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Title:
SMALL INTERFERING RNA FOR TREATING NEOVASCULAR RETINAL DISEASES AND DNA TETRAHEDRAL COMPLEX THEREOF
Document Type and Number:
WIPO Patent Application WO/2024/045251
Kind Code:
A1
Abstract:
A small interfering RNA and use thereof. The small interfering RNA specifically targets a Runt-related transcription factor 1 gene and a homotypic mRNA thereof and, by means of silencing the expression of a complementary gene thereof, the expression level of RUNX1 is down-regulated. The siRNA is used for preparing a drug for treating neovascular ophthalmic diseases and has very good application prospects. The siRNA and TDNs are designed into TDNs siRNA nucleic acid molecules with a spatial tetrahedral structure, improving stability, helping the siRNA enter cells efficiently, and improving the degradation efficacy of the siRNA on RUNX1 mRNA, thereby reducing the expression of RUNX1, and preventing the generation of new blood vessels.

Inventors:
LUO DELUN (CN)
XU XIAOXIAO (CN)
WU DAYING (CN)
Application Number:
PCT/CN2022/121379
Publication Date:
March 07, 2024
Filing Date:
September 26, 2022
Export Citation:
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Assignee:
CHENGDU GENREZE GENE TECH CO LTD (CN)
International Classes:
C12N15/113; A61K31/713
Foreign References:
US20190350961A12019-11-21
CN113286811A2021-08-20
CN114404608A2022-04-29
Other References:
LAM, J. D. ET AL.: "Identification of RUNX1 as a mediator of aberrant retinal angiogenesis", DIABETES, vol. 66, 31 July 2017 (2017-07-31), XP055611471, DOI: 10.2337/db16-1035
XIU HAN, WU ZHENGHONG: "Advances in self-assemblied DNA nanocages as drug delivery systems", JOURNAL OF CHINA PHARMACEUTICAL UNIVERSITY, vol. 48, no. 6, 25 December 2017 (2017-12-25), pages 663 - 669, XP093143938
RIDDELL ALEXANDRA, MCBRIDE MARTIN, BRAUN THOMAS, NICKLIN STUART A, CAMERON EWAN, LOUGHREY CHRISTOPHER M, MARTIN TAMARA P: "RUNX1: an emerging therapeutic target for cardiovascular disease", CARDIOVASCULAR RESEARCH, OXFORD UNIVERSITY PRESS, GB, vol. 116, no. 8, 1 July 2020 (2020-07-01), GB , pages 1410 - 1423, XP093143933, ISSN: 0008-6363, DOI: 10.1093/cvr/cvaa034
ZHAO DAN, CUI WEITONG, LIU MENGTING, LI JIAJIE, SUN YUE, SHI SIRONG, LIN SHIYU, LIN YUNFENG: "Tetrahedral Framework Nucleic Acid Promotes the Treatment of Bisphosphonate-Related Osteonecrosis of the Jaws by Promoting Angiogenesis and M2 Polarization", APPLIED MATERIALS & INTERFACES, AMERICAN CHEMICAL SOCIETY, US, vol. 12, no. 40, 7 October 2020 (2020-10-07), US , pages 44508 - 44522, XP093143934, ISSN: 1944-8244, DOI: 10.1021/acsami.0c13839
Attorney, Agent or Firm:
LECOME INTELLECTUAL PROPERTY AGENT LTD. (CN)
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