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1. WO2016025339 - METHODS AND MATERIALS FOR ACTIVATING AN INTERNAL RIBOSOME ENTRY SITE IN EXON 5 OF THE DMD GENE

Publication Number WO/2016/025339
Publication Date 18.02.2016
International Application No. PCT/US2015/044366
International Filing Date 07.08.2015
IPC
C12N 15/66 2006.01
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15Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
09Recombinant DNA-technology
63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
66General methods for inserting a gene into a vector to form a recombinant vector using cleavage and ligation; Use of non-functional linkers or adaptors, e.g. linkers containing the sequence for a restriction endonuclease
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15Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
09Recombinant DNA-technology
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A61K 31/7088 2006.01
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KPREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
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7088Compounds having three or more nucleosides or nucleotides
C07H 21/04 2006.01
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07ORGANIC CHEMISTRY
HSUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
21Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
04with deoxyribosyl as saccharide radical
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21Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
02with ribosyl as saccharide radical
A61P 21/00 2006.01
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61MEDICAL OR VETERINARY SCIENCE; HYGIENE
PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
21Drugs for disorders of the muscular or neuromuscular system
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A61K 31/573
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61MEDICAL OR VETERINARY SCIENCE; HYGIENE
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A61P 21/00
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21Drugs for disorders of the muscular or neuromuscular system
A61P 21/04
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
21Drugs for disorders of the muscular or neuromuscular system
04for myasthenia gravis
Applicants
  • RESEARCH INSTITUTE AT NATIONWIDE CHILDREN'S HOSPITAL [US]/[US]
  • UNIVERSITY OF WESTERN AUSTRALIA [AU]/[AU]
Inventors
  • FLANIGAN, Kevin
  • WEIN, Nicolas
  • WILTON, Steven
Agents
  • NOLAND, Greta, E.
Priority Data
62/035,39509.08.2014US
Publication Language English (EN)
Filing Language English (EN)
Designated States
Title
(EN) METHODS AND MATERIALS FOR ACTIVATING AN INTERNAL RIBOSOME ENTRY SITE IN EXON 5 OF THE DMD GENE
(FR) PROCÉDÉS ET MATÉRIAUX D'ACTIVATION D'UN SITE D'ENTRÉE DE RIBOSOME INTERNE DANS L'EXON 5 DU GÈNE DMD
Abstract
(EN)
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.
(FR)
La présente invention porte sur l'apport d'oligomères pour le traitement de patients dont le gène DMD comporte une mutation 5' autre qu'une duplication de l'exon 2 du DMD. L'invention concerne des procédés et des matériaux d'activation d'un site d'entrée de ribosome interne dans l'exon 5 du gène DMD résultant en la translation d'un isoforme tronqué fonctionnel de la dystrophine. Les procédés et matériaux peuvent servir au traitement de myopathies primitives issues de mutations 5' dans le gène DMD comme la myopathie de Duchenne ou la dystrophie de Becker.
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