CA2360997A1 - Non-invasive method for detecting target rna - Google Patents

Non-invasive method for detecting target rna Download PDF

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Publication number
CA2360997A1
CA2360997A1 CA002360997A CA2360997A CA2360997A1 CA 2360997 A1 CA2360997 A1 CA 2360997A1 CA 002360997 A CA002360997 A CA 002360997A CA 2360997 A CA2360997 A CA 2360997A CA 2360997 A1 CA2360997 A1 CA 2360997A1
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CA
Canada
Prior art keywords
antisense
target
rna
gene
oligomer
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Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
Abandoned
Application number
CA002360997A
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English (en)
French (fr)
Inventor
Patrick L. Iversen
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Sarepta Therapeutics Inc
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Individual
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
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Publication of CA2360997A1 publication Critical patent/CA2360997A1/en
Abandoned legal-status Critical Current

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    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113Non-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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/50Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P11/00Drugs for disorders of the respiratory system
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • A61P25/14Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P31/00Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
    • A61P31/12Antivirals
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00Antineoplastic agents
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00Drugs for disorders of the cardiovascular system
    • A61P9/14Vasoprotectives; Antihaemorrhoidals; Drugs for varicose therapy; Capillary stabilisers
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113Non-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
    • C12N15/1135Non-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 oncogenes or tumor suppressor genes
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12QMEASURING OR TESTING PROCESSES INVOLVING ENZYMES, NUCLEIC ACIDS OR MICROORGANISMS; COMPOSITIONS OR TEST PAPERS THEREFOR; PROCESSES OF PREPARING SUCH COMPOSITIONS; CONDITION-RESPONSIVE CONTROL IN MICROBIOLOGICAL OR ENZYMOLOGICAL PROCESSES
    • C12Q1/00Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions
    • C12Q1/68Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions involving nucleic acids
    • C12Q1/6876Nucleic acid products used in the analysis of nucleic acids, e.g. primers or probes
    • C12Q1/6883Nucleic acid products used in the analysis of nucleic acids, e.g. primers or probes for diseases caused by alterations of genetic material
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00Medicinal preparations containing peptides
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/31Chemical structure of the backbone
    • C12N2310/314Phosphoramidates
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/32Chemical structure of the sugar
    • C12N2310/323Chemical structure of the sugar modified ring structure
    • C12N2310/3233Morpholino-type ring
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/35Nature of the modification
    • C12N2310/351Conjugate
    • C12N2310/3517Marker; Tag
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12QMEASURING OR TESTING PROCESSES INVOLVING ENZYMES, NUCLEIC ACIDS OR MICROORGANISMS; COMPOSITIONS OR TEST PAPERS THEREFOR; PROCESSES OF PREPARING SUCH COMPOSITIONS; CONDITION-RESPONSIVE CONTROL IN MICROBIOLOGICAL OR ENZYMOLOGICAL PROCESSES
    • C12Q2600/00Oligonucleotides characterized by their use
    • C12Q2600/158Expression markers

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biomedical Technology (AREA)
  • General Health & Medical Sciences (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Molecular Biology (AREA)
  • Veterinary Medicine (AREA)
  • Physics & Mathematics (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • Biophysics (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Analytical Chemistry (AREA)
  • Plant Pathology (AREA)
  • Oncology (AREA)
  • Pathology (AREA)
  • Immunology (AREA)
  • Virology (AREA)
  • Heart & Thoracic Surgery (AREA)
  • Psychology (AREA)
  • Neurology (AREA)
  • Neurosurgery (AREA)
  • Epidemiology (AREA)
  • Communicable Diseases (AREA)
  • Vascular Medicine (AREA)
CA002360997A 1999-01-29 2000-01-28 Non-invasive method for detecting target rna Abandoned CA2360997A1 (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US11784699P 1999-01-29 1999-01-29
US60/117,846 1999-01-29
PCT/US2000/002475 WO2000045167A2 (en) 1999-01-29 2000-01-28 Non-invasive method for detecting target rna

Publications (1)

Publication Number Publication Date
CA2360997A1 true CA2360997A1 (en) 2000-08-03

Family

ID=22375152

Family Applications (2)

Application Number Title Priority Date Filing Date
CA002360997A Abandoned CA2360997A1 (en) 1999-01-29 2000-01-28 Non-invasive method for detecting target rna
CA2359317A Expired - Lifetime CA2359317C (en) 1999-01-29 2000-01-29 Method of treating restenosis by antisense targeting of c-myc

Family Applications After (1)

Application Number Title Priority Date Filing Date
CA2359317A Expired - Lifetime CA2359317C (en) 1999-01-29 2000-01-29 Method of treating restenosis by antisense targeting of c-myc

Country Status (9)

Country Link
US (1) US6365351B1 (enExample)
EP (2) EP1173561A2 (enExample)
JP (2) JP2002535015A (enExample)
KR (2) KR20010102992A (enExample)
AT (1) ATE445700T1 (enExample)
AU (2) AU778057B2 (enExample)
CA (2) CA2360997A1 (enExample)
DE (1) DE60043149D1 (enExample)
WO (2) WO2000045167A2 (enExample)

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US20010024783A1 (en) * 1999-01-29 2001-09-27 Avi Biopharma, Inc. Non-invasive method for detecting target RNA
JP2003533986A (ja) * 2000-05-04 2003-11-18 エイブイアイ バイオファーマ, インコーポレイテッド スプライス領域アンチセンス組成物および方法
AU2001263197B2 (en) * 2000-05-17 2006-11-09 The Board Of Regents Of The University Of Nebraska Antisense enzyme inhibitors for metabolic redirection
US7264925B2 (en) * 2000-08-30 2007-09-04 Avi Biopharma, Inc. Method for analysis of oligonucleotide analogs
US7754238B2 (en) 2002-05-03 2010-07-13 Avi Biopharma, Inc. Delivery of microparticle-conjugated drugs for inhibition of stenosis
EP2351844B1 (en) * 2003-04-29 2014-06-11 Sarepta Therapeutics, Inc. Compositions for enhancing transport and antisense efficacy of nucleic acid analog into cells
EP1668145A4 (en) * 2003-08-07 2010-03-10 Avi Biopharma Inc SENSE ANTIVIRUS COMPOUND AND METHOD FOR TREATING SSRNA VIRUS INFECTION
US20050222068A1 (en) * 2003-10-23 2005-10-06 Mourich Dan V Method and antisense composition for selective inhibition of HIV infection in hematopoietic cells
US20050171044A1 (en) * 2003-12-24 2005-08-04 Stein David A. Oligonucleotide compound and method for treating nidovirus infections
WO2005072527A2 (en) 2004-01-23 2005-08-11 Avi Biopharma, Inc. Antisense oligomers and methods for inducing immune tolerance and immunosuppression
US20050288246A1 (en) * 2004-05-24 2005-12-29 Iversen Patrick L Peptide conjugated, inosine-substituted antisense oligomer compound and method
USRE48960E1 (en) 2004-06-28 2022-03-08 The University Of Western Australia Antisense oligonucleotides for inducing exon skipping and methods of use thereof
ATE498685T1 (de) 2004-06-28 2011-03-15 Univ Western Australia Antisense-oligonukleotide zur induktion von exon- skipping sowie verfahren zur verwendung davon
CA2875436A1 (en) * 2004-07-02 2006-08-17 Sarepta Therapeutics, Inc. Antisense antibacterial method and compound
US8129352B2 (en) 2004-09-16 2012-03-06 Avi Biopharma, Inc. Antisense antiviral compound and method for treating ssRNA viral infection
US8357664B2 (en) * 2004-10-26 2013-01-22 Avi Biopharma, Inc. Antisense antiviral compound and method for treating influenza viral infection
US7524829B2 (en) * 2004-11-01 2009-04-28 Avi Biopharma, Inc. Antisense antiviral compounds and methods for treating a filovirus infection
EP1838875A4 (en) * 2004-12-30 2010-08-25 Todd M Hauser COMPOSITIONS AND METHODS FOR MODULATING GENE EXPRESSION USING SELF-PROTECTED OLIGONUCLEOTIDES
AU2006213686A1 (en) 2005-02-09 2006-08-17 Avi Bio Pharma, Inc. Antisense composition and method for treating muscle atrophy
US20060240032A1 (en) * 2005-03-31 2006-10-26 Hinrichs David J Immunomodulating compositions and methods for use in the treatment of human autoimmune diseases
US20060293268A1 (en) * 2005-05-05 2006-12-28 Rieder Aida E Antisense antiviral compounds and methods for treating foot and mouth disease
US8067571B2 (en) 2005-07-13 2011-11-29 Avi Biopharma, Inc. Antibacterial antisense oligonucleotide and method
US7790694B2 (en) * 2005-07-13 2010-09-07 Avi Biopharma Inc. Antisense antibacterial method and compound
US8524676B2 (en) * 2005-09-08 2013-09-03 Sarepta Therapeutics, Inc. Method for treating enterovirus or rhinovirus infection using antisense antiviral compounds
WO2007030576A2 (en) * 2005-09-08 2007-03-15 Avi Biopharma, Inc. Antisense antiviral compound and method for treating picornavirus infection
US8501704B2 (en) 2005-11-08 2013-08-06 Sarepta Therapeutics, Inc. Immunosuppression compound and treatment method
DE602007006457D1 (de) * 2006-03-07 2010-06-24 Avi Biopharma Inc Antivirale antisense-verbindung und verfahren zur behandlung von arenavirus-infektion
US8785407B2 (en) * 2006-05-10 2014-07-22 Sarepta Therapeutics, Inc. Antisense antiviral agent and method for treating ssRNA viral infection
ES2645410T3 (es) * 2006-05-10 2017-12-05 Sarepta Therapeutics, Inc. Análogos de oligonucleótidos que tienen enlaces intersubunitarios catiónicos
US20070265215A1 (en) * 2006-05-11 2007-11-15 Iversen Patrick L Antisense restenosis composition and method
US20100016215A1 (en) 2007-06-29 2010-01-21 Avi Biopharma, Inc. Compound and method for treating myotonic dystrophy
JP5864100B2 (ja) 2007-06-29 2016-02-17 サレプタ セラピューティクス インコーポレイテッド 組織特異的ペプチドコンジュゲートおよび方法
TR201902952T4 (tr) 2008-10-24 2019-03-21 Sarepta Therapeutics Inc Dmd için ekson atlama bileşimleri.
WO2010080554A1 (en) * 2008-12-17 2010-07-15 Avi Biopharma, Inc. Antisense compositions and methods for modulating contact hypersensitivity or contact dermatitis
US20110269665A1 (en) 2009-06-26 2011-11-03 Avi Biopharma, Inc. Compound and method for treating myotonic dystrophy
CA2780563C (en) 2009-11-12 2025-05-06 Univ Western Australia Antisense Molecules and Methods for Treating Pathologies
WO2011060320A1 (en) 2009-11-13 2011-05-19 Avi Biopharma, Inc. Antisense antiviral compound and method for treating influenza viral infection
EP2576574A2 (en) 2010-05-28 2013-04-10 Sarepta Therapeutics, Inc. Oligonucleotide analogues having modified intersubunit linkages and/or terminal groups
US8198429B2 (en) 2010-08-09 2012-06-12 Avi Biopharma, Inc. Antisense antiviral compounds and methods for treating a filovirus infection
WO2012031243A2 (en) * 2010-09-03 2012-03-08 Avi Biopharma, Inc. dsRNA MOLECULES COMPRISING OLIGONUCLEOTIDE ANALOGS HAVING MODIFIED INTERSUBUNIT LINKAGES AND/OR TERMINAL GROUPS
US9161948B2 (en) 2011-05-05 2015-10-20 Sarepta Therapeutics, Inc. Peptide oligonucleotide conjugates
JP5850519B2 (ja) 2011-05-09 2016-02-03 ネッパジーン株式会社 モルフォリノ搭載バブルリポソームを有効成分としてなる筋ジストロフィー治療薬
US20130085139A1 (en) 2011-10-04 2013-04-04 Royal Holloway And Bedford New College Oligomers
CN108864192A (zh) 2011-11-18 2018-11-23 萨勒普塔医疗公司 功能改性的寡核苷酸及其亚单元
NZ775701A (en) 2013-03-14 2022-08-26 Sarepta Therapeutics Inc Exon skipping compositions for treating muscular dystrophy
NZ732507A (en) 2013-03-15 2018-08-31 Sarepta Therapeutics Inc Improved compositions for treating muscular dystrophy
MA41795A (fr) 2015-03-18 2018-01-23 Sarepta Therapeutics Inc Exclusion d'un exon induite par des composés antisens dans la myostatine
US11020417B2 (en) 2015-06-04 2021-06-01 Sarepta Therapeutics, Inc Methods and compounds for treatment of lymphocyte-related diseases and conditions
MD3554553T2 (ro) 2016-12-19 2022-10-31 Sarepta Therapeutics Inc Conjugați de oligomeri de omitere a exonului, pentru distrofie musculară

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Also Published As

Publication number Publication date
AU779128B2 (en) 2005-01-06
CA2359317A1 (en) 2000-08-03
AU3353700A (en) 2000-08-18
DE60043149D1 (de) 2009-11-26
EP1153129A1 (en) 2001-11-14
EP1153129B1 (en) 2009-10-14
CA2359317C (en) 2014-01-21
US6365351B1 (en) 2002-04-02
WO2000045167A3 (en) 2000-12-21
ATE445700T1 (de) 2009-10-15
WO2000044897A1 (en) 2000-08-03
KR20010101760A (ko) 2001-11-14
JP2002537230A (ja) 2002-11-05
AU778057B2 (en) 2004-11-11
KR20010102992A (ko) 2001-11-17
KR100684547B1 (ko) 2007-02-20
JP2002535015A (ja) 2002-10-22
WO2000045167A2 (en) 2000-08-03
AU2745900A (en) 2000-08-18
EP1173561A2 (en) 2002-01-23

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