Title:
BASE EDITING SYSTEM AND METHOD FOR SPECIFICALLY REPAIRING HBB GENE MUTATIONS OF HUMANS, REAGENT KIT, AND APPLICATIONS THEREOF
Document Type and Number:
WIPO Patent Application WO/2018/227755
Kind Code:
A1
Abstract:
Provided are a method for specifically repairing, by using a base editing method, A>G pathogenic mutations causing β-thalassemia of humanms, a gRNA specifically targeting the mutations, and a base editing protein. By means of the method, HBB:c-79A>G and HBB:c-78A>G pathogenic mutations of humans can be precisely repaired. By importing the gene-editing system into human cells or individuals, the A>G pathogenic mutations can be precisely repaired, so as to cure the β-thalassemia disease.
Inventors:
HUANG JUNJIU (CN)
SONGYANG ZHOU (CN)
LIANG PUPING (CN)
SUN HONGWEI (CN)
ZHANG XIYA (CN)
SUN YING (CN)
SONGYANG ZHOU (CN)
LIANG PUPING (CN)
SUN HONGWEI (CN)
ZHANG XIYA (CN)
SUN YING (CN)
Application Number:
PCT/CN2017/098360
Publication Date:
December 20, 2018
Filing Date:
August 21, 2017
Export Citation:
Assignee:
UNIV SUN YAT SEN (CN)
International Classes:
C12N15/113; A61K48/00; A61P7/00; C12N5/10; C12N15/13; C12N15/90
Domestic Patent References:
WO2017070632A2 | 2017-04-27 | |||
WO2017059313A1 | 2017-04-06 | |||
WO2016154596A1 | 2016-09-29 |
Foreign References:
CN104805078A | 2015-07-29 |
Other References:
KOMOR. A.C.: "Programmable editing of a target base in genomic DNA", NATURE, vol. 533, no. 7603, 19 May 2016 (2016-05-19), pages 420 - 424, XP055551781, ISSN: 0028-0836, DOI: 10.1038/nature17946
KIM, D. ET AL.: "Genome-wide target specificities of CRISPR RNA-guided programmable deaminases.", NATURE BIOTECHNOLOGY, vol. 35, no. 5, 10 April 2017 (2017-04-10), pages 475 - 480, XP055383071, DOI: 10.1038/nbt.3852
GE, LUXING ET AL.: "The diversified development and application of CRISPR/Cas9 system (abstract)", JOURNAL OF AGRICULTURAL BIOTECHNOLOGY, vol. 25, no. 6, 9 June 2017 (2017-06-09), pages 939 - 953, XP055641721, ISSN: 1674-7968
KIM, D. ET AL.: "Genome-wide target specificities of CRISPR RNA-guided programmable deaminases.", NATURE BIOTECHNOLOGY, vol. 35, no. 5, 10 April 2017 (2017-04-10), pages 475 - 480, XP055383071, DOI: 10.1038/nbt.3852
GE, LUXING ET AL.: "The diversified development and application of CRISPR/Cas9 system (abstract)", JOURNAL OF AGRICULTURAL BIOTECHNOLOGY, vol. 25, no. 6, 9 June 2017 (2017-06-09), pages 939 - 953, XP055641721, ISSN: 1674-7968
Attorney, Agent or Firm:
GUANGZHOU NANFENG PATENT AGENCY CO. LTD. (CN)
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