Title:
GENE EDITING SYSTEMS AND METHODS FOR TREATING HEREDITARY ANGIOEDEMA
Document Type and Number:
WIPO Patent Application WO/2024/012435
Kind Code:
A1
Abstract:
The present disclosure generally relates to gene editing systems and methods for treating hereditary angioedema (HAE). Also disclosed are polynucleotides, vectors, cells, kits and compositions comprising the components of the gene editing systems, and methods related to treatment of HAE.
Inventors:
WANG LIJIE (CN)
HUANG LIGUO (CN)
WANG YICHUAN (CN)
LI PEIXUE (CN)
MOU XIAODUN (CN)
HUANG LIGUO (CN)
WANG YICHUAN (CN)
LI PEIXUE (CN)
MOU XIAODUN (CN)
Application Number:
PCT/CN2023/106730
Publication Date:
January 18, 2024
Filing Date:
July 11, 2023
Export Citation:
Assignee:
CORRECTSEQUENCE THERAPEUTICS CO LTD (CN)
International Classes:
C12N15/113
Domestic Patent References:
| WO2021158858A1 | 2021-08-12 | |||
| WO2020156575A1 | 2020-08-06 | |||
| WO2020156575A1 | 2020-08-06 |
Foreign References:
| CN2022104999W | 2022-07-11 | |||
| CN2023096482W |
Other References:
JOBERTY GÉRARD ET AL.: "A tandem guide RNA-based strategy for efficient CRISPR gene editing of cell populations with low heterogeneity of edited alleles", THE CRISPR JOURNAL, vol. 3, no. 2, 1 April 2020 (2020-04-01), pages 123 - 134, XP093093872, ISSN: 2573-1599, Retrieved from the Internet DOI: 10.1089/crispr.2019.0064
ALTSCHUL, S. F ET AL., J. MOL. BIOL, vol. 215, 1990, pages 403 - 410
BATZER ET AL., NUCLEIC ACID RES, vol. 19, 1991, pages 5081
OHTSUKA ET AL., J BIOL CHEM, vol. 260, 1985, pages 2605 - 2608
ROSSOLINI ET AL., MOL CELL PROBES, vol. 8, 1994, pages 91 - 98
WEIDMANN ET AL.: "The plasma contact system, a protease cascade at the nexus of inflammation. coagulation and immunity", MOLECULAR CELL RESEARCH, 2017
MATT ET AL.: "Hereditary angioedema: the plasma contact system out of control", J. THROMBOSIS AND HAEMOSTASIS, 2018
ZHOU ET AL., STEM CELLS, vol. 27, no. 11, 2009, pages 2667 - 74
HUANGFU ET AL., NATURE BIOTECHNOL, vol. 26, no. 7, 2008, pages 795
WOLTJEN ET AL., NATURE, vol. 458, no. 7239, 2009, pages 766 - 770
ZHOU ET AL., CELL STEM CELL, vol. 8, 2009, pages 381 - 384
SUMPIO ET AL.: "Cells in focus: endothelial cell,", INT. J. BIOCHEM CELL BIOL., 2002
WANG, LIJIE ET AL.: "Eliminating base-editor-induced genome-wide and transcriptome-wide off-target mutations", NATURE CELL BIOLOGY, vol. 23.5, 2021, pages 552 - 563, XP037452073, DOI: 10.1038/s41556-021-00671-4
ALTSCHUL, S. F ET AL., J. MOL. BIOL, vol. 215, 1990, pages 403 - 410
BATZER ET AL., NUCLEIC ACID RES, vol. 19, 1991, pages 5081
OHTSUKA ET AL., J BIOL CHEM, vol. 260, 1985, pages 2605 - 2608
ROSSOLINI ET AL., MOL CELL PROBES, vol. 8, 1994, pages 91 - 98
WEIDMANN ET AL.: "The plasma contact system, a protease cascade at the nexus of inflammation. coagulation and immunity", MOLECULAR CELL RESEARCH, 2017
MATT ET AL.: "Hereditary angioedema: the plasma contact system out of control", J. THROMBOSIS AND HAEMOSTASIS, 2018
ZHOU ET AL., STEM CELLS, vol. 27, no. 11, 2009, pages 2667 - 74
HUANGFU ET AL., NATURE BIOTECHNOL, vol. 26, no. 7, 2008, pages 795
WOLTJEN ET AL., NATURE, vol. 458, no. 7239, 2009, pages 766 - 770
ZHOU ET AL., CELL STEM CELL, vol. 8, 2009, pages 381 - 384
SUMPIO ET AL.: "Cells in focus: endothelial cell,", INT. J. BIOCHEM CELL BIOL., 2002
WANG, LIJIE ET AL.: "Eliminating base-editor-induced genome-wide and transcriptome-wide off-target mutations", NATURE CELL BIOLOGY, vol. 23.5, 2021, pages 552 - 563, XP037452073, DOI: 10.1038/s41556-021-00671-4
Attorney, Agent or Firm:
PEKSUNG INTELLECTUAL PROPERTY LTD. (CN)
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