BR0009247A - Method to validate / invalidate target (s) and pathways - Google Patents

Method to validate / invalidate target (s) and pathways

Info

Publication number
BR0009247A
BR0009247A BR0009247-9A BR0009247A BR0009247A BR 0009247 A BR0009247 A BR 0009247A BR 0009247 A BR0009247 A BR 0009247A BR 0009247 A BR0009247 A BR 0009247A
Authority
BR
Brazil
Prior art keywords
target
mrna
disease
condition
correlation
Prior art date
Application number
BR0009247-9A
Other languages
Portuguese (pt)
Inventor
Jonathan W Nyce
Original Assignee
Epigenesis Pharmaceuticals Inc
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 Epigenesis Pharmaceuticals Inc filed Critical Epigenesis Pharmaceuticals Inc
Publication of BR0009247A publication Critical patent/BR0009247A/en

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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
    • 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
    • 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)
  • Biomedical Technology (AREA)
  • Molecular Biology (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Health & Medical Sciences (AREA)
  • Biochemistry (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Analytical Chemistry (AREA)
  • Medicinal Chemistry (AREA)
  • Immunology (AREA)
  • Pathology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Epidemiology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (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

Patente de Invenção: "MéTODO PARA VALIDAR/INVALIDARALVO(S) E VIAS". Um método para determinar a existência de umacorrelação entre uma função de uma doença ou condição e umgene ou mRNA codificando um polipeptídio alvo suspeito de estarassociado com a doença ou condição, compreende obteroligonucleotídeos (oligos) consistindo em até cerca de 15% deadenosina (A), de preferência sem qualquer teor de adenosina, eque é anti-sentido em relação a um alvo selecionado do grupo queconsiste em genes alvo e seus mRNAs correspondentes, regiõesflanqueadoras genómicas e de mRNA selecionadas do grupo queconsiste nas bordas 3' e 5' intron-exon e da justasseção entreregiões codificadoras e não-codificadoras, e todos os segmentosde mRNA codificando polipeptídios associados com uma doençaou condição pré-selecionada; selecionar entre os oligos um queiniba ou elimine significativamente a expressão do polipeptídiocodificado pelo mRNA mediante hibridização "in vitro" no mRNAalvo; administrar a um indivíduo uma quantidade do oligoselecionado eficaz para hibridização 'in vivo' no mRNA alvo; eavaliar a função de um indivíduo que esteja associada com adoença ou condição antes e depois da administração do oligo;onde uma alteração maior que cerca de 70% no valor da funçãoindica uma correlação positiva; entre cerca de 40 e cerca de 70%,uma possível correlação; e abaixo de cerca de 30% uma falta decorrelação. O presente método de preferência administra oligos 'insitu' onde o alvo está localizado, por exemplo na respiração doindivíduo quando validando alvos associados com malignidade eoutras funções pulmonares e respiratórias, para que o agentetenha acesso direto aos pulmões. Alternativamente, estes oligosdesadenosina podem ser distribuídos diretamente ao SNC ououtros órgãos, tecidos e sistemas de órgãos, por meio deformulações de distribuição conhecidas.Invention Patent: "METHOD TO VALIDATE / INVALIDATE TARGET (S) AND ROUTES". A method for determining the existence of a correlation between a function of a disease or condition and umgene or mRNA encoding a target polypeptide suspected of being associated with the disease or condition, comprises obtaining oligonucleotides (oligos) consisting of up to about 15% deadenosine (A), preferably without any adenosine content, which is antisense towards a target selected from the group that consists of target genes and their corresponding mRNAs, genomic and mRNA flanking regions selected from the group that consists of the 3 'and 5' intron-exon edges and the justification between coding and non-coding regions, and all mRNA segments encoding polypeptides associated with a pre-selected disease or condition; select among the oligos one that significantly inhibits or eliminates the expression of the mRNA-encoded polypeptide by hybridizing "in vitro" to the target mRNA; administering to an individual an amount of the oligoselected effective for 'in vivo' hybridization to the target mRNA; and to evaluate the function of an individual that is associated with a disease or condition before and after the administration of the oligo, where a change greater than about 70% in the value of the function indicates a positive correlation; between about 40 and about 70%, a possible correlation; and below about 30% a lack of correlation. The present method preferably administers 'insitu' oligos where the target is located, for example in the individual's breathing when validating targets associated with malignancy and other pulmonary and respiratory functions, so that the agent has direct access to the lungs. Alternatively, these oligosdesadenosine can be delivered directly to the CNS or other organs, tissues and organ systems, through known distribution formulations.

BR0009247-9A 1999-03-05 2000-03-02 Method to validate / invalidate target (s) and pathways BR0009247A (en)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US12295099P 1999-03-05 1999-03-05
PCT/US2000/005643 WO2000051621A1 (en) 1999-03-05 2000-03-02 Method for validating/invalidating target(s) and pathways

Publications (1)

Publication Number Publication Date
BR0009247A true BR0009247A (en) 2001-11-20

Family

ID=22405857

Family Applications (1)

Application Number Title Priority Date Filing Date
BR0009247-9A BR0009247A (en) 1999-03-05 2000-03-02 Method to validate / invalidate target (s) and pathways

Country Status (10)

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

Families Citing this family (14)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP1255550A2 (en) 2000-02-17 2002-11-13 Cv Therapeutics, Inc. Method for identifying and using a 2b adenosine receptor antagonists to mediate mammalian cell proliferation
DE10346487A1 (en) 2003-10-02 2005-05-12 Transmit Technologietransfer Process for the preparation of a cell and / or tissue and / or disease phase specific drug
CN105385679B (en) 2006-02-13 2020-05-26 孟山都技术有限公司 Selecting and stabilizing dsRNA constructs
US8497246B2 (en) 2006-08-18 2013-07-30 Armagen Technologies, Inc. Methods for diagnosing and treating CNS disorders by trans-blood-brain barrier delivery of protein compositions
DE102007021443A1 (en) * 2007-05-08 2008-11-13 Brahms Aktiengesellschaft Diagnosis and risk stratification using NT-proET-1
JP5901877B2 (en) 2007-07-27 2016-04-13 アーメイゲン・テクノロジーズ・インコーポレイテッドＡｒｍａｇｅｎ Ｔｅｃｈｎｏｌｏｇｉｅｓ， Ｉｎｃ． Methods and compositions for increasing [alpha] -L-iduronidase activity in the central nervous system
JP5873003B2 (en) 2009-03-18 2016-03-01 アーメイゲン・テクノロジーズ・インコーポレイテッドＡｒｍａｇｅｎ Ｔｅｃｈｎｏｌｏｇｉｅｓ， Ｉｎｃ． Compositions and methods for blood brain barrier delivery of IgG decoy receptor fusion proteins
EP2485761B1 (en) 2009-10-09 2019-02-27 Armagen, Inc. Methods and compositions for increasing iduronate 2-sulfatase activity in the cns
CN102169121B (en) * 2010-02-25 2013-12-04 北京诺赛基因组研究中心有限公司 New application of human kinase SBK1 (SH3-binding domain kinase 1)
WO2013081706A1 (en) 2011-12-02 2013-06-06 Armagen Technologies, Inc. Methods and compositions for increasing arylsulfatase a activity in the cns
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 (en) 2015-05-15 2019-11-11 Sterna Biologicals Gmbh & Co Kg Gata-3 inhibitors for use in the treatment of th2-driven asthma
CN114480406B (en) * 2021-09-16 2024-01-30 广东翠点生物科技有限公司 IL-1 signal path response element and application thereof

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
CN1348376A (en) 2002-05-08
IL145034A0 (en) 2002-06-30
MXPA01008870A (en) 2004-08-12
AU3512300A (en) 2000-09-21
KR20020068262A (en) 2002-08-27
EP1165093A4 (en) 2002-07-24
CA2366055A1 (en) 2000-09-08
WO2000051621A1 (en) 2000-09-08
JP2002537792A (en) 2002-11-12
EP1165093A1 (en) 2002-01-02

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Legal Events

Date Code Title Description
B08F Application dismissed because of non-payment of annual fees [chapter 8.6 patent gazette]

Free format text: REFERENTE A 5A, 6A E 7A ANUIDADES.

B08K Patent lapsed as no evidence of payment of the annual fee has been furnished to inpi [chapter 8.11 patent gazette]

Free format text: REFERENTE AO DESPACHO 8.6 PUBLICADO NA RPI 1925 DE 27/11/2007.