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Title:
GRNA MOLECULE TARGETING INTRON I OR INTRON II OF HBB GENE, SYNTHETIC METHOD THEREOF, AND METHOD TO CORRECT TYPES OF HBB GENE MUTATIONS
Document Type and Number:
WIPO Patent Application WO/2022/147759
Kind Code:
A1
Abstract:
A gRNA molecule targeting Intron I or Intron II of β-globin gene, a synthetic method thereof, a method for constructing a repair system targeting β-globin gene mutation sites, a repair system, and a universal method to correct types of β-globin gene mutations in β-thalassemia are provided. Especially, the universal method can correct most types of HBB gene mutations in HSPCs, and the targeted HSPCs have normal function in vitro and no significant off-target effect.

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Inventors:
ZENG SHENG (CN)
CHEN XINPING (CN)
YANG ZONGFAN (CN)
FENG MEIMEI (CN)
WANG YE (CN)
Application Number:
PCT/CN2021/070839
Publication Date:
July 14, 2022
Filing Date:
January 08, 2021
Export Citation:
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Assignee:
SUSHENG BIOTECH HAINAN CO LTD (CN)
International Classes:
C12N15/113; A61K35/28; A61P7/06; C12N5/10; C12N15/85
Domestic Patent References:
WO2020253753A12020-12-24
WO2019079347A12019-04-25
Foreign References:
US20200377857A12020-12-03
CN107630018A2018-01-26
US20180030438A12018-02-01
US20200181651A12020-06-11
Attorney, Agent or Firm:
GUANGZHOU BANGZHUAN GAOZHI INTELLECTUAL PROPERTY AGENCY (SPECIAL GENERAL PARTNERSHIP) (CN)
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