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Title:
SIRNA FOR INHIBITING HTT GENE EXPRESSION, AND PRECURSOR AND APPLICATION THEREOF
Document Type and Number:
WIPO Patent Application WO/2021/115141
Kind Code:
A1
Abstract:
Provided are an siRNA for inhibiting HTT gene expression and a precursor sequence thereof, an expression vector comprising the siRNA or the precursor sequence thereof, an application thereof in preparation of a drug for treating Huntington's disease, and a pharmaceutical preparation and pharmaceutical composition containing the siRNA, the precursor sequence or the expression vector. The siRNA is selected from the following: SEQ ID NO:1, SEQ ID NO:2, SEQ ID NO:3, SEQ ID NO:4, SEQ ID NO.:5, or a combination thereof.

Inventors:
CHEN XI (CN)
ZHANG LI (CN)
PEI ZHONG (CN)
WU TENGTENG (CN)
LIU JIN (CN)
YU MENGCHAO (CN)
XIA YUGUI (CN)
TU SHENG (CN)
LIN QIAOFANG (CN)
Application Number:
PCT/CN2020/132256
Publication Date:
June 17, 2021
Filing Date:
November 27, 2020
Export Citation:
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Assignee:
NANJING YOUZHIYUAN PHARMACEUTICAL TECH CO LTD (CN)
International Classes:
C12N15/113; A61K31/7105; A61K31/713; A61P25/14; C12N15/63
Domestic Patent References:
WO2018204803A12018-11-08
WO2019060726A12019-03-28
Foreign References:
US10093927B22018-10-09
CN101052717A2007-10-10
CN109831916A2019-05-31
CN109971756A2019-07-05
Other References:
SAMBROOK ET AL.: "Molecular Cloning: Laboratory Manual", 1989, COLD SPRING HARBOR LABORATORY PRESS
See also references of EP 4074826A4
Attorney, Agent or Firm:
XU & PARTNERS, LLC. (CN)
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