Title:
GENE EDITING COMPOSITION OR KIT USED FOR IN VIVO GENE THERAPY
Document Type and Number:
WIPO Patent Application WO/2020/063178
Kind Code:
A1
Abstract:
A gene editing composition or kit used for in vivo gene therapy, comprising: 1) sgRNA that is linked to a nucleic acid molecule of a binding protein and that targets a target mutant gene, or a coding sequence thereof, 2) a template nucleic acid that repairs the target mutant gene, or a coding sequence thereof, 3) an sgRNA-guided nuclease, or a coding sequence thereof, 4) a homologous recombination-promoting protein that is fused with the nucleic acid binding protein, or a coding sequence thereof, wherein the nucleic acid binding protein is capable of binding to the nucleic acid molecule in 1) above. The sgRNA, composition or kit may be used to treat diseases caused by genetic mutations, such as hereditary diseases. The sgRNA, composition or kit may effectively improve the efficiency of homologous recombination repair, and may also achieve homologous recombination repair in non-dividing cells.
Inventors:
XUE TIAN (CN)
Application Number:
PCT/CN2019/100809
Publication Date:
April 02, 2020
Filing Date:
August 15, 2019
Export Citation:
Assignee:
UNIV SCIENCE & TECHNOLOGY CHINA (CN)
International Classes:
C12N15/113; A61K31/7088; A61K48/00; A61P1/16; A61P3/00; A61P9/10; A61P13/12; A61P21/00; C12N9/22
Domestic Patent References:
| WO2017180711A1 | 2017-10-19 |
Foreign References:
| CN109266648A | 2019-01-25 | |||
| CN106282228A | 2017-01-04 |
Other References:
CAI, Y. ET AL.: "In Vivo Genome Editing Rescues Photoreceptor Degeneration via a Cas9/RecA- mediated Homology-directed Repair Pathway", SCIENCE ADVANCES, vol. 5, 17 April 2019 (2019-04-17), pages 1 - 12, XP055699625
WU, W.H. ET AL.: "CRISPR Repair Reveals Causative Mutation in a Preclinical Model of Retinitis Pigmentosa", MOLECULAR THERAPY, vol. 24, no. 8, 28 June 2016 (2016-06-28), pages 1388 - 1394, XP055699649, DOI: 10.1038/mt.2016.107
SHAO, S. ET AL.: "Enhancing CRISPR/Cas9-mediated Homology-directed Repair in Mammalian Cells by Expressing Saccharomyces Cerevisiae Rad52", INTERNATIONAL JOURNAL OF BIOCHEMISTRY AND CELL BIOLOGY, vol. 92, 18 September 2017 (2017-09-18), pages 43 - 52, XP055627157, DOI: 10.1016/j.biocel.2017.09.012
COLLONNIER, C. ET AL.: "CRISPR-Cas9-mediated Efficient Directed Mutagenesis and RAD51-dependent and RAD51-independent Gene Targeting in the Moss Physcomitrella Patens", PLANT BIOTECHNOLOGY JOURNAL, vol. 15, 31 December 2017 (2017-12-31), pages 122 - 131, XP055699651, DOI: 10.1111/pbi.12596
DAHLMAN, J. E. ET AL.: "Orthogonal Gene Knockout and Activation with a Catalytically Active Cas9 Nuclease", NATURE BIOTECHNOLOGY, vol. 33, no. 11, 5 October 2015 (2015-10-05), pages 1159 - 1163, XP055381172, DOI: 10.1038/nbt.3390
WU, W.H. ET AL.: "CRISPR Repair Reveals Causative Mutation in a Preclinical Model of Retinitis Pigmentosa", MOLECULAR THERAPY, vol. 24, no. 8, 28 June 2016 (2016-06-28), pages 1388 - 1394, XP055699649, DOI: 10.1038/mt.2016.107
SHAO, S. ET AL.: "Enhancing CRISPR/Cas9-mediated Homology-directed Repair in Mammalian Cells by Expressing Saccharomyces Cerevisiae Rad52", INTERNATIONAL JOURNAL OF BIOCHEMISTRY AND CELL BIOLOGY, vol. 92, 18 September 2017 (2017-09-18), pages 43 - 52, XP055627157, DOI: 10.1016/j.biocel.2017.09.012
COLLONNIER, C. ET AL.: "CRISPR-Cas9-mediated Efficient Directed Mutagenesis and RAD51-dependent and RAD51-independent Gene Targeting in the Moss Physcomitrella Patens", PLANT BIOTECHNOLOGY JOURNAL, vol. 15, 31 December 2017 (2017-12-31), pages 122 - 131, XP055699651, DOI: 10.1111/pbi.12596
DAHLMAN, J. E. ET AL.: "Orthogonal Gene Knockout and Activation with a Catalytically Active Cas9 Nuclease", NATURE BIOTECHNOLOGY, vol. 33, no. 11, 5 October 2015 (2015-10-05), pages 1159 - 1163, XP055381172, DOI: 10.1038/nbt.3390
Attorney, Agent or Firm:
CHINA SCIENCE PATENT & TRADEMARK AGENT LTD. (CN)
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