Login| Sign Up| Help| Contact|

Patent Searching and Data


Title:
GUIDE RNA FOR EDITING POLYADENYLATION SIGNAL SEQUENCE OF TARGET RNA
Document Type and Number:
WIPO Patent Application WO/2022/138929
Kind Code:
A1
Abstract:
[Problem] To provide an antisense guide RNA, and nucleic acid encoding same, for editing the polyadenylation signal sequence of a target RNA, with which a highly efficient control of target RNA expression can be expected. [Solution] As a result of intensive investigations into art for the highly efficient control of the expression of a target RNA, the present inventors achieved the present invention by finding a guide RNA for ADAR-mediated editing of a target RNA, comprising an antisense region that is complementary to a portion of a target RNA containing a polyadenylation signal sequence.

Inventors:
ARAI TAKATOMO (JP)
MANDA MARIKO (JP)
Application Number:
PCT/JP2021/048231
Publication Date:
June 30, 2022
Filing Date:
December 24, 2021
Export Citation:
Click for automatic bibliography generation   Help
Assignee:
ASTELLAS PHARMA INC (JP)
International Classes:
C12N15/113; A61K35/76; A61K48/00; C12N1/15; C12N1/19; C12N1/21; C12N5/10; C12N15/09; C12N15/63; C12P19/34
Domestic Patent References:
WO2019111957A12019-06-13
WO2020051555A12020-03-12
WO2018161032A12018-09-07
WO2017050306A12017-03-30
WO2019071274A12019-04-11
WO2019005884A12019-01-03
WO2019071048A12019-04-11
WO2021117729A12021-06-17
WO2021020550A12021-02-04
Foreign References:
JP2017537618A2017-12-21
DE102015012522B32016-06-02
Other References:
JOUBERT ROMAINS, MARIOT VIRGINIE, CHARPENTIER MARINE, CONCORDET JEAN PAUL, DUMONCEAUX JULIE: "Gene Editing Targeting the DUX4 Polyadenylation Signal: A Therapy for FSHD?", JOURNAL OF PERSONALIZED MEDICINE, vol. 11, no. 1, pages 1 - 10, XP055945013, DOI: 10.3390/jpm11010007
"mRNA 3' end processing and metabolism", 4 June 2021, ISBN: 978-0-12-823573-7, article SHIN, J.: "Modulation of alternative cleavage and polyadenylation events by dCas9-mediated CRISPRpas", pages: 459 - 482, XP009537702, DOI: 10.1016/bs.mie.2021.04.016
MOL. CELL, vol. 33, 2009, pages 365 - 376
PROC. NATL. ACAD. SCI. USA, vol. 89, 1992, pages 4324 - 4328
EMBO J., vol. 15, 1996, pages 7168 - 7177
ELIFE, vol. 6, 2017, pages e25237
RNA, vol. 4, 1998, pages 1321 - 1331
NATURE, vol. 382, 1996, pages 642 - 646
NUCLEIC ACIDS RESEARCH, vol. 42, 2014, pages 5217 - 5233
GENE, vol. 95, 1990, pages 85 - 90
MOL. CELL, vol. 15, 2004, pages 173 - 183
CELL, vol. 91, 1997, pages 457 - 466
CELL, vol. 131, 2007, pages 1340 - 1353
SCIENCE, vol. 365, 2019, pages 382 - 386
RNA, vol. 6, 2000, pages 755 - 767
NUCLEIC ACIDS RESEARCH, vol. 45, 2017, pages 2797 - 2808
NATURE METHODS, vol. 16, 2019, pages 239 - 242
NAT. BIOTECHNOL., vol. 37, 2019, pages 1059 - 1069
ENG. DES. SEL., vol. 1, 2018, pages 471 - 478
CHEM. COMMUN., vol. 54, 2018, pages 2377 - 2380
PROTEIN ENG. DES. SEL., vol. 1, 2018, pages 471 - 478
NUCLEIC ACIDS RESEARCH, vol. 44, 2016, pages e157 - 9564
VIRUS RES, vol. 169, 2012, pages 63 - 71
GENE & DEVELOPMENT, vol. 25, 2011, pages 1770 - 1782
RNA, vol. 7, 2001, pages 846 - 858
MOL. THER., vol. 7, 2003, pages 237 - 247
BMC BIOTECH., vol. 8, no. 48, 2008
ANTIVIRAL CHEMISTRY & CHEMOTHERAPY, vol. 7, 1996, pages 86 - 93
GENE THER., vol. 4, 1997, pages 45 - 54
NAT. BIOTECHNOL., vol. 20, 2002, pages 497 - 500
INT. J. BIOCHEM. MOL. BIOL., vol. 4, 2013, pages 27 - 40
INTEGR. BIOL., vol. 1, 2009, pages 499 - 505
MOL. THER, vol. 7, 2003, pages 237 - 247
"Current Protocols in Molecular Biology", 1987, JOHN WILEY & SONS
BIOCHEMICAL SCIENCES, vol. 26, 2001, pages 376 - 384
J. MOL. BIOL., vol. 48, 1970, pages 443 - 453
NUCLEIC ACIDS RESEARCH, vol. 31, 2003, pages 100
GREEN, M.R.SAMBROOK, J.: "Molecular Cloning: A Laboratory Manual", 2012, COLD SPRING HARBOR LABORATORY PRESS
BIOINFORMATICS, vol. 25, 2009, pages 3244 - 3250
Attorney, Agent or Firm:
HAMAI Kousuke et al. (JP)
Download PDF: