Title:
NUCLEIC ACID MOLECULE COMPRISING MICROMOLECULAR DRUG-BASED ALTERNATIVE SPLICING REGULATORY ELEMENT
Document Type and Number:
WIPO Patent Application WO/2024/060205
Kind Code:
A1
Abstract:
Disclosed are a nucleic acid molecule comprising an alternative splicing regulatory element and a target gene located at the 3' terminus of the alternative splicing regulatory element, a transcript of the nucleic acid molecule, a vector comprising the nucleic acid molecule or the transcript, a recombinant virus, a cell, a pharmaceutical composition, a method for using the same in regulating the expression level of the target gene, and use thereof in gene therapy.
Inventors:
GUO YUXUAN (CN)
CHEN ZHAN (CN)
CHEN ZHAN (CN)
Application Number:
PCT/CN2022/120884
Publication Date:
March 28, 2024
Filing Date:
September 23, 2022
Export Citation:
Assignee:
BEIJING BIOTUNE MEDICAL TECH CO LTD (CN)
UNIV BEIJING (CN)
UNIV BEIJING (CN)
International Classes:
C12N15/11; C12N5/10; C12N7/01; C12N15/63
Domestic Patent References:
| WO2020033473A1 | 2020-02-13 | |||
| WO2021163556A1 | 2021-08-19 |
Foreign References:
| CN114174514A | 2022-03-11 |
Other References:
CHENG, YUANMING; LUO, CHUNLING; WEI, NING; FENG, YING: "Research Progress in the Alternative Splicing Regulator SRSF2", CHEMISTRY OF LIFE : COMMUNICATIONS OF THE CHINESE BIOCHEMICAL SOCIETY, SHANGHAI : ZHONGGUO SHENGWU HUAXUEHUI, CN, vol. 36, no. 6, 15 December 2016 (2016-12-15), CN , pages 766 - 773, XP009553913, ISSN: 1000-1336, DOI: 10.13488/j.smhx.20160604
ALEX MAS MONTEYS ET AL.: "Regulated Control of Gene Therapies by Drug-induced Splicing", NATURE, vol. 596, no. 7871, 28 July 2021 (2021-07-28), XP093094186, ISSN: 0028-0836, DOI: 10.1038/s41586-021-03770-2
ALEX MAS MONTEYS ET AL.: "Regulated Control of Gene Therapies by Drug-induced Splicing", NATURE, vol. 596, no. 7871, 28 July 2021 (2021-07-28), XP093094186, ISSN: 0028-0836, DOI: 10.1038/s41586-021-03770-2
Attorney, Agent or Firm:
JUN HE LAW OFFICES (CN)
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