Title:
RNA MOLECULE, CHIMERIC NA MOLECULE, DOUBLE-STRANDED RNA MOLECULE, AND DOUBLE-STRANDED CHIMERIC NA MOLECULE
Document Type and Number:
WIPO Patent Application WO/2022/154123
Kind Code:
A1
Abstract:
The purpose of the present invention is to provide a novel RNA molecule. An RNA molecule, which is for an RNA interference method using a mutant allele having a point mutation as a target gene, wherein: (1) a base sequence complementary to a coding region of the mutant allele is included; and (2) counting from the most 5'-side base of the base sequence complementary to the mutant allele, (2-1) the 5th or 6th base is a mismatch with the base of the mutant allele, (2-2) the 10th or 11th position corresponds to the position of the point mutation and the 10th or 11th base corresponds to the base included in the mutant allele, (2-3) in the 8th ribonucleotide, the 2' position of a pentose is modified with OCH3, a halogen or LNA, and (2-4) in the 7th ribonucleotide, the 2' position of a pentose is not modified with OCH3, a halogen or LNA. In this RNA molecule, a ribonucleotide may be substituted with a deoxyribonucleotide, etc. The RNA molecule may form a double-stranded RNA together with a complementary strand.
Inventors:
UI-TEI KUMIKO (JP)
KOBAYASHI YOSHIAKI (JP)
SAIGO KAORU (JP)
NATORI YUKIKAZU (JP)
KOBAYASHI YOSHIAKI (JP)
SAIGO KAORU (JP)
NATORI YUKIKAZU (JP)
Application Number:
PCT/JP2022/001433
Publication Date:
July 21, 2022
Filing Date:
January 17, 2022
Export Citation:
Assignee:
UNIV TOKYO (JP)
International Classes:
C12N15/113; A61K31/7088; A61K31/7105; A61K31/712; A61K31/713; A61K48/00; A61P3/06; A61P9/14; A61P11/04; A61P11/06; A61P17/00; A61P17/04; A61P19/08; A61P21/00; A61P25/00; A61P25/08; A61P25/14; A61P25/28; A61P27/00; A61P27/02; A61P37/08; C12N15/10
Domestic Patent References:
| WO2021010449A1 | 2021-01-21 |
Foreign References:
| JP2019535288A | 2019-12-12 | |||
| JP2017502665A | 2017-01-26 | |||
| JP2019088196A | 2019-06-13 | |||
| US20130130377A1 | 2013-05-23 | |||
| US20070259827A1 | 2007-11-08 |
Other References:
YOSHIAKI KOBAYASHI, DAIKI FUKUHARA, DAI AKASE, MISAKO AIDA, KUMIKO HODO: "2P-0081 Identification of two functional domains in the seed region of the siRNA guide strand by 2-OMe modification", 43RD ANNUAL MEETING OF MOLECULAR BIOLOGY SOCIETY OF JAPAN; ONLINE; DECEMBER 2-4, 2020, vol. 43, 1 January 2020 (2020-01-01), JP, pages 1036, XP009538433
JACKSON, A.L. ET AL., NATURE BIOTECHNOLOGY, vol. 21, 2003, pages 635 - 637
UI-TEI, K. ET AL., NUCLEIC ACIDS RES., vol. 36, 2008, pages 7100 - 7109
JACKSON, A.L. ET AL., NATURE BIOTECHNOLOGY, vol. 21, 2003, pages 635 - 637
UI-TEI, K. ET AL., NUCLEIC ACIDS RES., vol. 36, 2008, pages 7100 - 7109
Attorney, Agent or Firm:
ISSHIKI & CO. (JP)
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