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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)
Application Number:
PCT/CN2017/098360
Publication Date:
December 20, 2018
Filing Date:
August 21, 2017
Export Citation:
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Assignee:
UNIV SUN YAT SEN (CN)
International Classes:
C12N15/113; A61K48/00; A61P7/00; C12N5/10; C12N15/13; C12N15/90
Domestic Patent References:
WO2017070632A22017-04-27
WO2017059313A12017-04-06
WO2016154596A12016-09-29
Foreign References:
CN104805078A2015-07-29
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
Attorney, Agent or Firm:
GUANGZHOU NANFENG PATENT AGENCY CO. LTD. (CN)
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