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Title:
METHODS AND MEANS FOR EFFICIENT SKIPPING OF AT LEAST ONE OF THE FOLLOWING EXONS OF THE HUMAN DUCHENNE MUSCULAR DYSTROPHY GENE: 43, 46, 50- 53.
Document Type and Number:
WIPO Patent Application WO/2010/050802
Kind Code:
A3
Abstract:
The invention relates a method wherein a molecule is used for inducing and/or promoting skipping of at least one of exon 43, exon 46, exons 50-53 of the DMD pre- mRNA in a patient, preferably in an isolated cell of a patient, the method comprising providing said cell and/or said patient with a molecule. The invention also relates to said molecule as such.

Inventors:
DE KIMPE JOSEPHUS JOHANNES (NL)
PLATENBURG GERARDUS JOHANNES (NL)
VAN DEUTEKOM JUDITH CHRISTINA THEODORA (NL)
AARTSMA-RUS ANNEMIEKE (NL)
VAN OMMEN GARRIT-JAN BOUDEWIJN (NL)
Application Number:
PCT/NL2009/050113
Publication Date:
August 12, 2010
Filing Date:
March 11, 2009
Export Citation:
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Assignee:
ACADEMISCH ZIEKENHUIS LEIDEN (NL)
PROSENSA BV (NL)
PROSENSA TECHNOLOGIES BV (NL)
PROSENSA HOLDING BV (NL)
DE KIMPE JOSEPHUS JOHANNES (NL)
PLATENBURG GERARDUS JOHANNES (NL)
VAN DEUTEKOM JUDITH CHRISTINA THEODORA (NL)
AARTSMA-RUS ANNEMIEKE (NL)
VAN OMMEN GARRIT-JAN BOUDEWIJN (NL)
International Classes:
A61K31/7105; A61K48/00; A61P21/00; C12N5/10
Domestic Patent References:
WO2009054725A22009-04-30
WO2006000057A12006-01-05
Foreign References:
EP1857548A12007-11-21
Other References:
AARTSMA-RUS ANNEMIEKE ET AL: "Functional analysis of 114 exon-internal AONs for targeted DMD exon skipping: indication for steric hindrance of SR protein binding sites", OLIGONUCLEOTIDES, MARY ANN LIEBERT, NEW YORK, NY, US, vol. 15, no. 4, 1 December 2005 (2005-12-01), pages 284 - 297, XP002406791, ISSN: 1545-4576
WILTON STEVE D ET AL: "Antisense oligonucleotide-induced exon skipping across the human dystrophin gene transcript", MOLECULAR THERAPY, NATURE PUBLISHING GROUP, GB, vol. 15, no. 7, 1 July 2007 (2007-07-01), pages 1288 - 1296, XP002544728, ISSN: 1525-0024
AARTSMA-RUS ET AL: "Exploring the Frontiers of Therapeutic Exon Skipping for Duchenne Muscular Dystrophy by Double Targeting within One or Multiple Exons", MOLECULAR THERAPY, ACADEMIC PRESS, SAN DIEGO, CA, US, vol. 14, no. 3, 12 August 2006 (2006-08-12), pages 401 - 407, XP005844833, ISSN: 1525-0016
Attorney, Agent or Firm:
KETELAARS, Maarten (JS The Hague, NL)
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