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CA2366055A1 - Procede de validation/d'invalidation de cibles et des voies - Google Patents

Procede de validation/d'invalidation de cibles et des voies Download PDF

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Publication number
CA2366055A1
CA2366055A1 CA002366055A CA2366055A CA2366055A1 CA 2366055 A1 CA2366055 A1 CA 2366055A1 CA 002366055 A CA002366055 A CA 002366055A CA 2366055 A CA2366055 A CA 2366055A CA 2366055 A1 CA2366055 A1 CA 2366055A1
Authority
CA
Canada
Prior art keywords
receptor
adenosine
oligo
target
receptors
Prior art date
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
CA002366055A
Other languages
English (en)
Inventor
Jonathan W. Nyce
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.)
Epigenesis Pharmaceuticals Inc
Original Assignee
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
Application filed by Individual filed Critical Individual
Publication of CA2366055A1 publication Critical patent/CA2366055A1/fr
Abandoned legal-status Critical Current

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Classifications

    • 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
    • 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
    • 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
    • C12Q—MEASURING 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
    • C12Q1/00—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions
    • C12Q1/68—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions involving nucleic acids
    • C12Q1/6876—Nucleic acid products used in the analysis of nucleic acids, e.g. primers or probes
    • C12Q1/6883—Nucleic acid products used in the analysis of nucleic acids, e.g. primers or probes for diseases caused by alterations of genetic material
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K2123/00—Preparations for testing in vivo
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • 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
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/315—Phosphorothioates
    • 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
    • C12N2310/33—Chemical structure of the base
    • 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
    • C12N2310/33—Chemical structure of the base
    • C12N2310/334—Modified C
    • C12N2310/3341—5-Methylcytosine

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Biomedical Technology (AREA)
  • Molecular Biology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Engineering & Computer Science (AREA)
  • Biotechnology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • Analytical Chemistry (AREA)
  • Plant Pathology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Immunology (AREA)
  • Epidemiology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Medicinal Chemistry (AREA)
  • Pathology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)

Abstract

La présente invention concerne un procédé pour déterminer l'existence d'une corrélation entre une fonction et une affection ou un état pathologique et un gène ou un ARNm codant un polypeptide cible présumé d'être associé à une affection ou à une condition, qui consiste à: disposer d'oligonucléotides (oligos) comprenant au plus environ 15 % d'adénosine (A), de préférence ne contenant pas d'adénosine, et qui est un antisens à la cible sélectionnée dans le groupe constitué des gènes cibles et leurs ARNm correspondants, les régions génomiques et les régions flanquantes de l'ARNm sélectionnées dans le groupe constitué des frontières intron-exon 3' et 5' et la section de juxtaposition entre les régions codantes et non codantes, et tous les segments de l'ARNm codant des polypeptides associés à une affection ou à une condition prédéterminée; sélectionner parmi les oligos un qui présente une activité d'inhibition ou d'ablation notable vis-à-vis de l'expression d'un polypeptide codé par le ARNm lors d'une hybridation in vitro à l'ARNm cible; administrer à un sujet une quantité efficace d'oligo sélectionné pour une hybridation in vivo à l'ARNm cible; et évaluer une fonction sujet=s qui est associée à l'affection ou la condition avant et après l'administration de l'oligo; dans lequel une modification dans la valeur de la fonction=s supérieure à environ 70 % signifie une corrélation positive, entre environ 40 et environ 70 % une corrélation possible, et inférieure à 30 % un manque de corrélation. De préférence, ledit procédé effectue l'administration des oligos in situ où la cible est localisée, par exemple dans le système respiratoire du sujet=s lors d'une validation de cibles associées à des fonctions malignes et autres fonctions pulmonaires et respiratoires, de sorte que l'agent peut accéder directement aux poumons. Alternativement, de telles adénosines (desA) peuvent être délivrées directement au système central nerveux ou à d'autres organes, systèmes de tissus ou d'organes, au moyen de formulations d'administration connues.
CA002366055A 1999-03-05 2000-03-02 Procede de validation/d'invalidation de cibles et des voies Abandoned CA2366055A1 (fr)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US60/122,140 1999-02-26
US12295099P 1999-03-05 1999-03-05
PCT/US2000/005643 WO2000051621A1 (fr) 1999-03-05 2000-03-02 Procede de validation/d'invalidation de cibles et des voies

Publications (1)

Publication Number Publication Date
CA2366055A1 true CA2366055A1 (fr) 2000-09-08

Family

ID=22405857

Family Applications (1)

Application Number Title Priority Date Filing Date
CA002366055A Abandoned CA2366055A1 (fr) 1999-03-05 2000-03-02 Procede de validation/d'invalidation de cibles et des voies

Country Status (10)

Country Link
EP (1) EP1165093A4 (fr)
JP (1) JP2002537792A (fr)
KR (1) KR20020068262A (fr)
CN (1) CN1348376A (fr)
AU (1) AU3512300A (fr)
BR (1) BR0009247A (fr)
CA (1) CA2366055A1 (fr)
IL (1) IL145034A0 (fr)
MX (1) MXPA01008870A (fr)
WO (1) WO2000051621A1 (fr)

Families Citing this family (14)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP1255550A2 (fr) 2000-02-17 2002-11-13 Cv Therapeutics, Inc. Procede d'identification et d'utilisation d'antagonistes du recepteur d'adenosine a2b pour agir sur la proliferation cellulaire d'un mammifere
DE10346487A1 (de) * 2003-10-02 2005-05-12 Transmit Technologietransfer Verfahren zur Herstellung eines Zell- und/oder Gewebe- und/oder Krankheitsphasen-spezifischen Arzneimittels
CA2836368C (fr) 2006-02-13 2020-04-07 Monsanto Technology Llc Selection et stabilisation de constructions d'arn double brin
EP2051734B1 (fr) 2006-08-18 2016-10-05 Armagen Technologies, Inc. Agents pour barrière hémato-encéphalique
DE102007021443A1 (de) * 2007-05-08 2008-11-13 Brahms Aktiengesellschaft Diagnose und Risikostratifizierung mittels NT-proET-1
US8974791B2 (en) 2007-07-27 2015-03-10 Armagen Technologies, Inc. Methods and compositions for increasing α-L-iduronidase activity in the CNS
EP2408474B1 (fr) 2009-03-18 2019-06-26 Armagen, Inc. Compositions et procédés pour le transport de protéines de fusion igg-récepteur leurre à travers la barrière hémato-encéphalique
HUE044865T2 (hu) 2009-10-09 2019-11-28 Armagen Inc Eljárások és készítmények a központi idegrendszerben iduronát-2-szulfatáz-aktivitás növelésére
CN102169121B (zh) * 2010-02-25 2013-12-04 北京诺赛基因组研究中心有限公司 人类激酶sbk1的新应用
JP6266529B2 (ja) 2011-12-02 2018-01-24 アーマジェン・インコーポレイテッドＡｒｍａｇｅｎ， Ｉｎｃ． Ｃｎｓにおけるアリールスルファターゼａ活性を増加するための方法および組成物
US10906981B2 (en) 2013-07-19 2021-02-02 The Regents Of The University Of California Compositions and methods related to structures that cross the blood brain barrier
US10538589B2 (en) 2015-01-14 2020-01-21 Armagen Inc. Methods and compositions for increasing N-acetylglucosaminidase (NAGLU) activity in the CNS using a fusion antibody comprising an anti-human insulin receptor antibody and NAGLU
PT3093022T (pt) 2015-05-15 2019-11-11 Sterna Biologicals Gmbh & Co Kg Inibidores gata-3 para utilização no tratamento de asma desencadeada por th2
CN114480406B (zh) * 2021-09-16 2024-01-30 广东翠点生物科技有限公司 一种il-1信号通路响应元件及其应用

Family Cites Families (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5994315A (en) * 1995-06-07 1999-11-30 East Carolina University Low adenosine agent, composition, kit and method for treatment of airway disease
AU2725600A (en) * 1999-01-13 2000-08-01 Du Pont Pharmaceuticals Company Antagonist blockade of crf2 receptors for the treatment of psy chiatric disorders and the use of chimeric antisense oligonucleotides in (in vivo) cnsstudies of gene function

Also Published As

Publication number Publication date
JP2002537792A (ja) 2002-11-12
EP1165093A4 (fr) 2002-07-24
WO2000051621A1 (fr) 2000-09-08
IL145034A0 (en) 2002-06-30
EP1165093A1 (fr) 2002-01-02
BR0009247A (pt) 2001-11-20
KR20020068262A (ko) 2002-08-27
CN1348376A (zh) 2002-05-08
AU3512300A (en) 2000-09-21
MXPA01008870A (es) 2004-08-12

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