Title:
MULTIPLEX CRISPR/CAS9-MEDIATED TARGET GENE ACTIVATION SYSTEM
Document Type and Number:
WIPO Patent Application WO/2022/232442
Kind Code:
A3
Abstract:
Provided herein are multiplex crRNAs and multiplex sgRNAs, as well as RNA molecules thereof. Also provided are compositions and kits including the multiplex crRNAs and sgRNAs, which can be used in a multiplex targeted gene activation (mTGA) system. Also provided are methods that include administering a therapeutically effective amount of the mTGA system to a subject. In some examples, the method treats a disease associated with reduced or no expression of a gene, such as type I diabetes, Duchenne muscular dystrophy, a liver disease, or acute kidney disease.
Inventors:
IZPISUA BELMONTE JUAN CARLOS (US)
WANG CHAO (US)
LIAO HSIN-KAI (US)
REDDY PRADEEP (US)
WANG CHAO (US)
LIAO HSIN-KAI (US)
REDDY PRADEEP (US)
Application Number:
PCT/US2022/026805
Publication Date:
December 15, 2022
Filing Date:
April 28, 2022
Export Citation:
Assignee:
SALK INST FOR BIOLOGICAL STUDI (US)
IZPISUA BELMONTE JUAN CARLOS (US)
WANG CHAO (US)
LIAO HSIN KAI (US)
REDDY PRADEEP (US)
IZPISUA BELMONTE JUAN CARLOS (US)
WANG CHAO (US)
LIAO HSIN KAI (US)
REDDY PRADEEP (US)
International Classes:
C12N1/20; C12N15/87; C12N9/12
Domestic Patent References:
| WO2019236081A1 | 2019-12-12 |
Foreign References:
| US20210079394A1 | 2021-03-18 |
Other References:
JOHNSON, J.A. BRAGG, J.N. LAWRENCE, D.M. JACKSON, A.O.: "Sequence elements controlling expression of Barley stripe mosaic virus subgenomic RNAs in vivo", VIROLOGY, ELSEVIER, AMSTERDAM, NL, vol. 313, no. 1, 15 August 2003 (2003-08-15), AMSTERDAM, NL , pages 66 - 80, XP004452391, ISSN: 0042-6822, DOI: 10.1016/S0042-6822(03)00285-X
CALVIN KATE: "Biochemical Characterization of the RNA Splicing Endonuclease", THESIS, 3 April 2007 (2007-04-03), XP093019377, [retrieved on 20230131]
SAHRA-TAYLOR MULLINEUX; DENIS L.J. LAFONTAINE;: "Mapping the cleavage sites on mammalian pre-rRNAs: Where do we stand?", BIOCHIMIE, MASSON, PARIS, FR, vol. 94, no. 7, 1 February 2012 (2012-02-01), FR , pages 1521 - 1532, XP028510189, ISSN: 0300-9084, DOI: 10.1016/j.biochi.2012.02.001
HUMAR B, ET AL.: "NOVEL GERMLINE CDH1 MUTATIONS IN HEREDITARY DIFFUSE GASTRIC CANCER FAMILIES", HUMAN MUTATION, JOHN WILEY & SONS, INC., US, vol. 19, no. 05, 1 January 2002 (2002-01-01), US , pages 518 - 525, XP009017719, ISSN: 1059-7794, DOI: 10.1002/humu.10067
CALVIN KATE: "Biochemical Characterization of the RNA Splicing Endonuclease", THESIS, 3 April 2007 (2007-04-03), XP093019377, [retrieved on 20230131]
SAHRA-TAYLOR MULLINEUX; DENIS L.J. LAFONTAINE;: "Mapping the cleavage sites on mammalian pre-rRNAs: Where do we stand?", BIOCHIMIE, MASSON, PARIS, FR, vol. 94, no. 7, 1 February 2012 (2012-02-01), FR , pages 1521 - 1532, XP028510189, ISSN: 0300-9084, DOI: 10.1016/j.biochi.2012.02.001
HUMAR B, ET AL.: "NOVEL GERMLINE CDH1 MUTATIONS IN HEREDITARY DIFFUSE GASTRIC CANCER FAMILIES", HUMAN MUTATION, JOHN WILEY & SONS, INC., US, vol. 19, no. 05, 1 January 2002 (2002-01-01), US , pages 518 - 525, XP009017719, ISSN: 1059-7794, DOI: 10.1002/humu.10067
Attorney, Agent or Firm:
THIREAULT, Caitlin A. et al. (US)
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