Title:
GRNA TARGETING HSV ESSENTIAL GENES, CRISPR/CAS GENE EDITING SYSTEM, DELIVERY SYSTEM, AND USE
Document Type and Number:
WIPO Patent Application WO/2024/113302
Kind Code:
A1
Abstract:
Disclosed are gRNA targeting HSV essential genes, a CRISPR/Cas gene editing system, a delivery system, and use. Aiming at an HSV clinical strain genome sequence, the present invention designs and screens a gRNA series that directly targets the HSV essential genes VP16, ICP27, ICP4, or gD, and uses a viral vector or a non-viral vector to deliver nuclease Cas9 and gRNA targeting a viral gene. The HSV key gene-targeted gene editing can effectively inhibit HSV replication and spread of infection, and the gRNA combination cocktail strategy shows an effect superior to that of single-target editing. The method of the present invention and the gene therapy mediated by the viral vector can effectively eliminate HSV viruses with latent infections or recurrent infections, thus providing a potentially important means for the treatment of refractory HSV-1-related diseases such as viral encephalitis, herpes simplex keratitis, and infectious blindness.
Inventors:
WANG HUADONG (CN)
XU FUQIANG (CN)
YING MIN (CN)
LIU TONGTAN (CN)
SHI QING (CN)
XU FUQIANG (CN)
YING MIN (CN)
LIU TONGTAN (CN)
SHI QING (CN)
Application Number:
PCT/CN2022/135820
Publication Date:
June 06, 2024
Filing Date:
December 01, 2022
Export Citation:
Assignee:
SHENZHEN INST ADV TECH (CN)
International Classes:
A61K48/00; C12N15/113; A61P31/22; C12N15/63; C12N15/85; C12N15/90
Domestic Patent References:
| WO1992005284A1 | 1992-04-02 | |||
| WO2018017925A1 | 2018-01-25 | |||
| WO2022006745A1 | 2022-01-13 |
Foreign References:
| CN107580504A | 2018-01-12 | |||
| CN109468318A | 2019-03-15 | |||
| US20180251770A1 | 2018-09-06 |
Other References:
OH HYUNG SUK, NEUHAUSSER WERNER M, EGGAN PIERCE, ANGELOVA MAGDALENA, KIRCHNER RORY, EGGAN KEVIN C, KNIPE DAVID M: "Herpesviral lytic gene functions render the viral genome susceptible to novel editing by CRISPR/Cas9", ELIFE, ELIFE SCIENCES PUBLICATIONS LTD., GB, vol. 8, 2 December 2019 (2019-12-02), GB , XP093177737, ISSN: 2050-084X, DOI: 10.7554/eLife.51662
KHODADAD NASTARAN, FANI MONA, JAMEHDOR SALEH, NAHIDSAMIEI RAHIL, MAKVANDI MANOOCHEHR, KABOLI SAEED, TEIMOORI ALI, THEKKINIATH JOSE: "A knockdown of the herpes simplex virus type-1 gene in all-in-one CRISPR vectors", FOLIA HISTOCHEMICA ET CYTOBIOLOGICA, VESALIUS UNIVERSITY MEDICAL PUBLISHER, KRAKOW,, PL, vol. 58, no. 3, 1 January 2020 (2020-01-01), PL , pages 174 - 181, XP093177742, ISSN: 0239-8508, DOI: 10.5603/FHC.a2020.0020
KHODADAD NASTARAN, FANI MONA, JAMEHDOR SALEH, NAHIDSAMIEI RAHIL, MAKVANDI MANOOCHEHR, KABOLI SAEED, TEIMOORI ALI, THEKKINIATH JOSE: "A knockdown of the herpes simplex virus type-1 gene in all-in-one CRISPR vectors", FOLIA HISTOCHEMICA ET CYTOBIOLOGICA, VESALIUS UNIVERSITY MEDICAL PUBLISHER, KRAKOW,, PL, vol. 58, no. 3, 1 January 2020 (2020-01-01), PL , pages 174 - 181, XP093177742, ISSN: 0239-8508, DOI: 10.5603/FHC.a2020.0020
Attorney, Agent or Firm:
BEIJING CHENGHUI LAW FIRM (CN)
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