WO2003095647A3 - CHIMERIC snRNA MOLECULES CARRYING ANTISENSE SEQUENCES AGAINST THE SPLICE JUNCTIONS OF THE DYSTROPHIN GENE AND THEIR THERAPEUTIC APPLICATIONS - Google Patents

CHIMERIC snRNA MOLECULES CARRYING ANTISENSE SEQUENCES AGAINST THE SPLICE JUNCTIONS OF THE DYSTROPHIN GENE AND THEIR THERAPEUTIC APPLICATIONS Download PDF

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Publication number
WO2003095647A3
WO2003095647A3 PCT/IT2003/000273 IT0300273W WO03095647A3 WO 2003095647 A3 WO2003095647 A3 WO 2003095647A3 IT 0300273 W IT0300273 W IT 0300273W WO 03095647 A3 WO03095647 A3 WO 03095647A3
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WIPO (PCT)
Prior art keywords
splice junctions
gene
therapeutic applications
antisense sequences
dystrophin gene
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Ceased
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PCT/IT2003/000273
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French (fr)
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WO2003095647A2 (en
Inventor
Irene Bozzoni
Angelis Fernanda Gabriella De
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Universita degli Studi di Roma La Sapienza
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Universita degli Studi di Roma La Sapienza
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Priority to AU2003231928A priority Critical patent/AU2003231928A1/en
Priority to SI200331749T priority patent/SI1501931T1/en
Priority to EP03749984A priority patent/EP1501931B1/en
Priority to AT03749984T priority patent/ATE449170T1/en
Priority to DE60330136T priority patent/DE60330136D1/en
Priority to DK03749984.5T priority patent/DK1501931T3/en
Publication of WO2003095647A2 publication Critical patent/WO2003095647A2/en
Publication of WO2003095647A3 publication Critical patent/WO2003095647A3/en
Anticipated expiration legal-status Critical
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    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/11—Antisense
    • C12N2310/111—Antisense spanning the whole gene, or a large part of it

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Biomedical Technology (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Molecular Biology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Biochemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Physics & Mathematics (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Preparation Of Compounds By Using Micro-Organisms (AREA)
  • Acyclic And Carbocyclic Compounds In Medicinal Compositions (AREA)

Abstract

A gene is described that encodes for a snRNA modified such that it contains an antisense sequence complementary to at least one of the two splice junctions at 5´and/or 3´of one of the exons encoding for a protein of therapeutic interest, whose sequence should be skipped during the splicing process that converts the pre-mRNA to the mature mRNA. The gene is particularly advantageous which it contain segments complementary to both splice junctions. If introduced into appropriate vectors, the gene is useful in gene therapy, particularly in the treatment of Duchenne Muscular Dystrophies, dystrophic diseases.
PCT/IT2003/000273 2002-05-08 2003-05-06 CHIMERIC snRNA MOLECULES CARRYING ANTISENSE SEQUENCES AGAINST THE SPLICE JUNCTIONS OF THE DYSTROPHIN GENE AND THEIR THERAPEUTIC APPLICATIONS Ceased WO2003095647A2 (en)

Priority Applications (6)

Application Number Priority Date Filing Date Title
AU2003231928A AU2003231928A1 (en) 2002-05-08 2003-05-06 CHIMERIC snRNA MOLECULES CARRYING ANTISENSE SEQUENCES AGAINST THE SPLICE JUNCTIONS OF THE DYSTROPHIN GENE AND THEIR THERAPEUTIC APPLICATIONS
SI200331749T SI1501931T1 (en) 2002-05-08 2003-05-06 Chimeric snrna molecules carrying antisense sequences against the splice junctions of the dystrophin gene and their therapeutic applications
EP03749984A EP1501931B1 (en) 2002-05-08 2003-05-06 Chimeric snrna molecules carrying antisense sequences against the splice junctions of the dystrophin gene and their therapeutic applications
AT03749984T ATE449170T1 (en) 2002-05-08 2003-05-06 CHIMERIC SNRNA MOLECULES WITH ANTISENSE SEQUENCES AGAINST THE ßSPLICE JUNCTIONSß OF THE DYSTROPHIN GENE AND THEIR THERAPEUTIC USES
DE60330136T DE60330136D1 (en) 2002-05-08 2003-05-06 CHIMERIC SNRNA MOLECULES WITH ANTISENSE SEQUENCES AGAINST THE "SPLICE JUNCTIONS" OF THE DYSTROPHINE GENE AND THEIR THERAPEUTIC USES
DK03749984.5T DK1501931T3 (en) 2002-05-08 2003-05-06 Chimeric snRNA molecules that carry antisense sequences against the splicing compounds of the dystrophin gene and their therapeutic applications

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
IT2002RM000253A ITRM20020253A1 (en) 2002-05-08 2002-05-08 SNRNA CHEMICAL MOLECULES WITH ANTISENSE SEQUENCES FOR SPLICING JUNCTIONS OF THE DYSTROPHINE GENE AND THERAPEUTIC APPLICATIONS.
ITRM2002A000253 2002-05-08

Publications (2)

Publication Number Publication Date
WO2003095647A2 WO2003095647A2 (en) 2003-11-20
WO2003095647A3 true WO2003095647A3 (en) 2004-02-19

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PCT/IT2003/000273 Ceased WO2003095647A2 (en) 2002-05-08 2003-05-06 CHIMERIC snRNA MOLECULES CARRYING ANTISENSE SEQUENCES AGAINST THE SPLICE JUNCTIONS OF THE DYSTROPHIN GENE AND THEIR THERAPEUTIC APPLICATIONS

Country Status (11)

Country Link
EP (2) EP1501931B1 (en)
AT (1) ATE449170T1 (en)
AU (1) AU2003231928A1 (en)
CY (1) CY1109746T1 (en)
DE (1) DE60330136D1 (en)
DK (1) DK1501931T3 (en)
ES (1) ES2336909T3 (en)
IT (1) ITRM20020253A1 (en)
PT (1) PT1501931E (en)
SI (1) SI1501931T1 (en)
WO (1) WO2003095647A2 (en)

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US9243245B2 (en) 2007-10-26 2016-01-26 Academisch Ziekenhuis Leiden Means and methods for counteracting muscle disorders

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US9441221B2 (en) 2007-03-30 2016-09-13 Rutgers, The State University Of New Jersey Compositions and methods for gene silencing
US8907075B2 (en) 2007-03-30 2014-12-09 Samuel Ian Gunderson Compositions and methods for gene silencing
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Cited By (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US9243245B2 (en) 2007-10-26 2016-01-26 Academisch Ziekenhuis Leiden Means and methods for counteracting muscle disorders
US9499818B2 (en) 2007-10-26 2016-11-22 BioMarin Technologies, B.V. Methods and means for efficient skipping of at least one of the exons 51-53, 55, 57 and 59 of the human duchenne muscular dystrophy gene
US9528109B2 (en) 2007-10-26 2016-12-27 Biomarin Technologies B.V. Methods and means for efficient skipping of exon 45 in duchenne muscular dystrophy pre-mRNA
US9139828B2 (en) 2008-05-14 2015-09-22 Prosensa Technologies B.V. Method for efficient exon (44) skipping in duchenne muscular dystrophy and associated means

Also Published As

Publication number Publication date
EP1501931A2 (en) 2005-02-02
PT1501931E (en) 2010-02-23
CY1109746T1 (en) 2014-09-10
EP1501931B1 (en) 2009-11-18
AU2003231928A1 (en) 2003-11-11
ITRM20020253A0 (en) 2002-05-08
DE60330136D1 (en) 2009-12-31
ES2336909T3 (en) 2010-04-19
AU2003231928A8 (en) 2003-11-11
WO2003095647A2 (en) 2003-11-20
SI1501931T1 (en) 2010-03-31
ATE449170T1 (en) 2009-12-15
EP2151497A2 (en) 2010-02-10
EP2151497B1 (en) 2014-01-08
DK1501931T3 (en) 2010-03-29
ITRM20020253A1 (en) 2003-11-10
EP2151497A3 (en) 2010-03-10

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