WO2005003350A8 - RNA INTERFERENCE MEDIATED TREATMENT OF ALZHEIMER’S DISEASE USING SHORT INTERFERING NUCLEIC ACID (siNA) - Google Patents
RNA INTERFERENCE MEDIATED TREATMENT OF ALZHEIMER’S DISEASE USING SHORT INTERFERING NUCLEIC ACID (siNA)Info
- Publication number
- WO2005003350A8 WO2005003350A8 PCT/US2004/020516 US2004020516W WO2005003350A8 WO 2005003350 A8 WO2005003350 A8 WO 2005003350A8 US 2004020516 W US2004020516 W US 2004020516W WO 2005003350 A8 WO2005003350 A8 WO 2005003350A8
- Authority
- WO
- WIPO (PCT)
- Prior art keywords
- nucleic acid
- short interfering
- sina
- rna
- interfering nucleic
- Prior art date
Links
Classifications
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K49/00—Preparations for testing in vivo
- A61K49/0004—Screening or testing of compounds for diagnosis of disorders, assessment of conditions, e.g. renal clearance, gastric emptying, testing for diabetes, allergy, rheuma, pancreas functions
- A61K49/0008—Screening agents using (non-human) animal models or transgenic animal models or chimeric hosts, e.g. Alzheimer disease animal model, transgenic model for heart failure
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- 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
- C12N15/111—General methods applicable to biologically active non-coding nucleic acids
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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
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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
- C12N15/1131—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 viruses
- C12N15/1132—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 viruses against retroviridae, e.g. HIV
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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
- C12N15/1137—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 enzymes
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- 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
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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
- C12N15/115—Aptamers, i.e. nucleic acids binding a target molecule specifically and with high affinity without hybridising therewith ; Nucleic acids binding to non-nucleic acids, e.g. aptamers
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- 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
- C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12Y—ENZYMES
- C12Y103/00—Oxidoreductases acting on the CH-CH group of donors (1.3)
- C12Y103/01—Oxidoreductases acting on the CH-CH group of donors (1.3) with NAD+ or NADP+ as acceptor (1.3.1)
- C12Y103/01022—3-Oxo-5alpha-steroid 4-dehydrogenase (NADP+) (1.3.1.22), i.e. cortisone alpha-reductase
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12Y—ENZYMES
- C12Y104/00—Oxidoreductases acting on the CH-NH2 group of donors (1.4)
- C12Y104/03—Oxidoreductases acting on the CH-NH2 group of donors (1.4) with oxygen as acceptor (1.4.3)
- C12Y104/03003—D-Amino-acid oxidase (1.4.3.3)
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12Y—ENZYMES
- C12Y114/00—Oxidoreductases acting on paired donors, with incorporation or reduction of molecular oxygen (1.14)
- C12Y114/19—Oxidoreductases acting on paired donors, with incorporation or reduction of molecular oxygen (1.14) with oxidation of a pair of donors resulting in the reduction of molecular oxygen to two molecules of water (1.14.19)
- C12Y114/19001—Stearoyl-CoA 9-desaturase (1.14.19.1), i.e. DELTA9-desaturase
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12Y—ENZYMES
- C12Y207/00—Transferases transferring phosphorus-containing groups (2.7)
- C12Y207/07—Nucleotidyltransferases (2.7.7)
- C12Y207/07049—RNA-directed DNA polymerase (2.7.7.49), i.e. telomerase or reverse-transcriptase
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12Y—ENZYMES
- C12Y207/00—Transferases transferring phosphorus-containing groups (2.7)
- C12Y207/11—Protein-serine/threonine kinases (2.7.11)
- C12Y207/11001—Non-specific serine/threonine protein kinase (2.7.11.1), i.e. casein kinase or checkpoint kinase
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12Y—ENZYMES
- C12Y207/00—Transferases transferring phosphorus-containing groups (2.7)
- C12Y207/11—Protein-serine/threonine kinases (2.7.11)
- C12Y207/11013—Protein kinase C (2.7.11.13)
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12Y—ENZYMES
- C12Y301/00—Hydrolases acting on ester bonds (3.1)
- C12Y301/03—Phosphoric monoester hydrolases (3.1.3)
- C12Y301/03048—Protein-tyrosine-phosphatase (3.1.3.48)
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12Y—ENZYMES
- C12Y604/00—Ligases forming carbon-carbon bonds (6.4)
- C12Y604/01—Ligases forming carbon-carbon bonds (6.4.1)
- C12Y604/01002—Acetyl-CoA carboxylase (6.4.1.2)
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K38/00—Medicinal preparations containing peptides
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- 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
- C12N2310/10—Type of nucleic acid
- C12N2310/11—Antisense
- C12N2310/111—Antisense spanning the whole gene, or a large part of it
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- 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
- C12N2310/12—Type of nucleic acid catalytic nucleic acids, e.g. ribozymes
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- 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
- C12N2310/12—Type of nucleic acid catalytic nucleic acids, e.g. ribozymes
- C12N2310/121—Hammerhead
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- 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 N.A.
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/31—Chemical structure of the backbone
- C12N2310/315—Phosphorothioates
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- C12N2310/00—Structure or type of the nucleic acid
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- C12N2310/31—Chemical structure of the backbone
- C12N2310/317—Chemical structure of the backbone with an inverted bond, e.g. a cap structure
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/31—Chemical structure of the backbone
- C12N2310/318—Chemical structure of the backbone where the PO2 is completely replaced, e.g. MMI or formacetal
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- 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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- 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
- C12N2310/322—2'-R Modification
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- 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
- C12N2310/33—Chemical structure of the base
- C12N2310/332—Abasic residue
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- 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
- C12N2310/34—Spatial arrangement of the modifications
- C12N2310/346—Spatial arrangement of the modifications having a combination of backbone and sugar modifications
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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
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- C12N2320/00—Applications; Uses
- C12N2320/30—Special therapeutic applications
- C12N2320/32—Special delivery means, e.g. tissue-specific
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- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- C12N2330/00—Production
- C12N2330/30—Production chemically synthesised
Abstract
Priority Applications (3)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
CA002528963A CA2528963A1 (en) | 2003-06-27 | 2004-06-25 | Rna interference mediated treatment of alzheimer's disease using short interfering nucleic acid (sina) |
EP04756160A EP1644498A2 (en) | 2003-06-27 | 2004-06-25 | RNA INTERFERENCE MEDIATED TREATMENT OF ALZHEIMER'S DISEASE USING SHORT INTERFERING NUCLEIC ACID (siNA) |
US10/562,561 US20060247194A1 (en) | 2000-08-30 | 2004-06-25 | Rna interference mediated treatment of alzheimer's disease using short interfering nucleic acid (sina) |
Applications Claiming Priority (20)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
US10/607,933 | 2003-06-27 | ||
US10/607,933 US20080032942A1 (en) | 2000-08-30 | 2003-06-27 | RNA interference mediated treatment of Alzheimer's disease using short interfering nucleic acid (siNA) |
US10/693,059 US20080039414A1 (en) | 2002-02-20 | 2003-10-23 | RNA interference mediated inhibition of gene expression using chemically modified short interfering nucleic acid (siNA) |
US10/693,059 | 2003-10-23 | ||
US10/720,448 | 2003-11-24 | ||
US10/720,448 US8273866B2 (en) | 2002-02-20 | 2003-11-24 | RNA interference mediated inhibition of gene expression using chemically modified short interfering nucleic acid (SINA) |
US10/727,780 | 2003-12-03 | ||
US10/727,780 US20050233329A1 (en) | 2002-02-20 | 2003-12-03 | Inhibition of gene expression using duplex forming oligonucleotides |
US10/757,803 | 2004-01-14 | ||
US10/757,803 US20050020525A1 (en) | 2002-02-20 | 2004-01-14 | RNA interference mediated inhibition of gene expression using chemically modified short interfering nucleic acid (siNA) |
US54348004P | 2004-02-10 | 2004-02-10 | |
US60/543,480 | 2004-02-10 | ||
US10/780,447 US7491805B2 (en) | 2001-05-18 | 2004-02-13 | Conjugates and compositions for cellular delivery |
US10/780,447 | 2004-02-13 | ||
US10/826,966 US20050032733A1 (en) | 2001-05-18 | 2004-04-16 | RNA interference mediated inhibition of gene expression using chemically modified short interfering nucleic acid (SiNA) |
US10/826,966 | 2004-04-16 | ||
PCT/US2004/013456 WO2005041859A2 (en) | 2003-04-30 | 2004-04-30 | Conjugates and compositions for cellular delivery. |
USPCT/US04/13456 | 2004-04-30 | ||
PCT/US2004/016390 WO2005019453A2 (en) | 2001-05-18 | 2004-05-24 | RNA INTERFERENCE MEDIATED INHIBITION OF GENE EXPRESSION USING CHEMICALLY MODIFIED SHORT INTERFERING NUCLEIC ACID (siNA) |
USPCT/US04/16390 | 2004-05-24 |
Related Parent Applications (5)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
US10/693,059 Continuation-In-Part US20080039414A1 (en) | 2000-02-11 | 2003-10-23 | RNA interference mediated inhibition of gene expression using chemically modified short interfering nucleic acid (siNA) |
US10/720,448 Continuation-In-Part US8273866B2 (en) | 2000-02-11 | 2003-11-24 | RNA interference mediated inhibition of gene expression using chemically modified short interfering nucleic acid (SINA) |
US10/727,780 Continuation-In-Part US20050233329A1 (en) | 2000-02-11 | 2003-12-03 | Inhibition of gene expression using duplex forming oligonucleotides |
US10/757,803 Continuation-In-Part US20050020525A1 (en) | 2000-02-11 | 2004-01-14 | RNA interference mediated inhibition of gene expression using chemically modified short interfering nucleic acid (siNA) |
US10/826,966 Continuation-In-Part US20050032733A1 (en) | 2000-02-11 | 2004-04-16 | RNA interference mediated inhibition of gene expression using chemically modified short interfering nucleic acid (SiNA) |
Publications (3)
Publication Number | Publication Date |
---|---|
WO2005003350A2 WO2005003350A2 (en) | 2005-01-13 |
WO2005003350A8 true WO2005003350A8 (en) | 2005-05-19 |
WO2005003350A3 WO2005003350A3 (en) | 2005-08-04 |
Family
ID=36141761
Family Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
PCT/US2004/020516 WO2005003350A2 (en) | 2000-08-30 | 2004-06-25 | RNA INTERFERENCE MEDIATED TREATMENT OF ALZHEIMER’S DISEASE USING SHORT INTERFERING NUCLEIC ACID (siNA) |
Country Status (2)
Country | Link |
---|---|
CA (1) | CA2528963A1 (en) |
WO (1) | WO2005003350A2 (en) |
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US20030190635A1 (en) * | 2002-02-20 | 2003-10-09 | Mcswiggen James A. | RNA interference mediated treatment of Alzheimer's disease using short interfering RNA |
US20030175950A1 (en) * | 2001-05-29 | 2003-09-18 | Mcswiggen James A. | RNA interference mediated inhibition of HIV gene expression using short interfering RNA |
AU2003207708A1 (en) * | 2002-02-20 | 2003-09-09 | Sirna Therapeutics, Inc. | Rna interference mediated inhibition of map kinase genes |
JP2005517427A (en) * | 2002-02-20 | 2005-06-16 | サーナ・セラピューティクス・インコーポレイテッド | RNA interference-mediated inhibition of hepatitis C virus (HCV) gene expression using short interfering nucleic acids (siNA) |
US8680063B2 (en) | 2003-09-12 | 2014-03-25 | University Of Massachusetts | RNA interference for the treatment of gain-of-function disorders |
CA2579638C (en) | 2003-09-12 | 2016-04-19 | University Of Massachusetts | Rna interference targeting non-disease causing single nucleotide polymorphisms within a gene encoding a gain-of-function mutant huntingtin protein |
WO2005096781A2 (en) | 2004-04-06 | 2005-10-20 | University Of Massachusetts | Methods and compositions for treating gain-of-function disorders using rna interference |
JP2008538500A (en) * | 2005-04-22 | 2008-10-30 | アカデミス ツィーケンホイス ライデン | Regulation of exon recognition in pre-mRNA by interference with SR protein binding and RNA secondary structure |
US20070105803A1 (en) | 2005-08-18 | 2007-05-10 | Muthiah Manoharan | Methods and compositions for treating neurological disease |
GB0605337D0 (en) | 2006-03-17 | 2006-04-26 | Genomica Sau | Treatment of CNS conditions |
AU2007329539A1 (en) * | 2006-12-01 | 2008-06-12 | George Mason University | Inhibition of brain enzymes involved in cerebral amyloid angiopathy and macular degeneration |
WO2008129023A2 (en) * | 2007-04-19 | 2008-10-30 | Vib Vzw | Oligonucleotide compositions for the treatment of alzheimer's disease |
EP2432499A2 (en) | 2009-05-20 | 2012-03-28 | Schering Corporation | Modulation of pilr receptors to treat microbial infections |
WO2011031600A1 (en) | 2009-09-10 | 2011-03-17 | Schering Corporation | Use of il-33 antagonists to treat fibrotic disease |
EP2862929B1 (en) | 2009-12-09 | 2017-09-06 | Quark Pharmaceuticals, Inc. | Compositions and methods for treating diseases, disorders or injury of the CNS |
US8691227B2 (en) | 2009-12-17 | 2014-04-08 | Merck Sharp & Dohme Corp. | Methods of treating multiple sclerosis, rheumatoid arthritis and inflammatory bowel disease using agonists antibodies to PILR-α |
ES2657608T3 (en) | 2012-12-05 | 2018-03-06 | Alnylam Pharmaceuticals, Inc. | Compositions of pcsk9 arni and methods of use thereof |
EP3340994A1 (en) | 2015-08-25 | 2018-07-04 | Alnylam Pharmaceuticals, Inc. | Methods and compositions for treating a proprotein convertase subtilisin kexin (pcsk9) gene-associated disorder |
EP3759127A4 (en) | 2018-03-02 | 2022-03-30 | Ionis Pharmaceuticals, Inc. | Compounds and methods for the modulation of amyloid-beta precursor protein |
EP3898979A4 (en) * | 2018-12-19 | 2022-10-12 | Alnylam Pharmaceuticals, Inc. | Amyloid precursor protein (app) rnai agent compositions and methods of use thereof |
EP4096786A4 (en) * | 2020-01-31 | 2024-04-10 | Paros Bio Inc | Gene therapy for neurodegenerative disorders using polynucleotide silencing and replacement |
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CN1136327A (en) * | 1993-09-28 | 1996-11-20 | 综合医院公司 | Using antisense oligonucleotides to modulate nerve growth and to reverse 'beta'/A4 amyloid-induced morphology |
DE69514351T2 (en) * | 1994-06-01 | 2000-08-10 | Hybridon Inc | Branched oligonucleotides as pathogen-inhibiting agents |
DE19956568A1 (en) * | 1999-01-30 | 2000-08-17 | Roland Kreutzer | Method and medicament for inhibiting the expression of a given gene |
-
2004
- 2004-06-25 CA CA002528963A patent/CA2528963A1/en not_active Abandoned
- 2004-06-25 WO PCT/US2004/020516 patent/WO2005003350A2/en active Application Filing
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CA2528963A1 (en) | 2005-01-13 |
WO2005003350A2 (en) | 2005-01-13 |
WO2005003350A3 (en) | 2005-08-04 |
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