Title:
ANTISENSE OLIGONUCLEOTIDE DIRECTED REMOVAL OF PROTEOLYTIC CLEAVAGE SITES, THE HCHWA-D MUTATION, AND TRINUCLEOTIDE REPEAT EXPANSIONS
Document Type and Number:
WIPO Patent Application WO/2015/053624
Kind Code:
A3
Abstract:
The invention relates to means and methods for removing a proteolytic cleavage site, the HCHWA-D mutation or the amino acids encoded by a trinucleotide repeat expansion from a protein comprising providing a cell that expresses pre-m RNA encoding said protein with an anti-sense oligonucleotide that induces skipping of the exonic sequence that comprises said proteolytic cleavage site, HCHWA-D mutation or trinucleotide repeat expansion, respectively, the method further comprising allowing translation of m RNA produced from said pre-m RNA.
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Inventors:
VAN ROON-MOM WILHELMINA MARIA CLASINA (NL)
EVERS MELVIN MAURICE (NL)
PEPERS BARRY ANTONIUS (NL)
AARTSMA-RUS ANNEMIEKE (NL)
VAN OMMEN GARRIT-JAN BOUDEWIJN (NL)
EVERS MELVIN MAURICE (NL)
PEPERS BARRY ANTONIUS (NL)
AARTSMA-RUS ANNEMIEKE (NL)
VAN OMMEN GARRIT-JAN BOUDEWIJN (NL)
Application Number:
PCT/NL2014/050698
Publication Date:
August 27, 2015
Filing Date:
October 07, 2014
Export Citation:
Assignee:
ACADEMISCH ZIEKENHUIS LEIDEN (NL)
International Classes:
C12N15/113; A61K31/7125; A61P25/28
Domestic Patent References:
| WO2012018257A1 | 2012-02-09 | |||
| WO2008018795A1 | 2008-02-14 |
Other References:
MELVIN M. EVERS ET AL: "Ataxin-3 protein modification as a treatment strategy for spinocerebellar ataxia type 3: Removal of the CAG containing exon", NEUROBIOLOGY OF DISEASE, vol. 58, 6 May 2013 (2013-05-06), pages 49 - 56, XP055163564, ISSN: 0969-9961, DOI: 10.1016/j.nbd.2013.04.019
JOSHUA LEE ET AL: "Antisense Therapy in Neurology", JOURNAL OF PERSONALIZED MEDICINE, vol. 3, no. 3, 2 August 2013 (2013-08-02), pages 144 - 176, XP055163807, DOI: 10.3390/jpm3030144
AARTSMA-RUS ANNEMIEKE ET AL: "Antisense-mediated exon skipping: A versatile tool with therapeutic and research applications", RNA, COLD SPRING HARBOR LABORATORY PRESS, US, vol. 13, no. 10, 7 August 2007 (2007-08-07), pages 1609 - 1624, XP009144451, ISSN: 1355-8382
IOANNIS ZALACHORAS ET AL: "Antisense-Mediated RNA Targeting: Versatile and Expedient Genetic Manipulation in the Brain", FRONTIERS IN MOLECULAR NEUROSCIENCE, vol. 4, 19 July 2011 (2011-07-19), XP055163591, DOI: 10.3389/fnmol.2011.00010
PIETRO SPITALI ET AL: "Splice Modulating Therapies for Human Disease", CELL, vol. 148, no. 6, 16 March 2012 (2012-03-16), pages 1085 - 1088, XP028471668, ISSN: 0092-8674, [retrieved on 20120227], DOI: 10.1016/J.CELL.2012.02.014
MELVIN M. EVERS ET AL: "Targeting Several CAG Expansion Diseases by a Single Antisense Oligonucleotide", PLOS ONE, vol. 6, no. 9, 1 September 2011 (2011-09-01), pages e24308 - e24308, XP055007055, ISSN: 1932-6203, DOI: 10.1371/journal.pone.0024308
AGNIESZKA FISZER ET AL: "An evaluation of oligonucleotide-based therapeutic strategies for polyQ diseases", BMC MOLECULAR BIOLOGY, BIOMED CENTRAL LTD, GB, vol. 13, no. 1, 7 March 2012 (2012-03-07), pages 6, XP021124662, ISSN: 1471-2199, DOI: 10.1186/1471-2199-13-6
HU JIAXIN ET AL: "Allele-selective inhibition of ataxin-3 (ATX3) expression by antisense oligomers and duplex RNAs", BIOLOGICAL CHEMISTRY, WALTER DE GRUYTER GMBH & CO, BERLIN, DE, vol. 392, no. 4, 25 July 2011 (2011-07-25), pages 315 - 325, XP008154586, ISSN: 1431-6730, [retrieved on 20110725], DOI: 10.1515/BC.2011.045
JOSHUA LEE ET AL: "Antisense Therapy in Neurology", JOURNAL OF PERSONALIZED MEDICINE, vol. 3, no. 3, 2 August 2013 (2013-08-02), pages 144 - 176, XP055163807, DOI: 10.3390/jpm3030144
AARTSMA-RUS ANNEMIEKE ET AL: "Antisense-mediated exon skipping: A versatile tool with therapeutic and research applications", RNA, COLD SPRING HARBOR LABORATORY PRESS, US, vol. 13, no. 10, 7 August 2007 (2007-08-07), pages 1609 - 1624, XP009144451, ISSN: 1355-8382
IOANNIS ZALACHORAS ET AL: "Antisense-Mediated RNA Targeting: Versatile and Expedient Genetic Manipulation in the Brain", FRONTIERS IN MOLECULAR NEUROSCIENCE, vol. 4, 19 July 2011 (2011-07-19), XP055163591, DOI: 10.3389/fnmol.2011.00010
PIETRO SPITALI ET AL: "Splice Modulating Therapies for Human Disease", CELL, vol. 148, no. 6, 16 March 2012 (2012-03-16), pages 1085 - 1088, XP028471668, ISSN: 0092-8674, [retrieved on 20120227], DOI: 10.1016/J.CELL.2012.02.014
MELVIN M. EVERS ET AL: "Targeting Several CAG Expansion Diseases by a Single Antisense Oligonucleotide", PLOS ONE, vol. 6, no. 9, 1 September 2011 (2011-09-01), pages e24308 - e24308, XP055007055, ISSN: 1932-6203, DOI: 10.1371/journal.pone.0024308
AGNIESZKA FISZER ET AL: "An evaluation of oligonucleotide-based therapeutic strategies for polyQ diseases", BMC MOLECULAR BIOLOGY, BIOMED CENTRAL LTD, GB, vol. 13, no. 1, 7 March 2012 (2012-03-07), pages 6, XP021124662, ISSN: 1471-2199, DOI: 10.1186/1471-2199-13-6
HU JIAXIN ET AL: "Allele-selective inhibition of ataxin-3 (ATX3) expression by antisense oligomers and duplex RNAs", BIOLOGICAL CHEMISTRY, WALTER DE GRUYTER GMBH & CO, BERLIN, DE, vol. 392, no. 4, 25 July 2011 (2011-07-25), pages 315 - 325, XP008154586, ISSN: 1431-6730, [retrieved on 20110725], DOI: 10.1515/BC.2011.045
Attorney, Agent or Firm:
JANSEN, C.M. (Johan de Wittlaan 7, JR Den Haag, NL)
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