Title:
SYSTEM FOR HIGHLY EFFICIENT MODIFICATION OF TARGET SEQUENCES
Document Type and Number:
WIPO Patent Application WO/2020/022476
Kind Code:
A1
Abstract:
Provided is a technology for more efficient genetic modification, including target gene disruption, using a guide RNA-dependent nuclease. The present invention also addresses the problem of different cleavage efficiencies for each target sequence. Specifically, by the present invention, a new system was developed in which, when developing a target cleavage system that utilizes a guide RNA-dependent nuclease, only the nuclease gene is integrated into a negative-strand RNA viral vector, which has the properties of high infection efficiency and high transgene expression, and, after the viral vector has been introduced into cells in advance and the nuclease has been caused to be expressed, guide RNA is separately supplied to the cells. As a result, an increase in cleavage efficiency and a reduction in recleavage were both successfully achieved, and the efficiency of target gene modification was improved. The present invention is useful as an efficient genetic modification technology, and also has the feature of making it possible to adjust the enhancement and attenuation of genetic modification efficiency by adjusting cleavage efficiency through adjustment of the amount of guide RNA that is introduced.
Inventors:
KUSANO KOHJI (JP)
KITOGO TAKAYUKI (JP)
SHU TSUGUMINE (JP)
MORI TOYOTAKA (JP)
KITOGO TAKAYUKI (JP)
SHU TSUGUMINE (JP)
MORI TOYOTAKA (JP)
Application Number:
PCT/JP2019/029381
Publication Date:
January 30, 2020
Filing Date:
July 26, 2019
Export Citation:
Assignee:
ID PHARMA CO LTD (JP)
International Classes:
C12N15/09; C12N15/86
Domestic Patent References:
| WO2017223330A1 | 2017-12-28 | |||
| WO2010008054A1 | 2010-01-21 |
Other References:
PARK, A. ET AL.: "Highly Efficient Sendai Virus Mediated CRISPR/CAS9 Gene Edi ting in Hematopoietic Stem Cells", MOLECULAR THERAPY, vol. 25, no. 5, 2017, pages 136 - 137 , 290
Attorney, Agent or Firm:
HARUNA, Masao et al. (JP)
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