Title:
PRODUCT FOR TREATING HEPATOCYTE ALB GENE-BASED DISEASE
Document Type and Number:
WIPO Patent Application WO/2021/083073
Kind Code:
A1
Abstract:
The present invention provides a product for treating a hepatocyte Alb gene-based disease, comprising a reagent capable of inserting a target gene into intron 13 of Alb in hepatocytes and co-expressing the target gene and Alb. The disease comprises hereditary liver metabolic diseases or diseases that can be treated with metabolic cross correction using the liver. The target gene comprises a gene that is mutated or deleted in the disease.
Inventors:
LI DALI (CN)
CHEN XI (CN)
NIU XURAN (CN)
XI ZAIXI (CN)
LIU MINGYAO (CN)
CHEN XI (CN)
NIU XURAN (CN)
XI ZAIXI (CN)
LIU MINGYAO (CN)
Application Number:
PCT/CN2020/123621
Publication Date:
May 06, 2021
Filing Date:
October 26, 2020
Export Citation:
Assignee:
UNIV EAST CHINA NORMAL (CN)
BIORAY LABORATORIES INC (CN)
BIORAY LABORATORIES INC (CN)
International Classes:
C12N15/113; A61K48/00; A61P7/04; C07K14/755; C12N9/22
Domestic Patent References:
| WO2019079527A1 | 2019-04-25 |
Foreign References:
| CN111492060A | 2020-08-04 | |||
| CN105940013A | 2016-09-14 |
Other References:
CHEN HAINAN, SHI MI, GILAM AVITAL, ZHENG QI, ZHANG YIN, AFRIKANOVA IVKA, LI JINLING, GLUZMAN ZOYA, JIANG RUHONG, KONG LING-JIE, CH: "Hemophilia A ameliorated in mice by CRISPR-based in vivo genome editing of human Factor VIII.", SCIENTIFIC REPORTS, vol. 9, no. 1, 14 November 2019 (2019-11-14), pages 1 - 15, XP055807516, ISSN: 2045-2322, DOI: 10.1038/s41598-019-53198-y
WANG QIHAN, HUAI CONG, SUN RUILIN, ZHUANG HUA, CHEN HONGYAN, FEI JIAN, LU DARU: "A Quick and Efficient Method to Generate Hemophilia B Mouse Models by the CRISPR/Cas System", HEREDITAS, vol. 37, no. 11, 30 November 2015 (2015-11-30), pages 1143 - 1148, XP055789498, ISSN: 0253-9772, DOI: 10.16288/j.yczz.15-117
WANG QIHAN, HUAI CONG, SUN RUILIN, ZHUANG HUA, CHEN HONGYAN, FEI JIAN, LU DARU: "A Quick and Efficient Method to Generate Hemophilia B Mouse Models by the CRISPR/Cas System", HEREDITAS, vol. 37, no. 11, 30 November 2015 (2015-11-30), pages 1143 - 1148, XP055789498, ISSN: 0253-9772, DOI: 10.16288/j.yczz.15-117
Attorney, Agent or Firm:
BEIJING JUNHUI INTELLECTUAL PROPERTY AGENT OFFICE (ORDINARY PARTNERSHIP) (CN)
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