of SEQ ID NO: 1, and a nucleic acid that encodes the microRNA miR-23a, an expression cassette and a vector based thereon, as well as an AAV9-based recombinant virus for expressing the SMN1 gene in target cells, a pharmaceutical composition that includes said recombinant virus, and various uses of the above recombinant virus and the above composition.
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