BRPI0115814B8 - moléculas de rna de filamento duplo, seu método de preparação e composição farmacêutica compreendendo as mesmas - Google Patents

moléculas de rna de filamento duplo, seu método de preparação e composição farmacêutica compreendendo as mesmas

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Publication number
BRPI0115814B8
BRPI0115814B8 BRPI0115814A BR0115814A BRPI0115814B8 BR PI0115814 B8 BRPI0115814 B8 BR PI0115814B8 BR PI0115814 A BRPI0115814 A BR PI0115814A BR 0115814 A BR0115814 A BR 0115814A BR PI0115814 B8 BRPI0115814 B8 BR PI0115814B8
Authority
BR
Brazil
Prior art keywords
rna
double
rnai
dsrna
stranded rna
Prior art date
Application number
BRPI0115814A
Other languages
English (en)
Other versions
BR0115814A (pt
BRPI0115814B1 (pt
Inventor
Luehrmann Reinhard
Elbashir Sayda
Tuschl Thomas
Matthias Wilm
Lendeckel Winfried
Original Assignee
Europaeisches Laboratorium Fuer Molekularbiologie Embl
Max Planck Gesellschaft
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
Family has litigation
First worldwide family litigation filed litigation Critical https://patents.darts-ip.com/?family=40529293&utm_source=google_patent&utm_medium=platform_link&utm_campaign=public_patent_search&patent=BRPI0115814(B8) "Global patent litigation dataset” by Darts-ip is licensed under a Creative Commons Attribution 4.0 International License.
Priority claimed from PCT/US2001/010188 external-priority patent/WO2001075164A2/en
Application filed by Europaeisches Laboratorium Fuer Molekularbiologie Embl, Max Planck Gesellschaft filed Critical Europaeisches Laboratorium Fuer Molekularbiologie Embl
Publication of BR0115814A publication Critical patent/BR0115814A/pt
Publication of BRPI0115814B1 publication Critical patent/BRPI0115814B1/pt
Publication of BRPI0115814B8 publication Critical patent/BRPI0115814B8/pt

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    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
    • C07H21/00—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
    • C07H21/02—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids with ribosyl as saccharide radical
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • 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
    • A61K45/00—Medicinal preparations containing active ingredients not provided for in groups A61K31/00 - A61K41/00
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P31/00—Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
    • A61P31/12—Antivirals
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00—Antineoplastic agents
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P37/00—Drugs for immunological or allergic disorders
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P37/00—Drugs for immunological or allergic disorders
    • A61P37/02—Immunomodulators
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P37/00—Drugs for immunological or allergic disorders
    • A61P37/02—Immunomodulators
    • A61P37/06—Immunosuppressants, e.g. drugs for graft rejection
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
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    • 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
    • C12N15/10—Processes for the isolation, preparation or purification of DNA or RNA
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    • 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
    • C12N15/10—Processes for the isolation, preparation or purification of DNA or RNA
    • C12N15/1034—Isolating an individual clone by screening libraries
    • C12N15/1079—Screening libraries by altering the phenotype or phenotypic trait of the host
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    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • 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
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    • 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
    • C12N15/111—General methods applicable to biologically active non-coding nucleic acids
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    • 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
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2217/00—Genetically modified animals
    • A01K2217/07—Animals genetically altered by homologous recombination
    • A01K2217/075—Animals genetically altered by homologous recombination inducing loss of function, i.e. knock out
    • 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
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/0012—Galenical forms characterised by the site of application
    • A61K9/0019—Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/321—2'-O-R Modification
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/50—Physical structure
    • C12N2310/53—Physical structure partially self-complementary or closed
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2330/00—Production
    • C12N2330/30—Production chemically synthesised

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
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  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • Biomedical Technology (AREA)
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  • Biotechnology (AREA)
  • General Health & Medical Sciences (AREA)
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Abstract

"moléculas de rna pequenas mediando interferência de rna". a presente invenção refere-se a rna de filamento duplo (dsrna) que induz silenciamento do gene pós-transcripcional específico de seqüência em muitos organismos através de um processo conhecido como interferência de rna (rnai). usando um sistema in vitro de drosófila, foi demonstrado que fragmentos de rna curtos de 19-23 nt são os mediadores específicos de seqüência de rnai. os rnas de interferência curtos (sirnas) são gerados por uma reação de processamento do tipo rnase iii de dsrna longo. dúplexes de sirna quimicamente sintetizado com extremidades 3' se sobrepondo fazem a mediação eficiente da clivagem de rna alvo no lisato, e o sítio de clivagem está localizado próximo ao centro da região estendida pelo sirna guia. ainda, é provida evidência de que a direção do processamento de dsrna determina se rna alvo de sentido ou anti-sentido pode ser clivado pelo complexo de sirnp produzido.
BRPI0115814A 2000-12-01 2001-11-29 moléculas de rna de filamento duplo, seu método de preparação e composição farmacêutica compreendendo as mesmas BRPI0115814B8 (pt)

Applications Claiming Priority (7)

Application Number Priority Date Filing Date Title
EP00126325.0 2000-12-01
EP00126325 2000-12-01
US27966101P 2001-03-30 2001-03-30
US60/279,661 2001-03-30
USPCT/US01/00188 2001-03-30
PCT/US2001/010188 WO2001075164A2 (en) 2000-03-30 2001-03-30 Rna sequence-specific mediators of rna interference
PCT/EP2001/013968 WO2002044321A2 (en) 2000-12-01 2001-11-29 Rna interference mediating small rna molecules

Publications (3)

Publication Number Publication Date
BR0115814A BR0115814A (pt) 2004-03-23
BRPI0115814B1 BRPI0115814B1 (pt) 2019-10-15
BRPI0115814B8 true BRPI0115814B8 (pt) 2021-05-25

Family

ID=40529293

Family Applications (1)

Application Number Title Priority Date Filing Date
BRPI0115814A BRPI0115814B8 (pt) 2000-12-01 2001-11-29 moléculas de rna de filamento duplo, seu método de preparação e composição farmacêutica compreendendo as mesmas

Country Status (27)

Country Link
US (25) US20040259247A1 (pt)
EP (3) EP1873259B1 (pt)
JP (5) JP4095895B2 (pt)
KR (2) KR100872437B1 (pt)
CN (1) CN100523215C (pt)
AT (1) ATE373724T2 (pt)
AU (3) AU2002235744B8 (pt)
BR (1) BRPI0115814B8 (pt)
CA (1) CA2429814C (pt)
CY (1) CY1119062T1 (pt)
CZ (2) CZ308053B6 (pt)
DE (1) DE60130583T3 (pt)
DK (2) DK2813582T3 (pt)
ES (2) ES2215494T5 (pt)
HU (1) HU230458B1 (pt)
IL (3) IL155991A0 (pt)
LT (1) LTPA2021005I1 (pt)
MX (1) MXPA03004836A (pt)
NO (2) NO333713B1 (pt)
NZ (1) NZ525888A (pt)
PL (1) PL218876B1 (pt)
PT (1) PT1407044E (pt)
RU (2) RU2322500C2 (pt)
SI (1) SI1407044T2 (pt)
TR (1) TR200401292T3 (pt)
WO (1) WO2002044321A2 (pt)
ZA (1) ZA200303929B (pt)

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