Title:
METHODS AND MATERIALS FOR ACTIVATING AN INTERNAL RIBOSOME ENTRY SITE IN EXON 5 OF THE DMD GENE
Document Type and Number:
WIPO Patent Application WO/2016/025339
Kind Code:
A3
Abstract:
The present invention relates to the delivery of oligomers for treating patients with a 5' mutation in their DMD gene other than a DMD exon 2 duplication. The invention provides methods and materials for activating an internal ribosome entry site in exon 5 of the DMD gene resulting in translation of a functional truncated isoform of dystrophin. The methods and materials can be used for the treatment of muscular dystrophies arising from 5' mutations in the DMD gene such as Duchenne Muscular Dystrophy or Becker Muscular Dystrophy.
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Inventors:
FLANIGAN KEVIN (US)
WEIN NICOLAS (US)
WILTON STEVEN (AU)
WEIN NICOLAS (US)
WILTON STEVEN (AU)
Application Number:
PCT/US2015/044366
Publication Date:
April 21, 2016
Filing Date:
August 07, 2015
Export Citation:
Assignee:
RES INST NATIONWIDE CHILDRENS HOSPITAL (US)
UNIV WESTERN AUSTRALIA (AU)
UNIV WESTERN AUSTRALIA (AU)
International Classes:
A61K31/7088; C12N15/66; A61P21/00; C07H21/02; C07H21/04; C12N15/63
Other References:
WEIN ET AL.: "Successful use of Out-of-Frame Exon 2 Skipping induces IRES-Driven expression of the N-Truncated dystrophin isoform: promising approach for treating other 5' Dystrophin Mutations.", MOLECULAR THERAPY, vol. 22, no. Supp. 1, May 2014 (2014-05-01), pages S294 - S295, XP055275684
GREER ET AL.: "Targeted exon skipping to correct exon duplications in the dystrophin gene .", MOL THER NUCLEIC ACIDS, vol. 3, 18 March 2014 (2014-03-18), pages e155, XP002728584
GOYENVALLE ET AL.: "Engineering multiple U7snRNA constructs to induce single and multiexon- skipping for Duchenne muscular dystrophy.", MOL THER., vol. 20, no. 6, 2012, pages 1212 - 21, XP008158627
WEIN ET AL.: "Translation from a DMD exon 5 IRES results in a functional dystrophin isoform that attenuates dystrophinopathy in humans and mice.", NAT MED., vol. 20, no. 9, 10 August 2014 (2014-08-10), pages 992 - 1000, XP055275685
GREER ET AL.: "Targeted exon skipping to correct exon duplications in the dystrophin gene .", MOL THER NUCLEIC ACIDS, vol. 3, 18 March 2014 (2014-03-18), pages e155, XP002728584
GOYENVALLE ET AL.: "Engineering multiple U7snRNA constructs to induce single and multiexon- skipping for Duchenne muscular dystrophy.", MOL THER., vol. 20, no. 6, 2012, pages 1212 - 21, XP008158627
WEIN ET AL.: "Translation from a DMD exon 5 IRES results in a functional dystrophin isoform that attenuates dystrophinopathy in humans and mice.", NAT MED., vol. 20, no. 9, 10 August 2014 (2014-08-10), pages 992 - 1000, XP055275685
Attorney, Agent or Firm:
NOLAND, Greta, E. et al. (Gerstein & Borun LLP233 S. Wacker Drive,6300 Willis Towe, Chicago IL, US)
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