Title:
PROTEIN MUTANT AND USE THEREOF IN TREATMENT OF DISEASE RELATED TO HBB GENE MUTATION
Document Type and Number:
WIPO Patent Application WO/2024/098383
Kind Code:
A1
Abstract:
Disclosed are a protein mutant with a sequence having more than 80% identity to SEQ ID NO. 1, a gRNA used in combination with the protein mutant, a gene editing system, an expression vector, a modified cell, a pharmaceutical composition, and use thereof in the treatment of thalassemia or sickle cell anemia related to an HBB gene mutation. The protein mutant has a substitution at one or more of positions 549, 555, 559, and 606 of SEQ ID NO. 1.
Inventors:
DAI XUECHEN (CN)
WANG DAN (CN)
JI ZEYANG (CN)
LI ANDUO (CN)
SHEN XUECHUN (CN)
JIANG YUAN (CN)
ZHANG WENWEI (CN)
WANG DAN (CN)
JI ZEYANG (CN)
LI ANDUO (CN)
SHEN XUECHUN (CN)
JIANG YUAN (CN)
ZHANG WENWEI (CN)
Application Number:
PCT/CN2022/131403
Publication Date:
May 16, 2024
Filing Date:
November 11, 2022
Export Citation:
Assignee:
BGI SHENZHEN (CN)
International Classes:
C12N9/22; A61K35/28; A61K38/46; A61P7/06; C07K19/00; C12N5/10; C12N15/113; C12N15/55; C12N15/90
Attorney, Agent or Firm:
FASHENG INTELLECTUAL PROPERTY COMPANY, LTD. (CN)
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