Title:
RNA MODULATING OLIGONUCLEOTIDES WITH IMPROVED CHARACTERISTICS FOR TREATMENT OF NEUROMUSCULAR DISORDERS
Document Type and Number:
Japanese Patent JP2018027080
Kind Code:
A
Abstract:
PROBLEM TO BE SOLVED: To provide an improved oligonucleotide and its use for treating, ameliorating, preventing, delaying and/or treating a human cis-element repeat instability associated genetic neuromuscular or neurodegenerative disorder.SOLUTION: An oligonucleotide comprises a 2'-O-methyl RNA nucleotide residue, has a backbone where at least one phosphate moiety is replaced by a phosphorothioate moiety, comprises one or more 5-methylpyrimidine bases, and can hybridize to a repetitive element having (CAG)n as a repetitive nucleotide unit.SELECTED DRAWING: None
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Inventors:
PETER CHRISTIAN DE VISSER
SUSAN ALLEGONDA MARIA MULDERS
SUSAN ALLEGONDA MARIA MULDERS
Application Number:
JP2017133005A
Publication Date:
February 22, 2018
Filing Date:
July 06, 2017
Export Citation:
Assignee:
BIOMARIN TECH BV
International Classes:
C12N15/113; A61K31/7125; A61K48/00; A61P21/00; A61P21/02; A61P25/00; A61P25/14; A61P25/28; A61P43/00; C07H21/02
Domestic Patent References:
| JP2010500023A | 2010-01-07 | |||
| JP2011510678A | 2011-04-07 |
Foreign References:
| WO2011097614A1 | 2011-08-11 | |||
| WO2011097641A1 | 2011-08-11 |
Other References:
PLOS ONE, vol. vol.6 no.9 e24308, JPN6017003145, 2011, pages 1 - 11, ISSN: 0004223211
JOURNAL OF NEUROSCIENCE RESEARCH, vol. 89, JPN6017003146, 2011, pages 275 - 285, ISSN: 0004056981
PNAS, vol. 109, no. 11, JPN6017003154, March 2012 (2012-03-01), pages 4221 - 4226, ISSN: 0004056982
PNAS, vol. 106, no. 33, JPN6017003153, 2009, pages 13915 - 13920, ISSN: 0004056983
THE JOURNAL OF CLINICAL INVESTIGATION, vol. 121, no. 2, JPN6017003148, 2011, pages 500 - 507, ISSN: 0004056984
HUMAN MOLECULAR GENETICS, vol. vol.19 Review Issue 1, JPN6017003150, 2010, pages 90 - 97, ISSN: 0004056985
BMC MOLECULAR BIOLOGY, vol. 13, JPN5015005619, March 2012 (2012-03-01), pages 1 - 12, ISSN: 0004056986
BIOCHEMISTRY, vol. 49, no. 47, JPN5015005618, 2010, pages 10166 - 10178, ISSN: 0004056987
JOURNAL OF NEUROSCIENCE RESEARCH, vol. 89, JPN6017003146, 2011, pages 275 - 285, ISSN: 0004056981
PNAS, vol. 109, no. 11, JPN6017003154, March 2012 (2012-03-01), pages 4221 - 4226, ISSN: 0004056982
PNAS, vol. 106, no. 33, JPN6017003153, 2009, pages 13915 - 13920, ISSN: 0004056983
THE JOURNAL OF CLINICAL INVESTIGATION, vol. 121, no. 2, JPN6017003148, 2011, pages 500 - 507, ISSN: 0004056984
HUMAN MOLECULAR GENETICS, vol. vol.19 Review Issue 1, JPN6017003150, 2010, pages 90 - 97, ISSN: 0004056985
BMC MOLECULAR BIOLOGY, vol. 13, JPN5015005619, March 2012 (2012-03-01), pages 1 - 12, ISSN: 0004056986
BIOCHEMISTRY, vol. 49, no. 47, JPN5015005618, 2010, pages 10166 - 10178, ISSN: 0004056987
Attorney, Agent or Firm:
Ikeda adult
Junichiro Sakamaki
Masakazu Noda
Kazuhiro Yamaguchi
Junichiro Sakamaki
Masakazu Noda
Kazuhiro Yamaguchi