Title:
人工単一ガイドＲＮＡ及びその用途
Document Type and Number:
Japanese Patent JP6800171
Kind Code:
B2
Abstract:
The present invention provides a novel artificial sgRNA having an activity equal to or higher than that of a natural form sgRNA and improved in stability in vivo, and an efficient CRISPR/Cas9 system by combining the artificial sgRNA and Cas9. Even when a nucleotide linker region for forming a single strand by linking the 3'-terminal of crRNA and the 5'-terminal of tracrRNA in sgRNA is substituted with an amino acid derivative linker, an activity equal to or higher than that of the original sgRNA is retained in an in vitro DNA cleavage assay. Furthermore, even when the linker region existing between stem-loop 1 and stem-loop 2 of tracrRNA and/or the loop portion of stem-loop 2 are/is substituted with an amino acid derivative linker, or even when an amino acid derivative linker is added/inserted into the vicinity of the 5'-terminal and/or the 3'-terminal of sgRNA, an activity equal to or higher than that of the original sgRNA is retained. The stability in vivo can be improved by introducing one or more amino acid derivative linkers into sgRNA.
Inventors:
Eriko Aoki
Tadaaki Oki
Takashi Kinoshita
Tadaaki Oki
Takashi Kinoshita
Application Number:
JP2017563895A
Publication Date:
December 16, 2020
Filing Date:
January 30, 2017
Export Citation:
Assignee:
Bonac Co., Ltd.
International Classes:
A61K31/7088; A61K47/65; A61K48/00; C07C237/22; C07F9/24; C12N15/09; C12N15/11
Foreign References:
| WO2015099187A1 | ||||
| WO2015006747A2 | ||||
| WO2015093495A1 |
Other References:
Molecular Cell,2014年10月,Vol. 56, No. 2,pp. 333-339
Benzenepropanamide, N-hydroxy-alpha-[(2-hydroxyacetyl)amino]-,REGISTRY (STN),2009年 2月 9日,REGISTRY No. 1103513-06-9,STN on the Web [retrieved on 2017.04.17]
Benzenepropanamide, N-hydroxy-alpha-[(2-hydroxyacetyl)amino]-,REGISTRY (STN),2009年 2月 9日,REGISTRY No. 1103513-06-9,STN on the Web [retrieved on 2017.04.17]
Attorney, Agent or Firm:
Takashima Ichi
Kyoko Doi
Mitsunori Kamata
Yaeko Tamura
Hirofumi Toma
Atsuko Akai
Tomiya Tozaki
Koichiro Muraoka
Kyoko Doi
Mitsunori Kamata
Yaeko Tamura
Hirofumi Toma
Atsuko Akai
Tomiya Tozaki
Koichiro Muraoka