Title:
AAV GENE THERAPY FOR TREATING A NERVOUS SYSTEM DISEASE
Document Type and Number:
WIPO Patent Application WO/2024/002056
Kind Code:
A1
Abstract:
Provided is a recombinant adeno-associated virus (rAAV) expression vector, comprising a gene encoding a TRIM72 protein or its variant or functional fragment thereof, wherein said recombinant AAV expression vector comprises a neuron-specific promoter. Further provided is a pharmaceutical composition comprising the rAAV expression vector and use thereof.
Inventors:
JIA YICHANG (CN)
GUO WEI (CN)
PENG LIN (CN)
GUO WEI (CN)
PENG LIN (CN)
Application Number:
PCT/CN2023/102706
Publication Date:
January 04, 2024
Filing Date:
June 27, 2023
Export Citation:
Assignee:
SINEUGENE THERAPEUTICS CO LTD (CN)
International Classes:
C12N7/01; A61K48/00; A61P9/10; C12N15/12
Foreign References:
| CN104877971A | 2015-09-02 | |||
| US20190153406A1 | 2019-05-23 | |||
| CN113292658A | 2021-08-24 |
Other References:
RADHIYANTI, P.T. ET AL.: "Comparative study of neuron-specific promoters in mouse brain transduced by intravenously administered AAV-PHP.eB", NEUROSCIENCE LETTERS, vol. 756, 11 May 2021 (2021-05-11), XP086621185, DOI: 10.1016/j.neulet.2021.135956
PALEO, B.J. ET AL.: "Enhancing membrane repair increases regeneration in a sciatic injury model", PLOS ONE, vol. 15, no. 4, 9 April 2020 (2020-04-09), XP093119214, DOI: 10.1371/journal.pone.0231194
NIEUWENHUIS BART; HAENZI BARBARA; HILTON SAM; CARNICER-LOMBARTE ALEJANDRO; HOBO BARBARA; VERHAAGEN JOOST; FAWCETT JAMES W.: "Optimization of adeno-associated viral vector-mediated transduction of the corticospinal tract: comparison of four promoters", GENE THERAPY, NATURE PUBLISHING GROUP, LONDON, GB, vol. 28, no. 1-2, 1 January 1900 (1900-01-01), GB , pages 56 - 74, XP037380036, ISSN: 0969-7128, DOI: 10.1038/s41434-020-0169-1
WAN, CHANJUAN ; YUJUAN, WANG: "Advances in research on TRIM72 protein", SHENGWU-JISHU = BIOTECHNOLOGY, HEILONGJIANG-SHENG WEISHENGWU XUEHUI; HEILONGJIANG-SHENG SHENGWU GONGCHENG XUEHUI; HEILONGJIANG-SHENG KEXUEYUAN, YINGYONG WEISHENGWU YANJIUSUO, CN, vol. 25, no. 1, 31 December 2015 (2015-12-31), CN , pages 93 - 97, XP009552229, ISSN: 1004-311X, DOI: 10.16519/j.cnki.1004-311x.2015.01.019
PALEO, B.J. ET AL.: "Enhancing membrane repair increases regeneration in a sciatic injury model", PLOS ONE, vol. 15, no. 4, 9 April 2020 (2020-04-09), XP093119214, DOI: 10.1371/journal.pone.0231194
NIEUWENHUIS BART; HAENZI BARBARA; HILTON SAM; CARNICER-LOMBARTE ALEJANDRO; HOBO BARBARA; VERHAAGEN JOOST; FAWCETT JAMES W.: "Optimization of adeno-associated viral vector-mediated transduction of the corticospinal tract: comparison of four promoters", GENE THERAPY, NATURE PUBLISHING GROUP, LONDON, GB, vol. 28, no. 1-2, 1 January 1900 (1900-01-01), GB , pages 56 - 74, XP037380036, ISSN: 0969-7128, DOI: 10.1038/s41434-020-0169-1
WAN, CHANJUAN ; YUJUAN, WANG: "Advances in research on TRIM72 protein", SHENGWU-JISHU = BIOTECHNOLOGY, HEILONGJIANG-SHENG WEISHENGWU XUEHUI; HEILONGJIANG-SHENG SHENGWU GONGCHENG XUEHUI; HEILONGJIANG-SHENG KEXUEYUAN, YINGYONG WEISHENGWU YANJIUSUO, CN, vol. 25, no. 1, 31 December 2015 (2015-12-31), CN , pages 93 - 97, XP009552229, ISSN: 1004-311X, DOI: 10.16519/j.cnki.1004-311x.2015.01.019
Attorney, Agent or Firm:
SHANGHAI DIANSHI PARTNERS , P.C. et al. (CN)
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