WO2003087367A3 - Means and methods for the specific inhibition of genes in cells and tissue of the cns and/or eye - Google Patents

Means and methods for the specific inhibition of genes in cells and tissue of the cns and/or eye Download PDF

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Publication number
WO2003087367A3
WO2003087367A3 PCT/EP2003/004002 EP0304002W WO03087367A3 WO 2003087367 A3 WO2003087367 A3 WO 2003087367A3 EP 0304002 W EP0304002 W EP 0304002W WO 03087367 A3 WO03087367 A3 WO 03087367A3
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WIPO (PCT)
Prior art keywords
cns
eye
tissue
methods
cells
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Application number
PCT/EP2003/004002
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French (fr)
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WO2003087367A2 (en
Inventor
Ralf Wilhelm Schulte
Frank Goehring
Original Assignee
Lynkeus Biotech Gmbh
Ralf Wilhelm Schulte
Frank Goehring
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.)
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Publication date
Application filed by Lynkeus Biotech Gmbh, Ralf Wilhelm Schulte, Frank Goehring filed Critical Lynkeus Biotech Gmbh
Priority to JP2003584308A priority Critical patent/JP5578388B2/en
Priority to US10/511,656 priority patent/US20050222061A1/en
Priority to AU2003224087A priority patent/AU2003224087B2/en
Priority to MXPA04010282A priority patent/MXPA04010282A/en
Priority to EP03720485A priority patent/EP1495121A2/en
Priority to CA002482903A priority patent/CA2482903A1/en
Publication of WO2003087367A2 publication Critical patent/WO2003087367A2/en
Publication of WO2003087367A3 publication Critical patent/WO2003087367A3/en
Priority to US12/828,624 priority patent/US20110021605A1/en

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    • 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
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    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/713—Double-stranded nucleic acids or oligonucleotides
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    • C12N15/09—Recombinant DNA-technology
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    • 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
    • G—PHYSICS
    • G01—MEASURING; TESTING
    • G01N—INVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
    • G01N33/00—Investigating or analysing materials by specific methods not covered by groups G01N1/00 - G01N31/00
    • G01N33/48—Biological material, e.g. blood, urine; Haemocytometers
    • G01N33/50—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing
    • G01N33/5005—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing involving human or animal cells
    • G01N33/5091—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing involving human or animal cells for testing the pathological state of an organism
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    • G01N—INVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
    • G01N33/00—Investigating or analysing materials by specific methods not covered by groups G01N1/00 - G01N31/00
    • G01N33/48—Biological material, e.g. blood, urine; Haemocytometers
    • G01N33/50—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing
    • G01N33/68—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing involving proteins, peptides or amino acids
    • G01N33/6893—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing involving proteins, peptides or amino acids related to diseases not provided for elsewhere
    • G—PHYSICS
    • G01—MEASURING; TESTING
    • G01N—INVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
    • G01N33/00—Investigating or analysing materials by specific methods not covered by groups G01N1/00 - G01N31/00
    • G01N33/48—Biological material, e.g. blood, urine; Haemocytometers
    • G01N33/50—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing
    • G01N33/68—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing involving proteins, peptides or amino acids
    • G01N33/6893—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing involving proteins, peptides or amino acids related to diseases not provided for elsewhere
    • G01N33/6896—Neurological disorders, e.g. Alzheimer's disease
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; CARE OF BIRDS, FISHES, INSECTS; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2217/00—Genetically modified animals
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; CARE OF BIRDS, FISHES, INSECTS; 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
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/14—Type of nucleic acid interfering N.A.
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    • C12N2320/00—Applications; Uses
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    • G01N2800/00—Detection or diagnosis of diseases
    • G01N2800/16—Ophthalmology
    • G—PHYSICS
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    • G01N—INVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
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    • G01N2800/28—Neurological disorders

Abstract

Described is a method for the specific modulation of the expression of target genes in cells and/or tissues of the CNS and/or eye, wherein a composition comprising one or more doubled stranded oligoribonucleotides (dsRNA) is introduced into the cell, tissue or organism outside the blood-brain or blood-retina barriers. Furthermore, a method for the identification and validation of the function of a gene is provided, wherein the method provides a test cell, test tissue or test organism, which allow information to be gained on the function of the target gene. In addition, compositions and kits are described useful for those methods. In particular, components and methods for the diagnostic use and/or therapy of disorders related to the CNS and/or eye are provided which are based on RNA interference.
PCT/EP2003/004002 2002-04-18 2003-04-16 Means and methods for the specific inhibition of genes in cells and tissue of the cns and/or eye WO2003087367A2 (en)

Priority Applications (7)

Application Number Priority Date Filing Date Title
JP2003584308A JP5578388B2 (en) 2002-04-18 2003-04-16 Means and methods for specific inhibition of genes in the central nervous system and / or cells and tissues of the eye
US10/511,656 US20050222061A1 (en) 2002-04-18 2003-04-16 Means and methods for the specific inhibition of genes in cells and tissue of the cns and/or eye
AU2003224087A AU2003224087B2 (en) 2002-04-18 2003-04-16 Means and methods for the specific inhibition of genes in cells and tissue of the CNS and/or eye
MXPA04010282A MXPA04010282A (en) 2002-04-18 2003-04-16 Means and methods for the specific inhibition of genes in cells and tissue of the cns and/or eye.
EP03720485A EP1495121A2 (en) 2002-04-18 2003-04-16 Means and methods for the specific inhibition of genes in cells and tissue of the cns and/or eye
CA002482903A CA2482903A1 (en) 2002-04-18 2003-04-16 Means and methods for the specific inhibition of genes in cells and tissue of the cns and/or eye
US12/828,624 US20110021605A1 (en) 2002-04-18 2010-07-01 Means and methods for the specific inhibition of genes in cells and tissue of the cns and/or eye

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
EP02008761 2002-04-18
EP02008761.5 2002-04-18
US43117302P 2002-12-05 2002-12-05
US60/431,173 2002-12-05

Related Child Applications (1)

Application Number Title Priority Date Filing Date
US12/828,624 Continuation US20110021605A1 (en) 2002-04-18 2010-07-01 Means and methods for the specific inhibition of genes in cells and tissue of the cns and/or eye

Publications (2)

Publication Number Publication Date
WO2003087367A2 WO2003087367A2 (en) 2003-10-23
WO2003087367A3 true WO2003087367A3 (en) 2004-04-29

Family

ID=47711411

Family Applications (2)

Application Number Title Priority Date Filing Date
PCT/EP2003/004002 WO2003087367A2 (en) 2002-04-18 2003-04-16 Means and methods for the specific inhibition of genes in cells and tissue of the cns and/or eye
PCT/EP2003/004003 WO2003087368A2 (en) 2002-04-18 2003-04-16 Means and methods for the specific modulation of target genes in the cns and the eye and methods for their identification

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Application Number Title Priority Date Filing Date
PCT/EP2003/004003 WO2003087368A2 (en) 2002-04-18 2003-04-16 Means and methods for the specific modulation of target genes in the cns and the eye and methods for their identification

Country Status (9)

Country Link
US (5) US8202845B2 (en)
EP (2) EP1495121A2 (en)
JP (2) JP5578388B2 (en)
AU (3) AU2003222820A1 (en)
CA (2) CA2482904A1 (en)
ES (1) ES2397060T3 (en)
HK (1) HK1072074A1 (en)
MX (2) MXPA04010283A (en)
WO (2) WO2003087367A2 (en)

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