Title:
GENE THERAPY FOR TREATING BETA-HEMOGLOBINOPATHIES
Document Type and Number:
WIPO Patent Application WO/2022/206986
Kind Code:
A1
Abstract:
Provided are gene therapy technologies, including specifically designed and tested guide RNA sequences for improved base editors, useful for increasing the expression of the gamma-globin gene. The guide RNA sequences may target the BCL11A erythroid enhancer or the gamma-globin promoter, or both at the same time. The base editors can include nucleobase deaminase inhibitor that inhibits the editing activity of the base editors until they are bound to the target sites. These gene therapy technologies are useful for treating diseases including beta-thalassemia and sickle cell anemia, among others.
Inventors:
CHEN JIA (CN)
YANG BEI (CN)
YANG LI (CN)
HAN WENYAN (CN)
SUN SHANGWU (CN)
ZHANG YING (CN)
YANG BEI (CN)
YANG LI (CN)
HAN WENYAN (CN)
SUN SHANGWU (CN)
ZHANG YING (CN)
Application Number:
PCT/CN2022/084982
Publication Date:
October 06, 2022
Filing Date:
April 02, 2022
Export Citation:
Assignee:
UNIV SHANGHAI TECH (CN)
International Classes:
C12N15/90; A61K35/28; A61P7/06; C12N9/22; C12N15/85
Domestic Patent References:
| WO2020156575A1 | 2020-08-06 | |||
| WO2019080917A1 | 2019-05-02 | |||
| WO2019217942A1 | 2019-11-14 | |||
| WO2020221291A1 | 2020-11-05 |
Foreign References:
| CN110029096A | 2019-07-19 | |||
| CN111757937A | 2020-10-09 | |||
| CN109715198A | 2019-05-03 |
Other References:
NICOLE M. GAUDELLI, ALEXIS C. KOMOR, HOLLY A. REES, MICHAEL S. PACKER, AHMED H. BADRAN, DAVID I. BRYSON & DAVID R. LIU: "Programmable base editing of A.T to G.C in genomic DNA without DNA cleavage (Includes Methods)", NATURE, NATURE PUBLISHING GROUP UK, LONDON, vol. 551, no. 7681, 23 November 2017 (2017-11-23), London, pages 464 - 471+16PP, XP002785203, ISSN: 0028-0836, DOI: 10.1038/nature24644
Attorney, Agent or Firm:
SHANGHAI BESHINING LAW OFFICE (CN)
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