CA2558160C - Oligonucleotide anti-sens ciblant la myostatine ou le fox01 pour le traitement de la perte de masse musculaire - Google Patents

Oligonucleotide anti-sens ciblant la myostatine ou le fox01 pour le traitement de la perte de masse musculaire Download PDF

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Publication number
CA2558160C
CA2558160C CA2558160A CA2558160A CA2558160C CA 2558160 C CA2558160 C CA 2558160C CA 2558160 A CA2558160 A CA 2558160A CA 2558160 A CA2558160 A CA 2558160A CA 2558160 C CA2558160 C CA 2558160C
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Prior art keywords
oligonucleotide
myostatin
muscle
gene
oligonucleotides
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Expired - Fee Related
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CA2558160A
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CA2558160A1 (fr
Inventor
Roderic M. K. Dale
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Lakewood-Amedex Inc
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Lakewood-Amedex Inc
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Publication of CA2558160A1 publication Critical patent/CA2558160A1/fr
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Expired - Fee Related legal-status Critical Current
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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
    • C12N15/1136—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 against growth factors, growth regulators, cytokines, lymphokines or hormones
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00—Drugs for disorders of the muscular or neuromuscular system
    • 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
    • C12N15/1138—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 against receptors or cell surface proteins
    • 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
    • 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
    • C12N2310/12—Type of nucleic acid catalytic nucleic acids, e.g. ribozymes
    • 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
    • C12N2310/14—Type of nucleic acid interfering N.A.
    • 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
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Chemical & Material Sciences (AREA)
  • Biomedical Technology (AREA)
  • Molecular Biology (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Biotechnology (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Public Health (AREA)
  • Microbiology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Biochemistry (AREA)
  • Animal Behavior & Ethology (AREA)
  • Medicinal Chemistry (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Physical Education & Sports Medicine (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Neurology (AREA)
  • Endocrinology (AREA)
  • Epidemiology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)

Abstract

L'invention concerne des compositions d'oligonucléotides ciblées sur des gènes impliqués dans l'atrophie musculaire et/ou la croissance musculaire. Dans certaines formes d'exécution, les oligonucléotides sont modifiés. Dans des variantes d'exécution, les compositions contiennent un oligonucléotide, ou plus d'un oligonucléotide. L'invention concerne également des procédés et des kits utilisant les compositions de l'invention pour le traitement des états d'atrophie musculaire et/ou pour le développement de la croissance musculaire.
CA2558160A 2006-06-30 2006-06-30 Oligonucleotide anti-sens ciblant la myostatine ou le fox01 pour le traitement de la perte de masse musculaire Expired - Fee Related CA2558160C (fr)

Applications Claiming Priority (1)

Application Number Priority Date Filing Date Title
PCT/US2006/025657 WO2008005002A1 (fr) 2006-06-30 2006-06-30 Compositions et procédés pour le traitement de l'atrophie musculaire

Publications (2)

Publication Number Publication Date
CA2558160A1 CA2558160A1 (fr) 2007-12-30
CA2558160C true CA2558160C (fr) 2017-01-03

Family

ID=37102772

Family Applications (1)

Application Number Title Priority Date Filing Date
CA2558160A Expired - Fee Related CA2558160C (fr) 2006-06-30 2006-06-30 Oligonucleotide anti-sens ciblant la myostatine ou le fox01 pour le traitement de la perte de masse musculaire

Country Status (4)

Country Link
EP (1) EP2124968A4 (fr)
AU (1) AU2006345724B2 (fr)
CA (1) CA2558160C (fr)
WO (1) WO2008005002A1 (fr)

Families Citing this family (9)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AU2004235396B2 (en) 2003-04-29 2010-11-25 Sarepta Therapeutics, Inc. Compositions for enhancing transport and antisense efficacy of nucleic acid analog into cells
US20050288246A1 (en) 2004-05-24 2005-12-29 Iversen Patrick L Peptide conjugated, inosine-substituted antisense oligomer compound and method
US8067571B2 (en) 2005-07-13 2011-11-29 Avi Biopharma, Inc. Antibacterial antisense oligonucleotide and method
US8097596B2 (en) 2006-06-30 2012-01-17 Lakewood-Amedex, Inc. Compositions and methods for the treatment of muscle wasting
US20100016215A1 (en) 2007-06-29 2010-01-21 Avi Biopharma, Inc. Compound and method for treating myotonic dystrophy
US9161948B2 (en) 2011-05-05 2015-10-20 Sarepta Therapeutics, Inc. Peptide oligonucleotide conjugates
US11020417B2 (en) 2015-06-04 2021-06-01 Sarepta Therapeutics, Inc Methods and compounds for treatment of lymphocyte-related diseases and conditions
MX2019006882A (es) 2016-12-19 2019-08-16 Sarepta Therapeutics Inc Conjugados de oligomeros de omision de exon para distrofia muscular.
AU2019206634B2 (en) * 2018-01-12 2024-06-27 Keros Therapeutics, Inc. Activin receptor type IIB variants and methods of use thereof

Family Cites Families (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP1002068B1 (fr) * 1997-07-14 2009-09-09 University of Liège Mutation du gene de la myostatine a l'origine de l'hypertrophie musculaire chez les mammiferes
US6656475B1 (en) * 1997-08-01 2003-12-02 The Johns Hopkins University School Of Medicine Growth differentiation factor receptors, agonists and antagonists thereof, and methods of using same
US20050124566A1 (en) * 2001-05-18 2005-06-09 Sirna Therapeutics, Inc. RNA interference mediated inhibition of myostatin gene expression using short interfering nucleic acid (siNA)
EP2351765A3 (fr) * 2001-07-10 2012-02-22 Lakewood-Amedex, Inc Compositions pharmacologiques contenant des oligonucleotides et leur utilisation
AU2006213686A1 (en) * 2005-02-09 2006-08-17 Avi Bio Pharma, Inc. Antisense composition and method for treating muscle atrophy
CN100393320C (zh) * 2005-06-24 2008-06-11 奥林格斯技术有限公司 治疗肌肉萎缩的寡核苷酸药物

Also Published As

Publication number Publication date
CA2558160A1 (fr) 2007-12-30
EP2124968A1 (fr) 2009-12-02
WO2008005002A1 (fr) 2008-01-10
AU2006345724B2 (en) 2013-11-21
AU2006345724A1 (en) 2008-01-10
EP2124968A4 (fr) 2013-10-23

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