Title:
NUCLEIC ACID MOLECULES FOR PSEUDOURIDYLATION
Document Type and Number:
WIPO Patent Application WO/2019/191232
Kind Code:
A3
Abstract:
The invention relates to nucleic acid molecules for pseudouridylation of a target uridine in a target RNA in a mammalian cell, wherein the nucleic acid molecule comprises a guide region capable of forming a partially double stranded nucleic acid complex with the target RNA comprising the target uridine, wherein the partially double stranded nucleic acid complex is capable of engaging a mammalian pseudouridylation enzyme, wherein the guide region assists in positioning the target uridine in the partially double stranded nucleic acid complex for it to be converted to a pseudouridine by the mammalian pseudouridylation enzyme.
Inventors:
KLEIN BART (NL)
TURUNEN JANNE (NL)
VAN SINT FIET LENKA (NL)
DA SILVA PEDRO DUARTE MORAIS FERNANDES (NL)
BOUDET JULIEN AUGUSTE (NL)
YU YI-TAO (US)
ADACHI HIRONORI (US)
ZOYSA MEEMANAGE DE (US)
TURUNEN JANNE (NL)
VAN SINT FIET LENKA (NL)
DA SILVA PEDRO DUARTE MORAIS FERNANDES (NL)
BOUDET JULIEN AUGUSTE (NL)
YU YI-TAO (US)
ADACHI HIRONORI (US)
ZOYSA MEEMANAGE DE (US)
Application Number:
PCT/US2019/024282
Publication Date:
December 19, 2019
Filing Date:
March 27, 2019
Export Citation:
Assignee:
UNIV ROCHESTER (US)
PROQR THERAPEUTICS II BV (NL)
PROQR THERAPEUTICS II BV (NL)
International Classes:
C12N15/113; A61K31/713; C12N15/10; C12N5/10
Domestic Patent References:
| WO2007064952A2 | 2007-06-07 | |||
| WO1998050586A1 | 1998-11-12 |
Other References:
M. XIAO ET AL: "Functionality and substrate specificity of human box H/ACA guide RNAs", RNA, vol. 15, no. 1, 25 November 2008 (2008-11-25), US, pages 176 - 186, XP055622093, ISSN: 1355-8382, DOI: 10.1261/rna.1361509
P. RICHARD ET AL: "Cotranscriptional Recognition of Human Intronic Box H/ACA snoRNAs Occurs in a Splicing-Independent Manner", MOLECULAR AND CELLULAR BIOLOGY, vol. 26, no. 7, 14 March 2006 (2006-03-14), pages 2540 - 2549, XP055622161, ISSN: 0270-7306, DOI: 10.1128/MCB.26.7.2540-2549.2006
CHEN WANG ET AL: "Architecture and assembly of mammalian H/ACA small nucleolar and telomerase ribonucleoproteins", THE EMBO JOURNAL, vol. 23, no. 8, 25 March 2004 (2004-03-25), pages 1857 - 1867, XP055621981, ISSN: 0261-4189, DOI: 10.1038/sj.emboj.7600181
XINLIANG ZHAO ET AL: "An H/ACA guide RNA directs U2 pseudouridylation at two different sites in the branchpoint recognition region in Xenopus oocytes", RNA, December 2002 (2002-12-01), United States, pages 1515 - 1525, XP055621861, Retrieved from the Internet DOI: 10.1017/S1355838202022537
B. E. JADY: "A small nucleolar guide RNA functions both in 2'-O-ribose methylation and pseudouridylation of the U5 spliceosomal RNA", THE EMBO JOURNAL, vol. 20, no. 3, 1 February 2001 (2001-02-01), pages 541 - 551, XP055622269, ISSN: 0261-4189, DOI: 10.1093/emboj/20.3.541
MICHELLE S. SCOTT ET AL: "Human miRNA Precursors with Box H/ACA snoRNA Features", PLOS COMPUTATIONAL BIOLOGY, vol. 5, no. 9, 18 September 2009 (2009-09-18), pages e1000507, XP055622091, DOI: 10.1371/journal.pcbi.1000507
JOHN KARIJOLICH ET AL: "Converting nonsense codons into sense codons by targeted pseudouridylation", NATURE, vol. 474, no. 7351, 15 June 2011 (2011-06-15), London, pages 395 - 398, XP055379443, ISSN: 0028-0836, DOI: 10.1038/nature10165
GUOWEI WU ET AL: "Pseudouridines in U2 snRNA stimulate the ATPase activity of Prp5 during spliceosome assembly", THE EMBO JOURNAL, vol. 35, no. 6, 12 February 2016 (2016-02-12), pages 654 - 667, XP055621833, ISSN: 0261-4189, DOI: 10.15252/embj.201593113
"Protein engineering", vol. 560, 27 April 2015, ACADEMIC PRESS, AMSTERDAM, NL, ISSN: 0076-6879, article GUOWEI WU ET AL: "Pseudouridine in mRNA", pages: 187 - 217, XP055621792, DOI: 10.1016/bs.mie.2015.03.009
M.-L. BORTOLIN ET AL: "Elements essential for accumulation and function of small nucleolar RNAs directing site-specific pseudouridylation of ribosomal RNAs", THE EMBO JOURNAL, vol. 18, no. 2, 15 January 1999 (1999-01-15), pages 457 - 469, XP055621726, DOI: 10.1093/emboj/18.2.457
P. RICHARD ET AL: "Cotranscriptional Recognition of Human Intronic Box H/ACA snoRNAs Occurs in a Splicing-Independent Manner", MOLECULAR AND CELLULAR BIOLOGY, vol. 26, no. 7, 14 March 2006 (2006-03-14), pages 2540 - 2549, XP055622161, ISSN: 0270-7306, DOI: 10.1128/MCB.26.7.2540-2549.2006
CHEN WANG ET AL: "Architecture and assembly of mammalian H/ACA small nucleolar and telomerase ribonucleoproteins", THE EMBO JOURNAL, vol. 23, no. 8, 25 March 2004 (2004-03-25), pages 1857 - 1867, XP055621981, ISSN: 0261-4189, DOI: 10.1038/sj.emboj.7600181
XINLIANG ZHAO ET AL: "An H/ACA guide RNA directs U2 pseudouridylation at two different sites in the branchpoint recognition region in Xenopus oocytes", RNA, December 2002 (2002-12-01), United States, pages 1515 - 1525, XP055621861, Retrieved from the Internet
B. E. JADY: "A small nucleolar guide RNA functions both in 2'-O-ribose methylation and pseudouridylation of the U5 spliceosomal RNA", THE EMBO JOURNAL, vol. 20, no. 3, 1 February 2001 (2001-02-01), pages 541 - 551, XP055622269, ISSN: 0261-4189, DOI: 10.1093/emboj/20.3.541
MICHELLE S. SCOTT ET AL: "Human miRNA Precursors with Box H/ACA snoRNA Features", PLOS COMPUTATIONAL BIOLOGY, vol. 5, no. 9, 18 September 2009 (2009-09-18), pages e1000507, XP055622091, DOI: 10.1371/journal.pcbi.1000507
JOHN KARIJOLICH ET AL: "Converting nonsense codons into sense codons by targeted pseudouridylation", NATURE, vol. 474, no. 7351, 15 June 2011 (2011-06-15), London, pages 395 - 398, XP055379443, ISSN: 0028-0836, DOI: 10.1038/nature10165
GUOWEI WU ET AL: "Pseudouridines in U2 snRNA stimulate the ATPase activity of Prp5 during spliceosome assembly", THE EMBO JOURNAL, vol. 35, no. 6, 12 February 2016 (2016-02-12), pages 654 - 667, XP055621833, ISSN: 0261-4189, DOI: 10.15252/embj.201593113
"Protein engineering", vol. 560, 27 April 2015, ACADEMIC PRESS, AMSTERDAM, NL, ISSN: 0076-6879, article GUOWEI WU ET AL: "Pseudouridine in mRNA", pages: 187 - 217, XP055621792, DOI: 10.1016/bs.mie.2015.03.009
M.-L. BORTOLIN ET AL: "Elements essential for accumulation and function of small nucleolar RNAs directing site-specific pseudouridylation of ribosomal RNAs", THE EMBO JOURNAL, vol. 18, no. 2, 15 January 1999 (1999-01-15), pages 457 - 469, XP055621726, DOI: 10.1093/emboj/18.2.457
Attorney, Agent or Firm:
DAVENPORT, Andrew, M. et al. (US)
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