RNA MOLECULE, CHIMERIC NA MOLECULE, DOUBLE-STRANDED RNA MOLECULE, AND DOUBLE-STRANDED CHIMERIC NA MOLECULE

WIPO Patent Application WO/2022/154122

A1

The purpose of the present invention is to provide a novel RNA molecule. An RNA molecule for RNA interference for which the target gene is a mutant allele that has a point mutation. The RNA molecule (1) has a base sequence that is complementary to a coding region of the mutant allele, and (2) counting from the base that is furthest to the 5' side of the base sequence that is complementary to the mutant allele, (2-1) the 5th or 6th base is mismatched with the base of the mutant allele, (2-2) the 10th or 11th base corresponds to the location of the point mutation, and (2-3) at the 6th–8th bases or the 7th and 8th bases, the pentose is modified with OCH3 or the like at the 2' position. The ribonucleotides of the RNA molecule may be replaced with deoxyribonucleotides or the like, and the RNA molecule may form a double-stranded RNA with a complementary strand.

UI-TEI KUMIKO (JP)
KOBAYASHI YOSHIAKI (JP)
SATO ATSUSHI (JP)
ASANO YOSHIMASA (JP)
SUZUKI YURIA (JP)
LEDEY NAOMI (JP)
SAIGO KAORU (JP)
NATORI YUKIKAZU (JP)

PCT/JP2022/001431

July 21, 2022

January 17, 2022

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UNIV TOKYO (JP)

C12N15/113; A61K31/7088; A61K31/7105; A61K31/7115; 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

JP2021005335A

2021-01-14

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HODO KUMIKO: "Against the proto-oncogene KRAS siRNA development for tailor-made therapy", 1 January 2018 (2018-01-01), XP055951831
YOSHIAKI KOBAYASHI, ET AL.: "2P-0702 Establishment of effective and specific siRNA design method for all mammalian genes", 41ST ANNUAL MEETING OF THE MOLECULAR BIOLOGY SOCIETY OF JAPAN; NOV. 28 - 30, 2018, MOLECULAR BIOLOGY SOCIETY OF JAPAN, JP, vol. 41, 9 November 2018 (2018-11-09) - 30 November 2018 (2018-11-30), JP, pages 2P - 0702, XP009538603
ATSUSHI SATO, ET AL.: "3P-0344 Development of siRNA (SNPD-siRNA) that suppresses single-nucleotide mutated KRAS genes by distinguishing them from normal genes", 44TH ANNUAL MEETING OF THE MOLECULAR BIOLOGY SOCIETY OF JAPAN; DECEMBER 02-04, 2020, MOLECULAR BIOLOGY SOCIETY OF JAPAN, JP, vol. 44, 19 November 2020 (2020-11-19) - 4 December 2020 (2020-12-04), JP, pages 2111, XP009538604
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