Title:
DEVELOPMENT OF DUAL-gRNA APPROACH WITH UNDETECTABLE OFF-TARGET EFFECT TO CORRECT C9ORF72 REPEAT EXPANSION AND C9ORF72 PATHOLOGY
Document Type and Number:
WIPO Patent Application WO/2021/127886
Kind Code:
A1
Abstract:
A group of gRNAs is provided, and the group of gRNAs comprises a first gRNA molecule; and a second gRNA molecule capable of defining a region in a genome sequence with the first gRNA molecule, wherein the region in the genome comprises a target sequence in need of removal.
Inventors:
PIAO XUEJIAO (CN)
JIA YICHANG (CN)
JIA YICHANG (CN)
Application Number:
PCT/CN2019/127553
Publication Date:
July 01, 2021
Filing Date:
December 23, 2019
Export Citation:
Assignee:
UNIV TSINGHUA (CN)
International Classes:
C12N15/113; A61K48/00; C07H21/04
Domestic Patent References:
| WO2018064600A1 | 2018-04-05 | |||
| WO2017040813A2 | 2017-03-09 | |||
| WO2017109757A1 | 2017-06-29 | |||
| WO2016167780A1 | 2016-10-20 |
Other References:
SELVARAJ BHUVANEISH T., LIVESEY MATTHEW R., ZHAO CHEN, GREGORY JENNA M., JAMES OWAIN T., CLEARY ELAINE M., CHOUHAN AMIT K., GANE A: "C9ORF72 repeat expansion causes vulnerability of motor neurons to Ca2+-permeable AMPA receptor-mediated excitotoxicity", NATURE COMMUNICATIONS, vol. 9, no. 1, 1 December 2018 (2018-12-01), XP055824852, DOI: 10.1038/s41467-017-02729-0
MOCHTAR PRIBADI, YANG ZHONGAN, KIM TANYA S, SWARTZ ELLIOT W, HUANG ALDEN Y, CHEN JASON A, DOKURU DEEPIKA, BAEK JAEYUN, GAO FUYING,: "CRISPR-Cas9 targeted deletion of the C9orf72 repeat expansion mutation corrects cellular phenotypes in patient-derived iPS cells", BIORXIV, 1 May 2016 (2016-05-01), XP055429422, Retrieved from the Internet [retrieved on 20171128], DOI: 10.1101/051193
ZHANG YU, ZHANG GUO-YONG, LIN ZHEN-GUO: "C9ORF72 Mutation in FTD-ALSl Genetics Pathogenesis Clinical Feature and Therapy ZHANG", ZHONGGUO LINCHUANG SHENJING KEXUE = CHINESE JOURNAL CLINICAL NEUROSCIENCES., FUDAN DAXUE SHENJINGBINGXUE YANJIUSUO, CN, vol. 27, no. 5, 1 January 2019 (2019-01-01), CN, pages 559 - 564, XP055825333, ISSN: 1008-0678
MOCHTAR PRIBADI, YANG ZHONGAN, KIM TANYA S, SWARTZ ELLIOT W, HUANG ALDEN Y, CHEN JASON A, DOKURU DEEPIKA, BAEK JAEYUN, GAO FUYING,: "CRISPR-Cas9 targeted deletion of the C9orf72 repeat expansion mutation corrects cellular phenotypes in patient-derived iPS cells", BIORXIV, 1 May 2016 (2016-05-01), XP055429422, Retrieved from the Internet
ZHANG YU, ZHANG GUO-YONG, LIN ZHEN-GUO: "C9ORF72 Mutation in FTD-ALSl Genetics Pathogenesis Clinical Feature and Therapy ZHANG", ZHONGGUO LINCHUANG SHENJING KEXUE = CHINESE JOURNAL CLINICAL NEUROSCIENCES., FUDAN DAXUE SHENJINGBINGXUE YANJIUSUO, CN, vol. 27, no. 5, 1 January 2019 (2019-01-01), CN, pages 559 - 564, XP055825333, ISSN: 1008-0678
Attorney, Agent or Firm:
TSINGYIHUA INTELLECTUAL PROPERTY LLC (CN)
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