IL166546A0 - Further novel forms of interfering ran molecules - Google Patents

Further novel forms of interfering ran molecules

Info

Publication number
IL166546A0
IL166546A0 IL16654603A IL16654603A IL166546A0 IL 166546 A0 IL166546 A0 IL 166546A0 IL 16654603 A IL16654603 A IL 16654603A IL 16654603 A IL16654603 A IL 16654603A IL 166546 A0 IL166546 A0 IL 166546A0
Authority
IL
Israel
Prior art keywords
ran
interfering
molecules
stretch
further novel
Prior art date
Application number
IL16654603A
Other languages
English (en)
Original Assignee
Atugen Ag
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=31720998&utm_source=google_patent&utm_medium=platform_link&utm_campaign=public_patent_search&patent=IL166546(A0) "Global patent litigation dataset” by Darts-ip is licensed under a Creative Commons Attribution 4.0 International License.
Priority claimed from EP02017601A external-priority patent/EP1389637B1/de
Application filed by Atugen Ag filed Critical Atugen Ag
Publication of IL166546A0 publication Critical patent/IL166546A0/xx

Links

Classifications

    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/70Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088Compounds having three or more nucleosides or nucleotides
    • A61K31/713Double-stranded nucleic acids or oligonucleotides
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P1/00Drugs for disorders of the alimentary tract or the digestive system
    • A61P1/04Drugs for disorders of the alimentary tract or the digestive system for ulcers, gastritis or reflux esophagitis, e.g. antacids, inhibitors of acid secretion, mucosal protectants
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P1/00Drugs for disorders of the alimentary tract or the digestive system
    • A61P1/16Drugs for disorders of the alimentary tract or the digestive system for liver or gallbladder disorders, e.g. hepatoprotective agents, cholagogues, litholytics
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P1/00Drugs for disorders of the alimentary tract or the digestive system
    • A61P1/18Drugs for disorders of the alimentary tract or the digestive system for pancreatic disorders, e.g. pancreatic enzymes
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P13/00Drugs for disorders of the urinary system
    • A61P13/08Drugs for disorders of the urinary system of the prostate
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P15/00Drugs for genital or sexual disorders; Contraceptives
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P17/00Drugs for dermatological disorders
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P29/00Non-central analgesic, antipyretic or antiinflammatory agents, e.g. antirheumatic agents; Non-steroidal antiinflammatory drugs [NSAID]
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00Drugs for disorders of the metabolism
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00Drugs for disorders of the metabolism
    • A61P3/04Anorexiants; Antiobesity agents
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00Drugs for disorders of the metabolism
    • A61P3/08Drugs for disorders of the metabolism for glucose homeostasis
    • A61P3/10Drugs for disorders of the metabolism for glucose homeostasis for hyperglycaemia, e.g. antidiabetics
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00Antineoplastic agents
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00Antineoplastic agents
    • A61P35/02Antineoplastic agents specific for leukemia
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P37/00Drugs for immunological or allergic disorders
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00Drugs for disorders of the cardiovascular system
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00Drugs for disorders of the cardiovascular system
    • A61P9/10Drugs for disorders of the cardiovascular system for treating ischaemic or atherosclerotic diseases, e.g. antianginal drugs, coronary vasodilators, drugs for myocardial infarction, retinopathy, cerebrovascula insufficiency, renal arteriosclerosis
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/111General methods applicable to biologically active non-coding nucleic acids
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113Non-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
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113Non-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
    • C12N15/1137Non-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
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N5/00Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
    • C12N5/10Cells modified by introduction of foreign genetic material
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12QMEASURING 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
    • C12Q1/00Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions
    • C12Q1/68Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions involving nucleic acids
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12YENZYMES
    • C12Y207/00Transferases transferring phosphorus-containing groups (2.7)
    • C12Y207/11Protein-serine/threonine kinases (2.7.11)
    • C12Y207/11001Non-specific serine/threonine protein kinase (2.7.11.1), i.e. casein kinase or checkpoint kinase
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12YENZYMES
    • C12Y301/00Hydrolases acting on ester bonds (3.1)
    • C12Y301/03Phosphoric monoester hydrolases (3.1.3)
    • C12Y301/03048Protein-tyrosine-phosphatase (3.1.3.48)
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00Medicinal preparations containing peptides
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/13Decoys
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/14Type of nucleic acid interfering nucleic acids [NA]
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/31Chemical structure of the backbone
    • C12N2310/317Chemical structure of the backbone with an inverted bond, e.g. a cap structure
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/31Chemical structure of the backbone
    • C12N2310/318Chemical structure of the backbone where the PO2 is completely replaced, e.g. MMI or formacetal
    • C12N2310/3183Diol linkers, e.g. glycols or propanediols
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/32Chemical structure of the sugar
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/32Chemical structure of the sugar
    • C12N2310/3212'-O-R Modification
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/32Chemical structure of the sugar
    • C12N2310/3222'-R Modification
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/33Chemical structure of the base
    • C12N2310/332Abasic residue
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/34Spatial arrangement of the modifications
    • C12N2310/343Spatial arrangement of the modifications having patterns, e.g. ==--==--==--
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/34Spatial arrangement of the modifications
    • C12N2310/346Spatial arrangement of the modifications having a combination of backbone and sugar modifications
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/35Nature of the modification
    • C12N2310/352Nature of the modification linked to the nucleic acid via a carbon atom
    • C12N2310/3521Methyl
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/35Nature of the modification
    • C12N2310/352Nature of the modification linked to the nucleic acid via a carbon atom
    • C12N2310/3525MOE, methoxyethoxy
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/50Physical structure
    • C12N2310/53Physical structure partially self-complementary or closed
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/50Physical structure
    • C12N2310/53Physical structure partially self-complementary or closed
    • C12N2310/531Stem-loop; Hairpin
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2320/00Applications; Uses
    • C12N2320/50Methods for regulating/modulating their activity
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2320/00Applications; Uses
    • C12N2320/50Methods for regulating/modulating their activity
    • C12N2320/51Methods for regulating/modulating their activity modulating the chemical stability, e.g. nuclease-resistance

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Genetics & Genomics (AREA)
  • General Health & Medical Sciences (AREA)
  • Biomedical Technology (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Medicinal Chemistry (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Molecular Biology (AREA)
  • General Engineering & Computer Science (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Biotechnology (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Diabetes (AREA)
  • Hematology (AREA)
  • Epidemiology (AREA)
  • Obesity (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Virology (AREA)
  • Immunology (AREA)
  • Heart & Thoracic Surgery (AREA)
  • Endocrinology (AREA)
  • Cardiology (AREA)
  • Urology & Nephrology (AREA)
  • Analytical Chemistry (AREA)
  • Cell Biology (AREA)
IL16654603A 2002-08-05 2003-08-05 Further novel forms of interfering ran molecules IL166546A0 (en)

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
EP02017601A EP1389637B1 (de) 2002-08-05 2002-08-05 Interferierende RNS Moleküle mit glatten Enden
US40254102P 2002-08-12 2002-08-12
EP03008383 2003-04-10
PCT/EP2003/008666 WO2004015107A2 (en) 2002-08-05 2003-08-05 Further novel forms of interfering rna molecules

Publications (1)

Publication Number Publication Date
IL166546A0 true IL166546A0 (en) 2006-01-15

Family

ID=31720998

Family Applications (2)

Application Number Title Priority Date Filing Date
IL16654603A IL166546A0 (en) 2002-08-05 2003-08-05 Further novel forms of interfering ran molecules
IL16654605A IL166546A (en) 2002-08-05 2005-01-27 Double-stranded ribonucleic acid, preparations containing it, is used in drug preparation and in vitro method to inhibit the expression of a target gene

Family Applications After (1)

Application Number Title Priority Date Filing Date
IL16654605A IL166546A (en) 2002-08-05 2005-01-27 Double-stranded ribonucleic acid, preparations containing it, is used in drug preparation and in vitro method to inhibit the expression of a target gene

Country Status (19)

Country Link
US (15) US7452987B2 (de)
EP (6) EP2258847B2 (de)
JP (4) JP5490655B2 (de)
KR (1) KR101201664B1 (de)
CN (1) CN100543137C (de)
AT (1) ATE350473T2 (de)
AU (2) AU2003260370B2 (de)
BR (1) BRPI0313202A8 (de)
CA (1) CA2494930C (de)
CY (4) CY1106422T1 (de)
DE (1) DE60310944T3 (de)
DK (4) DK1857547T3 (de)
ES (1) ES2280826T5 (de)
IL (2) IL166546A0 (de)
MX (1) MXPA05001355A (de)
PT (1) PT1527176E (de)
SI (3) SI2258847T1 (de)
WO (1) WO2004015107A2 (de)
ZA (1) ZA200500459B (de)

Families Citing this family (268)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20080119427A1 (en) * 1996-06-06 2008-05-22 Isis Pharmaceuticals, Inc. Double Strand Compositions Comprising Differentially Modified Strands for Use in Gene Modulation
US5898031A (en) 1996-06-06 1999-04-27 Isis Pharmaceuticals, Inc. Oligoribonucleotides for cleaving RNA
US9096636B2 (en) 1996-06-06 2015-08-04 Isis Pharmaceuticals, Inc. Chimeric oligomeric compounds and their use in gene modulation
US7812149B2 (en) * 1996-06-06 2010-10-12 Isis Pharmaceuticals, Inc. 2′-Fluoro substituted oligomeric compounds and compositions for use in gene modulations
DE19956568A1 (de) 1999-01-30 2000-08-17 Roland Kreutzer Verfahren und Medikament zur Hemmung der Expression eines vorgegebenen Gens
DE10100586C1 (de) 2001-01-09 2002-04-11 Ribopharma Ag Verfahren zur Hemmung der Expression eines Ziegens
US20050020525A1 (en) * 2002-02-20 2005-01-27 Sirna Therapeutics, Inc. RNA interference mediated inhibition of gene expression using chemically modified short interfering nucleic acid (siNA)
US8202979B2 (en) * 2002-02-20 2012-06-19 Sirna Therapeutics, Inc. RNA interference mediated inhibition of gene expression using chemically modified short interfering nucleic acid
US8273866B2 (en) 2002-02-20 2012-09-25 Merck Sharp & Dohme Corp. RNA interference mediated inhibition of gene expression using chemically modified short interfering nucleic acid (SINA)
ATE450621T2 (de) 2000-03-30 2009-12-15 Whitehead Biomedical Inst Mediatoren von rns-interferenz, die rns- sequenzspezifisch sind
WO2002038738A2 (en) * 2000-11-09 2002-05-16 Cold Spring Harbor Laboratory Chimeric molecules to modulate gene expression
KR100872437B1 (ko) 2000-12-01 2008-12-05 막스-플랑크-게젤샤프트 츄어 푀르더룽 데어 비쎈샤프텐 에.파우. Rna 간섭을 매개하는 작은 rna 분자
US7767802B2 (en) 2001-01-09 2010-08-03 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of anti-apoptotic genes
US20050282188A1 (en) * 2001-05-18 2005-12-22 Sirna Therapeutics, Inc. RNA interference mediated inhibition of gene expression using short interfering nucleic acid (siNA)
WO2003064621A2 (en) 2002-02-01 2003-08-07 Ambion, Inc. HIGH POTENCY siRNAS FOR REDUCING THE EXPRESSION OF TARGET GENES
EP3415625A1 (de) 2002-02-01 2018-12-19 Life Technologies Corporation Doppelsträngige oligonukleotide
US20060009409A1 (en) 2002-02-01 2006-01-12 Woolf Tod M Double-stranded oligonucleotides
US7910724B2 (en) * 2002-02-20 2011-03-22 Sirna Therapeutics, Inc. RNA interference mediated inhibition of Fos gene expression using short interfering nucleic acid (siNA)
US8232383B2 (en) * 2002-02-20 2012-07-31 Sirna Therapeutics, Inc. RNA interference mediated inhibition of gene expression using chemically modified short interfering nucleic acid (siNA)
AU2003207708A1 (en) 2002-02-20 2003-09-09 Sirna Therapeutics, Inc. Rna interference mediated inhibition of map kinase genes
US20100240730A1 (en) * 2002-02-20 2010-09-23 Merck Sharp And Dohme Corp. RNA Interference Mediated Inhibition of Gene Expression Using Chemically Modified Short Interfering Nucleic Acid (siNA)
US9657294B2 (en) 2002-02-20 2017-05-23 Sirna Therapeutics, Inc. RNA interference mediated inhibition of gene expression using chemically modified short interfering nucleic acid (siNA)
US20090093439A1 (en) * 2002-02-20 2009-04-09 Sirna Therapeutics, Inc. RNA INTERFERENCE MEDIATED INHIBITION OF CHROMOSOME TRANSLOCATION GENE EXPRESSION USING SHORT INTERFERING NUCLEIC ACID (siNA)
US7928218B2 (en) * 2002-02-20 2011-04-19 Merck Sharp & Dohme Corp. RNA interference mediated inhibition of polycomb group protein EZH2 gene expression using short interfering nucleic acid (siNA)
US20090099117A1 (en) * 2002-02-20 2009-04-16 Sirna Therapeutics, Inc. RNA INTERFERENCE MEDIATED INHIBITION OF MYOSTATIN GENE EXPRESSION USING SHORT INTERFERING NUCLEIC ACID (siNA)
US9181551B2 (en) 2002-02-20 2015-11-10 Sirna Therapeutics, Inc. RNA interference mediated inhibition of gene expression using chemically modified short interfering nucleic acid (siNA)
ES2280826T5 (es) 2002-08-05 2017-08-03 Silence Therapeutics Gmbh Nuevas formas adicionales de moléculas de ARN de interferencia
WO2004014933A1 (en) * 2002-08-07 2004-02-19 University Of Massachusetts Compositions for rna interference and methods of use thereof
US7923547B2 (en) * 2002-09-05 2011-04-12 Sirna Therapeutics, Inc. RNA interference mediated inhibition of gene expression using chemically modified short interfering nucleic acid (siNA)
WO2004027030A2 (en) * 2002-09-18 2004-04-01 Isis Pharmaceuticals, Inc. Efficient reduction of target rna’s by single- and double-stranded oligomeric compounds
US7892793B2 (en) 2002-11-04 2011-02-22 University Of Massachusetts Allele-specific RNA interference
US9150606B2 (en) * 2002-11-05 2015-10-06 Isis Pharmaceuticals, Inc. Compositions comprising alternating 2'-modified nucleosides for use in gene modulation
US9150605B2 (en) * 2002-11-05 2015-10-06 Isis Pharmaceuticals, Inc. Compositions comprising alternating 2′-modified nucleosides for use in gene modulation
WO2004043979A2 (en) * 2002-11-05 2004-05-27 Isis Pharmaceuticals, Inc. Sugar surrogate-containing oligomeric compounds and compositions for use in gene modulation
US9827263B2 (en) * 2002-11-05 2017-11-28 Ionis Pharmaceuticals, Inc. 2′-methoxy substituted oligomeric compounds and compositions for use in gene modulations
US8653252B2 (en) 2003-03-21 2014-02-18 Santaris Pharma A/S Short interfering RNA (siRNA) analogues
ATE536408T1 (de) * 2003-04-02 2011-12-15 Dharmacon Inc Modifizierte polynukleotide zur verwendung bei rna-interferenz
AU2004252442B2 (en) 2003-06-02 2010-04-08 University Of Massachusetts Methods and compositions for enhancing the efficacy and specificity of FNAi
ES2712695T3 (es) 2003-06-02 2019-05-14 Univ Massachusetts Métodos y composiciones para controlar la eficacia de la silenciación del ARN
US7750144B2 (en) 2003-06-02 2010-07-06 University Of Massachusetts Methods and compositions for enhancing the efficacy and specificity of RNA silencing
US8575327B2 (en) 2003-06-12 2013-11-05 Alnylam Pharmaceuticals, Inc. Conserved HBV and HCV sequences useful for gene silencing
WO2004113496A2 (en) * 2003-06-20 2004-12-29 Isis Pharmaceuticals, Inc. Double stranded compositions comprising a 3’-endo modified strand for use in gene modulation
EP2530157B1 (de) * 2003-07-31 2016-09-28 Regulus Therapeutics Inc. Oligomerverbindungen und Zusammensetzungen zur Verwendung bei der Modulation von miRNAs
US20050136437A1 (en) * 2003-08-25 2005-06-23 Nastech Pharmaceutical Company Inc. Nanoparticles for delivery of nucleic acids and stable double-stranded RNA
CA2536333C (en) * 2003-08-28 2013-01-08 Jan Weiler Interfering rna duplex having blunt-ends and 3'-modifications
US20050074801A1 (en) * 2003-09-09 2005-04-07 Monia Brett P. Chimeric oligomeric compounds comprising alternating regions of northern and southern conformational geometry
US8680063B2 (en) 2003-09-12 2014-03-25 University Of Massachusetts RNA interference for the treatment of gain-of-function disorders
WO2005027980A1 (en) 2003-09-12 2005-03-31 University Of Massachusetts Rna interference for the treatment of gain-of-function disorders
US20060134787A1 (en) 2004-12-22 2006-06-22 University Of Massachusetts Methods and compositions for enhancing the efficacy and specificity of single and double blunt-ended siRNA
EP2330111A3 (de) 2004-01-30 2011-08-17 Quark Pharmaceuticals, Inc. Oligoribonucleotide und Verfahren zu ihrer Verwendung zur Behandlung fibrotischer und anderer Erkrankungen
US20080249039A1 (en) * 2004-01-30 2008-10-09 Santaris Pharma A/S Modified Short Interfering Rna (Modified Sirna)
US8569474B2 (en) 2004-03-09 2013-10-29 Isis Pharmaceuticals, Inc. Double stranded constructs comprising one or more short strands hybridized to a longer strand
US20070265220A1 (en) 2004-03-15 2007-11-15 City Of Hope Methods and compositions for the specific inhibition of gene expression by double-stranded RNA
KR101147147B1 (ko) 2004-04-01 2012-05-25 머크 샤프 앤드 돔 코포레이션 Rna 간섭의 오프 타겟 효과 감소를 위한 변형된폴리뉴클레오타이드
US20160230185A1 (en) 2013-03-14 2016-08-11 Monsanto Technology Llc Compositions and methods for controlling diabrotica
EP2290073A3 (de) 2004-05-28 2011-08-31 Asuragen, Inc. Verfahren und Zusammensetzungen mit MicroRNA
US8394947B2 (en) 2004-06-03 2013-03-12 Isis Pharmaceuticals, Inc. Positionally modified siRNA constructs
US20090048192A1 (en) * 2004-06-03 2009-02-19 Isis Pharmaceuticals, Inc. Double Strand Compositions Comprising Differentially Modified Strands for Use in Gene Modulation
CA2569036A1 (en) * 2004-06-03 2005-12-22 Balkrishen Bhat Chimeric gapped oligomeric compositions
JP4468989B2 (ja) 2004-08-16 2010-05-26 クアーク・ファーマスーティカルス、インコーポレイテッド Rtp801阻害剤の治療への使用
HUE033977T2 (en) 2004-08-23 2018-02-28 Sylentis Sau Treatment of eye disorders with siRNAs characterized by increased intraocular pressure
US7884086B2 (en) 2004-09-08 2011-02-08 Isis Pharmaceuticals, Inc. Conjugates for use in hepatocyte free uptake assays
AU2005288522B2 (en) 2004-09-28 2012-06-28 Quark Pharmaceuticals, Inc. Oligoribonucleotides and methods of use thereof for treatment of alopecia, acute renal failure and other diseases
CA2857878A1 (en) 2004-11-12 2006-12-28 Asuragen, Inc. Methods and compositions involving mirna and mirna inhibitor molecules
US7923207B2 (en) 2004-11-22 2011-04-12 Dharmacon, Inc. Apparatus and system having dry gene silencing pools
US7935811B2 (en) 2004-11-22 2011-05-03 Dharmacon, Inc. Apparatus and system having dry gene silencing compositions
US20060166234A1 (en) 2004-11-22 2006-07-27 Barbara Robertson Apparatus and system having dry control gene silencing compositions
WO2006060454A2 (en) * 2004-12-02 2006-06-08 B-Bridge International, Inc. Methods of designing small interfering rnas, antisense polynucleotides, and other hybridizing polynucleotides
WO2006063356A1 (en) * 2004-12-10 2006-06-15 Isis Phamaceuticals, Inc. Regulation of epigenetic control of gene expression
US20100266574A1 (en) * 2005-06-10 2010-10-21 Orna Mor Oligoribonucleotides and Methods of Use Thereof for Treatment of Fibrotic Conditions and Other Diseases
JP2009504782A (ja) 2005-08-18 2009-02-05 アルナイラム ファーマシューティカルズ インコーポレイテッド 神経疾患を治療するための方法および組成物
GB0520063D0 (en) * 2005-10-03 2005-11-09 Novartis Forschungsstiftung Methods for regulating metabolism and diagnosing disease
GB0521351D0 (en) 2005-10-20 2005-11-30 Genomica Sau Modulation of TRPV expression levels
GB0521716D0 (en) 2005-10-25 2005-11-30 Genomica Sau Modulation of 11beta-hydroxysteriod dehydrogenase 1 expression for the treatment of ocular diseases
WO2007056153A2 (en) * 2005-11-04 2007-05-18 Nastech Pharmaceutical Company Inc. Peptide-dicer substrate rna conjugates as delivery vehicles for sirna
JP5232990B2 (ja) * 2005-11-08 2013-07-10 国立大学法人愛媛大学 Akt遺伝子に特異的なsiRNA
KR100877824B1 (ko) 2005-11-11 2009-01-12 한국생명공학연구원 E2epf ucp-vhl 상호작용 및 그 용도
UY30097A1 (es) * 2006-01-20 2007-08-31 Atugen Ag Usos terapeuticos de inhibidores de rtp801
US7825099B2 (en) 2006-01-20 2010-11-02 Quark Pharmaceuticals, Inc. Treatment or prevention of oto-pathologies by inhibition of pro-apoptotic genes
DK1984382T3 (da) * 2006-01-27 2012-09-03 Santaris Pharma As LNA modificerede fosforthiolerede oligonukleotider
US8129515B2 (en) * 2006-01-27 2012-03-06 Isis Pharmaceuticals, Inc. Oligomeric compounds and compositions for the use in modulation of microRNAs
WO2007091269A2 (en) * 2006-02-08 2007-08-16 Quark Pharmaceuticals, Inc. NOVEL TANDEM siRNAS
US7910566B2 (en) * 2006-03-09 2011-03-22 Quark Pharmaceuticals Inc. Prevention and treatment of acute renal failure and other kidney diseases by inhibition of p53 by siRNA
AU2007229161B2 (en) * 2006-03-23 2012-07-12 Roche Innovation Center Copenhagen A/S Small internally segmented interfering RNA
FR2898908A1 (fr) 2006-03-24 2007-09-28 Agronomique Inst Nat Rech Procede de preparation de cellules aviaires differenciees et genes impliques dans le maintien de la pluripotence
GB0608838D0 (en) 2006-05-04 2006-06-14 Novartis Ag Organic compounds
WO2007141796A2 (en) 2006-06-09 2007-12-13 Quark Pharmaceuticals, Inc. Therapeutic uses of inhibitors of rtp801l
RU2553561C2 (ru) 2006-07-21 2015-06-20 Сайленс Терапьютикс Аг Средства ингибирования экспрессии протеинкиназы 3
EP1897941A1 (de) * 2006-09-11 2008-03-12 Max-Delbrück-Centrum Für Molekulare Medizin Mittel zur Erhöhung der intrazellulären Protoporphyrin IX Spiegel und dessen Verwendung
CA2663878A1 (en) * 2006-09-19 2008-03-27 Asuragen, Inc. Mir-200 regulated genes and pathways as targets for therapeutic intervention
US8809514B2 (en) 2006-09-22 2014-08-19 Ge Healthcare Dharmacon, Inc. Tripartite oligonucleotide complexes and methods for gene silencing by RNA interference
JP2010507387A (ja) * 2006-10-25 2010-03-11 クアーク・ファーマスーティカルス、インコーポレイテッド 新規のsiRNAおよびその使用方法
EP2061901A2 (de) 2006-10-31 2009-05-27 Noxxon Pharma AG Verfahren zum nachweis eines einzel- oder doppelsträngigen nukleinsäuremoleküls
EP2137205A2 (de) * 2007-02-26 2009-12-30 Quark Pharmaceuticals, Inc. Inhibitoren von rtp801 und ihre verwendung bei der behandlung von krankheiten
WO2008104978A2 (en) * 2007-02-28 2008-09-04 Quark Pharmaceuticals, Inc. Novel sirna structures
US7812002B2 (en) * 2007-03-21 2010-10-12 Quark Pharmaceuticals, Inc. Oligoribonucleotide inhibitors of NRF2 and methods of use thereof for treatment of cancer
WO2008143774A2 (en) * 2007-05-01 2008-11-27 University Of Massachusetts Methods and compositions for locating snp heterozygosity for allele specific diagnosis and therapy
NZ581201A (en) * 2007-05-11 2012-05-25 Enzon Pharmaceuticals Inc Rna antagonist compounds for the modulation of her3
US20100273854A1 (en) * 2007-06-15 2010-10-28 Hagar Kalinski Compositions and methods for inhibiting nadph oxidase expression
HRP20140522T1 (hr) 2007-06-27 2014-08-15 Quark Pharmaceuticals, Inc. Pripravci i metode za inhibiranje izražaja proapoptotiäśkih gena
EP2198050A1 (de) 2007-09-14 2010-06-23 Asuragen, INC. Mikrornas mit unterschiedlicher expression bei zervikalkarzinom und verwendungen davon
CN101849008A (zh) 2007-09-19 2010-09-29 应用生物系统有限公司 用于减少RNAi中的脱靶表型效应的siRNA的不依赖于序列的修饰形式和其稳定形式
CN103898110A (zh) * 2007-10-03 2014-07-02 夸克制药公司 新siRNA结构
US20100317714A1 (en) * 2007-11-29 2010-12-16 Suzhou Ribo Life Science Co., Ltd Complex molecule interfering the expression of target genes and its preparing methods
WO2009070805A2 (en) 2007-12-01 2009-06-04 Asuragen, Inc. Mir-124 regulated genes and pathways as targets for therapeutic intervention
US20110105584A1 (en) * 2007-12-12 2011-05-05 Elena Feinstein Rtp80il sirna compounds and methods of use thereof
US8614311B2 (en) 2007-12-12 2013-12-24 Quark Pharmaceuticals, Inc. RTP801L siRNA compounds and methods of use thereof
KR100949791B1 (ko) * 2007-12-18 2010-03-30 이동기 오프-타겟 효과를 최소화하고 RNAi 기구를 포화시키지않는 신규한 siRNA 구조 및 그 용도
EP2242854A4 (de) * 2008-01-15 2012-08-15 Quark Pharmaceuticals Inc Sirna-verbindungen und verfahren zur verwendung davon
EP2247729B1 (de) 2008-02-11 2019-05-01 Phio Pharmaceuticals Corp. Modifizierte rnai-polynukleotide und verwendungen davon
US8188060B2 (en) 2008-02-11 2012-05-29 Dharmacon, Inc. Duplex oligonucleotides with enhanced functionality in gene regulation
JP5540312B2 (ja) * 2008-02-15 2014-07-02 独立行政法人理化学研究所 環状1本鎖核酸複合体およびその製造方法
CA2718765A1 (en) * 2008-03-20 2009-09-24 Quark Pharmaceuticals, Inc. Novel sirna compounds for inhibiting rtp801
US8278287B2 (en) * 2008-04-15 2012-10-02 Quark Pharmaceuticals Inc. siRNA compounds for inhibiting NRF2
US8258111B2 (en) 2008-05-08 2012-09-04 The Johns Hopkins University Compositions and methods related to miRNA modulation of neovascularization or angiogenesis
CA2635187A1 (en) 2008-06-05 2009-12-05 The Royal Institution For The Advancement Of Learning/Mcgill University Oligonucleotide duplexes and uses thereof
WO2009147684A2 (en) 2008-06-06 2009-12-10 Quark Pharmaceuticals, Inc. Compositions and methods for treatment of ear disorders
TWI455944B (zh) 2008-07-01 2014-10-11 Daiichi Sankyo Co Ltd 雙股多核苷酸
US8309791B2 (en) * 2008-07-16 2012-11-13 Recombinectics, Inc. Method for producing a transgenic pig using a hyper-methylated transposon
WO2010008582A2 (en) 2008-07-18 2010-01-21 Rxi Pharmaceuticals Corporation Phagocytic cell drug delivery system
US9089610B2 (en) 2008-08-19 2015-07-28 Nektar Therapeutics Complexes of small-interfering nucleic acids
EP2340309A2 (de) 2008-09-22 2011-07-06 Rxi Pharmaceuticals Corporation Neutrale nanotransporter
JP2012504389A (ja) * 2008-09-22 2012-02-23 ダイセルナ ファーマシューティカルズ, インコーポレイテッド 修飾を有するdsRNAによる遺伝子発現の特異的な阻害のための組成物および方法
AU2009307677C1 (en) 2008-10-20 2022-09-22 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of transthyretin
CN102994500B (zh) * 2008-10-22 2015-04-01 夸克制药公司 治疗眼部疾病的方法
WO2010048585A2 (en) 2008-10-24 2010-04-29 Isis Pharmaceuticals, Inc. Oligomeric compounds and methods
CA2741294C (en) 2008-10-24 2018-04-24 Isis Pharmaceuticals, Inc. 5' and 2' bis-substituted nucleosides and oligomeric compounds prepared therefrom
US9074211B2 (en) 2008-11-19 2015-07-07 Rxi Pharmaceuticals Corporation Inhibition of MAP4K4 through RNAI
US20110288155A1 (en) 2008-12-18 2011-11-24 Elena Feinstein Sirna compounds and methods of use thereof
US9493774B2 (en) 2009-01-05 2016-11-15 Rxi Pharmaceuticals Corporation Inhibition of PCSK9 through RNAi
WO2010090762A1 (en) 2009-02-04 2010-08-12 Rxi Pharmaceuticals Corporation Rna duplexes with single stranded phosphorothioate nucleotide regions for additional functionality
WO2010091878A2 (en) 2009-02-13 2010-08-19 Silence Therapeutics Ag Means for inhibiting the expression of opa1
JP2012517815A (ja) 2009-02-18 2012-08-09 サイレンス・セラピューティクス・アーゲー Ang2の発現を阻害するための手段
JP2012520683A (ja) * 2009-03-19 2012-09-10 メルク・シャープ・エンド・ドーム・コーポレイション 低分子干渉核酸(siNA)を用いた結合組織増殖因子(CTGF)遺伝子発現のRNA干渉媒介性阻害
CA2753388C (en) 2009-03-23 2016-11-29 Quark Pharmaceuticals, Inc. Endo180 antibody to treat cancer and fibrotic disease
EP2440214A4 (de) 2009-06-08 2013-07-31 Quark Pharmaceuticals Inc Verfahren zur behandlung chronischer nierenkrankheiten
GB0910723D0 (en) 2009-06-22 2009-08-05 Sylentis Sau Novel drugs for inhibition of gene expression
US8916693B2 (en) 2009-09-17 2014-12-23 Nektar Therapeutics Monoconjugated chitosans as delivery agents for small interfering nucleic acids
US8796238B2 (en) 2009-09-17 2014-08-05 Sigma-Aldrich Co. Llc Short RNA mimetics
US8901097B2 (en) 2009-11-08 2014-12-02 Quark Pharmaceuticals, Inc. Methods for delivery of siRNA to the spinal cord and therapies arising therefrom
DK2504435T3 (da) 2009-11-26 2019-12-09 Quark Pharmaceuticals Inc Sirna-forbindelser omfattende terminale substitutioner
JP5685267B2 (ja) 2009-12-09 2015-03-18 日東電工株式会社 Hsp47発現の調節
EP2510098B1 (de) 2009-12-09 2015-02-11 Quark Pharmaceuticals, Inc. Verfahren und zusammensetzungen und zur behandlung von erkrankungen, störungen oder läsionen des zns
WO2011084193A1 (en) 2010-01-07 2011-07-14 Quark Pharmaceuticals, Inc. Oligonucleotide compounds comprising non-nucleotide overhangs
BR112012024049A2 (pt) 2010-03-24 2017-03-01 Rxi Pharmaceuticals Corp interferência de rna em indicações dérmicas e fibróticas
EP2550000A4 (de) 2010-03-24 2014-03-26 Advirna Inc Selbstfreisetzende rnai-verbindungen von reduzierter grösse
CN106074591B (zh) 2010-03-24 2020-01-14 菲奥医药公司 眼部症候中的rna干扰
EP2601204B1 (de) 2010-04-28 2016-09-07 Ionis Pharmaceuticals, Inc. Modifizierte nukleoside und daraus hergestellte oligomere verbindungen
EP3091027B1 (de) 2010-04-28 2018-01-17 Ionis Pharmaceuticals, Inc. 5'-modifizierte nukleoside und daraus hergestellte oligomere verbindungen
US20110306653A1 (en) 2010-05-14 2011-12-15 Tagcyx Biotechnologies Stabilization method of functional nucleic acid
EP2390327A1 (de) 2010-05-27 2011-11-30 Sylentis S.A. siRNA und deren Verwendung in Verfahren und Zusammensetzungen zur Behandlung und/oder Prävention von Augenleiden
RU2012153703A (ru) 2010-06-24 2014-07-27 Кварк Фармасьютикалс, Инк. Соединения двухцепочечных рнк к rhoa и их применение
PT2933333T (pt) * 2010-07-08 2018-04-24 Bonac Corp Molécula de ácido nucleico de cadeia simples para controlar a expressão génica
EP2412724A1 (de) 2010-07-29 2012-02-01 Centre National de la Recherche Scientifique (C.N.R.S) Regulierung der Glypican-4-Aktivität zur Modulierung des Schicksals von Stammzellen und Verwendungen davon
CN104059911B (zh) 2010-08-03 2017-01-04 株式会社博纳克 具有含氮脂环式骨架的单链核酸分子
WO2012027206A1 (en) 2010-08-24 2012-03-01 Merck Sharp & Dohme Corp. SINGLE-STRANDED RNAi AGENTS CONTAINING AN INTERNAL, NON-NUCLEIC ACID SPACER
CA2812046A1 (en) 2010-09-15 2012-03-22 Alnylam Pharmaceuticals, Inc. Modified irna agents
EP2631291B1 (de) 2010-10-22 2019-05-15 OliX Pharmaceuticals, Inc. Nukleinsäuremoleküle zur induzierung einer rna-interferenz und verwendungen davon
EP3766975A1 (de) 2010-10-29 2021-01-20 Sirna Therapeutics, Inc. Rna-interferenz-vermittelte inhibition von genexpression unter verwendung kurzer interferierender nukleinsäure (sina)
US20130324591A1 (en) 2010-12-06 2013-12-05 Quark Pharmaceuticals, Inc. Double stranded oligonucleotide compounds comprising positional modifications
EP2681314B1 (de) 2011-03-03 2017-11-01 Quark Pharmaceuticals, Inc. Zusammensetzungen und verfahren zur behandlung von lungenerkrankungen und läsionen
WO2012118911A1 (en) 2011-03-03 2012-09-07 Quark Pharmaceuticals, Inc. Oligonucleotide modulators of the toll-like receptor pathway
US9796979B2 (en) 2011-03-03 2017-10-24 Quark Pharmaceuticals Inc. Oligonucleotide modulators of the toll-like receptor pathway
US10196637B2 (en) 2011-06-08 2019-02-05 Nitto Denko Corporation Retinoid-lipid drug carrier
TWI658830B (zh) 2011-06-08 2019-05-11 日東電工股份有限公司 Hsp47表現調控強化用類視色素脂質體
JP6165723B2 (ja) 2011-06-30 2017-07-19 アローヘッド ファーマシューティカルズ インコーポレイテッド B型肝炎ウイルスの遺伝子発現を阻害するための組成物および方法
WO2013040251A2 (en) 2011-09-13 2013-03-21 Asurgen, Inc. Methods and compositions involving mir-135b for distinguishing pancreatic cancer from benign pancreatic disease
ES2745373T3 (es) 2011-10-18 2020-03-02 Dicerna Pharmaceuticals Inc Lípidos catiónicos de amina y uso de los mismos
WO2013070821A1 (en) 2011-11-08 2013-05-16 Quark Pharmaceuticals, Inc. Methods and compositions for treating diseases, disorders or injury of the nervous system
MY167390A (en) 2011-11-18 2018-08-16 Alnylam Pharmaceuticals Inc Rnai agents, compositions and methods of use thereof for treating transthyretin (ttr) associated diseases
IL303831A (en) 2011-11-18 2023-08-01 Alnylam Pharmaceuticals Inc Adapted RNAI factors
CN104080480A (zh) 2012-01-01 2014-10-01 奇比艾企业有限公司 用于选择性递送治疗剂和诊断剂的endo180靶向微粒
JP2016528161A (ja) 2012-01-12 2016-09-15 クォーク ファーマシューティカルズ インコーポレーティッドQuark Pharmaceuticals,Inc. 聴覚およびバランス障害を治療するための併用療法
WO2013123996A1 (en) 2012-02-24 2013-08-29 Astrazeneca Uk Limited Novel sirna inhibitors of human icam-1
US9980942B2 (en) 2012-05-02 2018-05-29 Children's Hospital Medical Center Rejuvenation of precursor cells
DK2853597T3 (en) * 2012-05-22 2019-04-08 Olix Pharmaceuticals Inc RNA INTERFERENCE-INducing NUCLEIC ACID MOLECULES WITH CELL PENETENING EQUIPMENT AND USE THEREOF
CN109810977A (zh) 2012-05-26 2019-05-28 株式会社博纳克 具有递送功能的基因表达调控用单链核酸分子
WO2014018375A1 (en) 2012-07-23 2014-01-30 Xenon Pharmaceuticals Inc. Cyp8b1 and uses thereof in therapeutic and diagnostic methods
ES2750125T3 (es) 2012-09-05 2020-03-25 Sylentis Sau ARNip y su uso en métodos y composiciones para el tratamiento y/o la prevención de afecciones oculares
GB201215857D0 (en) 2012-09-05 2012-10-24 Sylentis Sau siRNA and their use in methods and compositions for the treatment and/or prevention of eye conditions
DK2895607T3 (da) 2012-09-12 2021-05-25 Quark Pharmaceuticals Inc Dobbeltstrengede oligonukleotidmolekyler til ddit4 og fremgangsmåder til anvendelse deraf
AU2013315524B2 (en) 2012-09-12 2019-01-31 Quark Pharmaceuticals, Inc. Double-stranded oligonucleotide molecules to p53 and methods of use thereof
EP2968149B1 (de) 2013-03-14 2024-05-08 Dicerna Pharmaceuticals, Inc. Verfahren zur herstellung eines anionischen mittels
WO2014176259A1 (en) 2013-04-22 2014-10-30 Icahn School Of Medicine At Mount Sinai Mutations in pdgfrb and notch3 as causes of autosomal dominant infantile myofibromatosis
TW201534578A (zh) 2013-07-08 2015-09-16 Daiichi Sankyo Co Ltd 新穎脂質
EP3027223A1 (de) 2013-07-31 2016-06-08 QBI Enterprises Ltd. Verfahren zur verwendung von sphingolipid-polyalkylamin-oligonukleotidverbindungen
US9889200B2 (en) 2013-07-31 2018-02-13 Qbi Enterprises Ltd. Sphingolipid-polyalkylamine-oligonucleotide compounds
EP2853595A1 (de) 2013-09-30 2015-04-01 Soluventis GmbH NOTCH 1-spezifische siRNA-Moleküle
EP3052627B1 (de) * 2013-10-04 2018-08-22 Novartis AG Neuartige formate für organische verbindungen zur verwendung bei rna-interferenz
EP2865758A1 (de) 2013-10-22 2015-04-29 Sylentis, S.A.U. siRNA und deren Verwendung in Verfahren und Zusammensetzungen zur Hemmung der Expression des ORAI1-Gens
EP2865756A1 (de) 2013-10-22 2015-04-29 Sylentis, S.A.U. siRNA und deren Verwendung in Verfahren und Zusammensetzungen zur Hemmung der Expression des FLAP-Gens
EP2865757A1 (de) * 2013-10-22 2015-04-29 Sylentis, S.A.U. siRNA und deren Verwendung in Verfahren und Zusammensetzungen zur Hemmung der Expression des PDK1-Gens
CN106061488B (zh) 2013-12-02 2021-04-09 菲奥医药公司 癌症的免疫治疗
US10612020B2 (en) 2013-12-26 2020-04-07 Tokyo Medical University Artificial mimic miRNA for controlling gene expression, and use of same
WO2015099187A1 (ja) 2013-12-27 2015-07-02 株式会社ボナック 遺伝子発現制御のための人工マッチ型miRNAおよびその用途
CA2937118A1 (en) * 2014-01-17 2015-07-23 Kyowa Hakko Kirin Co., Ltd. Nucleic acid capable of inhibiting expression of .beta.2gpi
AU2015217301A1 (en) 2014-02-11 2016-08-25 Alnylam Pharmaceuticals, Inc. Ketohexokinase (KHK) iRNA compositions and methods of use thereof
US10011837B2 (en) 2014-03-04 2018-07-03 Sylentis Sau SiRNAs and their use in methods and compositions for the treatment and/or prevention of eye conditions
US11279934B2 (en) 2014-04-28 2022-03-22 Phio Pharmaceuticals Corp. Methods for treating cancer using nucleic acids targeting MDM2 or MYCN
CN107075570B (zh) * 2014-08-07 2021-01-05 财团法人工业技术研究院 小干扰rna以及含其之用于抑制半乳糖凝集素-12表现及/或促进脂肪分解的医药组成物及其用途
KR102506169B1 (ko) 2014-09-05 2023-03-08 피오 파마슈티칼스 코프. Tyr 또는 mmp1을 표적화하는 핵산을 사용한 노화 및 피부 장애의 치료 방법
FR3026409B1 (fr) * 2014-09-26 2018-03-30 Centre National De La Recherche Scientifique (Cnrs) Procede de criblage de molecules interferentes
SG11201703638XA (en) 2014-11-10 2017-06-29 Alnylam Pharmaceuticals Inc Hepatitis b virus (hbv) irna compositions and methods of use thereof
WO2016083624A1 (en) 2014-11-28 2016-06-02 Silence Therapeutics Gmbh Means for inhibiting the expression of edn1
US12359197B2 (en) 2014-12-12 2025-07-15 Etagen Pharma, Inc. Compositions and methods for editing nucleic acids in cells utilizing oligonucleotides
US11027023B2 (en) 2014-12-27 2021-06-08 Bonac Corporation Natural type miRNA for controlling gene expression, and use of same
WO2016149020A1 (en) * 2015-03-17 2016-09-22 Arrowhead Research Corporation Rna interference agents
US11142769B2 (en) 2015-03-27 2021-10-12 Bonac Corporation Single-stranded nucleic acid molecule having delivery function and gene expression regulating ability
US11001845B2 (en) 2015-07-06 2021-05-11 Phio Pharmaceuticals Corp. Nucleic acid molecules targeting superoxide dismutase 1 (SOD1)
US10808247B2 (en) 2015-07-06 2020-10-20 Phio Pharmaceuticals Corp. Methods for treating neurological disorders using a synergistic small molecule and nucleic acids therapeutic approach
JP6896703B2 (ja) 2015-07-31 2021-06-30 アルナイラム ファーマシューティカルズ, インコーポレイテッドAlnylam Pharmaceuticals, Inc. TTR関連疾患を治療または予防するためのトランスサイレチン(TTR)iRNA組成物およびその使用方法
KR20180038465A (ko) 2015-08-07 2018-04-16 애로우헤드 파마슈티컬스 인코포레이티드 B형 간염 바이러스 감염에 대한 rnai 치료법
MA44908A (fr) 2015-09-08 2018-07-18 Sylentis Sau Molécules d'arnsi et leur utilisation dans des procédés et des compositions pour inhiber l'expression du gène nrarp
CN109563509B (zh) 2015-10-19 2022-08-09 菲奥医药公司 靶向长非编码rna的减小尺寸的自递送核酸化合物
EP3371211B1 (de) 2015-11-04 2025-01-01 Icahn School of Medicine at Mount Sinai Rho-assoziierte proteinkinasehemmer zur behandlung von tumoren und krebs sowie identifizierung von kandidatenpatienten für solch eine behandlung
EP3377630A4 (de) 2015-11-16 2020-01-01 Olix Pharmaceuticals, Inc. Behandlung von altersbedingter makuladegeneration mit auf myd88 oder tlr3 abzielenden rna-komplexen
CN116064544A (zh) 2016-01-26 2023-05-05 日产化学株式会社 单链寡核苷酸
EP3411481A4 (de) 2016-02-02 2020-02-26 Olix Pharmaceuticals, Inc. Behandlung von angiogeneseassoziierten erkrankungen mit auf angpt2 und pdgfb abzielenden rna-komplexen
JP6944942B2 (ja) 2016-02-02 2021-10-06 オリックス ファーマシューティカルズ,インコーポレーテッド IL4Rα、TRPA1、またはF2RL1を標的とするRNA複合体を用いたアトピー性皮膚炎および喘息の治療
WO2023150553A1 (en) 2022-02-01 2023-08-10 University Of Rochester Gpr17 promoter-based targeting and transduction of glial progenitor cells
KR102775461B1 (ko) 2016-04-01 2025-02-28 어비디티 바이오사이언시스 인크. 핵산-폴리펩타이드 조성물 및 이의 용도
WO2017178883A2 (en) 2016-04-11 2017-10-19 Olix Pharmaceuticals, Inc. Treatment of idiopathic pulmonary fibrosis using rna complexes that target connective tissue growth factor
WO2017181088A1 (en) 2016-04-14 2017-10-19 University Of Florida Research Foundation, Incorporated Use of mir-223-3p as a cancer therapeutic and method for treating cancer using the same
WO2017189730A1 (en) 2016-04-26 2017-11-02 Icahn School Of Medicine At Mount Sinai Treatment of hippo pathway mutant tumors and methods of identifying subjects as candidates for treatment
EP3452055A4 (de) 2016-05-06 2019-11-06 Tod M. Woolf Verbesserte verfahren zur genomänderung mit und ohne programmierbaren nukleasen
KR101916652B1 (ko) 2016-06-29 2018-11-08 올릭스 주식회사 작은 간섭 rna의 rna 간섭효과 증진용 화합물 및 이의 용도
JOP20190015A1 (ar) 2016-08-04 2019-02-04 Arrowhead Pharmaceuticals Inc عوامل (ار ان ايه آي) لعدوى فيروس الالتهاب الكبدي الوبائي ب
EP3315125A1 (de) 2016-10-31 2018-05-02 Silence Therapeutics (London) Ltd Lipidnanopartikelformulierung
AU2017368050A1 (en) 2016-11-29 2019-06-20 Puretech Lyt, Inc. Exosomes for delivery of therapeutic agents
CN110381980A (zh) 2017-01-06 2019-10-25 艾维迪提生物科学有限责任公司 核酸-多肽组合物以及诱导外显子跳读的方法
US11572558B2 (en) 2017-02-06 2023-02-07 Nissan Chemical Corporation Single-stranded oligonucleotide
EP3580339A4 (de) 2017-02-10 2020-12-23 Research & Business Foundation Sungkyunkwan University Lange doppelsträngige rna für rna-interferenz
US11324820B2 (en) 2017-04-18 2022-05-10 Alnylam Pharmaceuticals, Inc. Methods for the treatment of subjects having a hepatitis b virus (HBV) infection
JP7167059B2 (ja) 2017-04-19 2022-11-08 フィオ ファーマシューティカルズ コーポレーション 核酸化合物の局所送達
GB201711809D0 (en) 2017-07-21 2017-09-06 Governors Of The Univ Of Alberta Antisense oligonucleotide
EP4219715A3 (de) * 2017-09-08 2023-09-06 MiNA Therapeutics Limited Stabilisierte cebpa-sarna-zusammensetzungen und verfahren zur verwendung
CA3075205A1 (en) * 2017-09-08 2019-03-14 Mina Therapeutics Limited Stabilized hnf4a sarna compositions and methods of use
KR20200089656A (ko) 2017-09-19 2020-07-27 알닐람 파마슈티칼스 인코포레이티드 트랜스타이레틴(ttr) 매개 아밀로이드증을 치료하기 위한 조성물 및 방법
MX2020004898A (es) * 2017-11-13 2020-10-05 Silence Therapeutics Gmbh Ácidos nucleicos para inhibir la expresión de un gen diana que comprende enlaces fosforoditioato.
CN118638788A (zh) 2017-12-06 2024-09-13 艾维迪提生物科学公司 治疗肌萎缩和强直性肌营养不良的组合物和方法
IL314908A (en) 2018-03-29 2024-10-01 Technion Res & Dev Foundation Vesicles comprising a pten inhibitor and uses of same
CA3103663A1 (en) 2018-06-18 2019-12-26 University Of Rochester Inhibition of re1-silencing transcription factor in the treatment of schizophrenia and other neuropsychiatric disorders
KR20210054502A (ko) 2018-06-21 2021-05-13 유니버시티 오브 로체스터 헌팅턴병을 치료하거나 이의 발병을 저해하는 방법
MA70658B1 (fr) 2018-08-13 2026-04-30 Alnylam Pharmaceuticals, Inc. Compositions d'agent d'arndb du virus de l'hépatite b (vhb) et leurs méthodes d'utilisation
CA3122289A1 (en) 2018-12-11 2020-06-18 University Of Rochester Methods of treating schizophrenia and other neuropsychiatric disorders
IL319265A (en) 2018-12-21 2025-04-01 Avidity Biosciences Inc Anti-transferrin receptor antibodies and their uses
WO2020167822A2 (en) 2019-02-13 2020-08-20 University Of Rochester Gene networks that mediate remyelination of the human brain
CA3137715A1 (en) 2019-04-25 2020-10-29 Avidity Biosciences, Inc. Nucleic acid compositions and methods of multi-exon skipping
JP7708737B2 (ja) 2019-07-18 2025-07-15 ユニバーシティー オブ ロチェスター 細胞の細胞型選択的免疫保護
EP4013402B1 (de) 2019-08-16 2024-11-06 Children's Hospital Medical Center Cdc-42-inhibitoren zur verwendung bei der behandlung von altersverbundener depression, sarkopenie oder gebrechlichkeit
WO2021049504A1 (ja) 2019-09-10 2021-03-18 第一三共株式会社 肝臓送達用GalNAc-オリゴヌクレオチドコンジュゲートおよび製造方法
WO2021188390A1 (en) 2020-03-19 2021-09-23 Avidity Biosciences, Inc. Compositions and methods of treating facioscapulohumeral muscular dystrophy
WO2021195469A1 (en) 2020-03-27 2021-09-30 Avidity Biosciences, Inc. Compositions and methods of treating muscle dystrophy
WO2021255262A1 (en) 2020-06-19 2021-12-23 Sylentis Sau siRNA AND COMPOSITIONS FOR PROPHYLACTIC AND THERAPEUTIC TREATMENT OF VIRUS DISEASES
US11459567B2 (en) 2020-06-24 2022-10-04 Patricia Virginia Elizalde Specific siRNA molecules, composition and use thereof for the treatment of triple negative breast cancer
EP4015634A1 (de) 2020-12-15 2022-06-22 Sylentis, S.A.U. Sirna und zusammensetzungen zur prophylaktischen und therapeutischen behandlung von viruskrankheiten
WO2022162161A1 (en) 2021-01-30 2022-08-04 E-Therapeutics Plc Conjugated oligonucleotide compounds, methods of making and uses thereof
WO2022162155A1 (en) 2021-01-30 2022-08-04 E-Therapeutics Plc Nucleic acids containing abasic nucleotides
CN117355534B (zh) 2021-01-30 2026-05-01 唐格拉姆治疗公司 缀合寡核苷酸化合物、其制备方法和用途
WO2022162157A1 (en) 2021-01-30 2022-08-04 E-Therapeutics Plc Conjugated oligonucleotide compounds, methods of making and uses thereof
WO2022162154A1 (en) 2021-01-30 2022-08-04 E-Therapeutics Plc Conjugated oligonucleotide compounds, methods of making and uses thereof
TW202328445A (zh) 2021-08-03 2023-07-16 美商艾拉倫製藥股份有限公司 甲狀腺素運載蛋白(TTR)iRNA組成物及其使用方法
IL309505A (en) 2021-09-03 2024-02-01 CureVac SE Novel lipid nanoparticles for delivery of nucleic acids
MX2024003266A (es) 2021-09-16 2024-04-03 Avidity Biosciences Inc Composiciones y metodos de tratamiento de la distrofia muscular facioescapulohumeral.
US12258563B2 (en) 2022-03-28 2025-03-25 Empirico Inc. Modified oligonucleotides
US12071621B2 (en) 2022-04-05 2024-08-27 Avidity Biosciences, Inc. Anti-transferrin receptor antibody-PMO conjugates for inducing DMD exon 44 skipping
EP4560020A1 (de) 2023-11-22 2025-05-28 Sylentis S.A.U. Sirna und zusammensetzungen zur prophylaktischen und therapeutischen behandlung von augenerkrankungen der retina
EP4560019A1 (de) 2023-11-22 2025-05-28 Sylentis S.A.U. Sirna und zusammensetzungen zur prophylaktischen und therapeutischen behandlung von augenerkrankungen der retina

Family Cites Families (85)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
GB992744A (en) 1962-01-18 1965-05-19 Dennis Harold Ward Plastic containers and lifting means therefore
US4217294A (en) 1979-03-30 1980-08-12 Union Carbide Corporation Method for producing mercaptan-containing organosilicon compounds
DK0942000T3 (da) 1989-10-24 2004-11-01 Isis Pharmaceuticals Inc 2'-modificerede oligonukleotider
US5872232A (en) 1990-01-11 1999-02-16 Isis Pharmaceuticals Inc. 2'-O-modified oligonucleotides
US6399754B1 (en) 1991-12-24 2002-06-04 Isis Pharmaceuticals, Inc. Sugar modified oligonucleotides
US6153737A (en) 1990-01-11 2000-11-28 Isis Pharmaceuticals, Inc. Derivatized oligonucleotides having improved uptake and other properties
US5914396A (en) 1990-01-11 1999-06-22 Isis Pharmaceuticals, Inc. 2'-O-modified nucleosides and phosphoramidites
US6005087A (en) 1995-06-06 1999-12-21 Isis Pharmaceuticals, Inc. 2'-modified oligonucleotides
US5670633A (en) 1990-01-11 1997-09-23 Isis Pharmaceuticals, Inc. Sugar modified oligonucleotides that detect and modulate gene expression
US5955589A (en) * 1991-12-24 1999-09-21 Isis Pharmaceuticals Inc. Gapped 2' modified oligonucleotides
US5811533A (en) 1990-06-11 1998-09-22 Nexstar Pharmaceuticals, Inc. High-affinity oligonucleotide ligands to vascular endothelial growth factor (VEGF)
US5378825A (en) 1990-07-27 1995-01-03 Isis Pharmaceuticals, Inc. Backbone modified oligonucleotide analogs
WO1992007065A1 (en) 1990-10-12 1992-04-30 MAX-PLANCK-Gesellschaft zur Förderung der Wissenschaften e.V. Modified ribozymes
FR2675803B1 (fr) 1991-04-25 1996-09-06 Genset Sa Oligonucleotides fermes, antisens et sens et leurs applications.
FR2678512B1 (fr) 1991-07-03 1995-06-30 Novacell Machine a internaliser.
KR940703846A (ko) 1991-12-24 1994-12-12 비. 린네 파샬 갭(gap)이 형성된 2′ 변성된 올리고뉴클레오티드(gapped 2′ modifed oligonucleotides)
FI946201A0 (fi) 1992-07-02 1994-12-30 Hybridon Inc Itsestabiloidut oligonukleotidit terapeuttisina aineina
CA2105364A1 (en) * 1993-09-01 1995-03-02 Eric T. Kool Stem-loop oligonucleotides containing parallel and antiparallel binding domains
DE4338704A1 (de) 1993-11-12 1995-05-18 Hoechst Ag Stabilisierte Oligonucleotide und deren Verwendung
US6060456A (en) * 1993-11-16 2000-05-09 Genta Incorporated Chimeric oligonucleoside compounds
IL111660A (en) * 1993-11-16 2005-05-17 Genta Inc Oligonucleoside compounds for effecting rnaseh-mediated cleavage of a target ribonucleic acid sequence and a pharmaceutical composition containing them
US5716824A (en) 1995-04-20 1998-02-10 Ribozyme Pharmaceuticals, Inc. 2'-O-alkylthioalkyl and 2-C-alkylthioalkyl-containing enzymatic nucleic acids (ribozymes)
AU4412096A (en) 1994-12-13 1996-07-03 Ribozyme Pharmaceuticals, Inc. Method and reagent for treatment of arthritic conditions, induction of graft tolerance and reversal of immune responses
GB9425118D0 (en) 1994-12-13 1995-02-08 Henkel Kgaa Two component natural hair colorant
US5760012A (en) * 1996-05-01 1998-06-02 Thomas Jefferson University Methods and compounds for curing diseases caused by mutations
DE19618797C2 (de) 1996-05-10 2000-03-23 Bertling Wolf Vehikel zum Transport molekularer Substanz
US5898031A (en) * 1996-06-06 1999-04-27 Isis Pharmaceuticals, Inc. Oligoribonucleotides for cleaving RNA
DE19631919C2 (de) 1996-08-07 1998-07-16 Deutsches Krebsforsch Anti-Sinn-RNA mit Sekundärstruktur
US6284267B1 (en) 1996-08-14 2001-09-04 Nutrimed Biotech Amphiphilic materials and liposome formulations thereof
GB9710475D0 (en) 1997-05-21 1997-07-16 Zeneca Ltd Gene silencing
US6133246A (en) * 1997-08-13 2000-10-17 Isis Pharmaceuticals Inc. Antisense oligonucleotide compositions and methods for the modulation of JNK proteins
GB9720148D0 (en) 1997-09-22 1997-11-26 Innes John Centre Innov Ltd Gene silencing materials and methods
JP2001517686A (ja) 1997-09-26 2001-10-09 ユーエイビー リサーチ ファンデーション 抗原性を減弱させた血球および、その利用法
US6617438B1 (en) * 1997-11-05 2003-09-09 Sirna Therapeutics, Inc. Oligoribonucleotides with enzymatic activity
US6506559B1 (en) 1997-12-23 2003-01-14 Carnegie Institute Of Washington Genetic inhibition by double-stranded RNA
BRPI9908967B1 (pt) 1998-03-20 2017-05-30 Benitec Australia Ltd processos para reprimir, retardar ou de outro modo reduzir a expressão de um gene alvo em uma célula de planta
AU760041B2 (en) 1998-04-08 2003-05-08 Commonwealth Scientific And Industrial Research Organisation Methods and means for obtaining modified phenotypes
WO1999054459A2 (en) 1998-04-20 1999-10-28 Ribozyme Pharmaceuticals, Inc. Nucleic acid molecules with novel chemical compositions capable of modulating gene expression
JP2002512794A (ja) 1998-04-29 2002-05-08 リボザイム・ファーマシューティカルズ・インコーポレーテッド ヌクレオチド三リン酸およびリボザイム内へのそれらの取り込み
AR020078A1 (es) 1998-05-26 2002-04-10 Syngenta Participations Ag Metodo para alterar la expresion de un gen objetivo en una celula de planta
GB9827152D0 (en) 1998-07-03 1999-02-03 Devgen Nv Characterisation of gene function using double stranded rna inhibition
WO2000044914A1 (en) 1999-01-28 2000-08-03 Medical College Of Georgia Research Institute, Inc. Composition and method for in vivo and in vitro attenuation of gene expression using double stranded rna
US6200924B1 (en) 1999-01-29 2001-03-13 E. I. Du Pont De Nemours And Company Porous highly fluorinated acidic polymer catalyst
DE19956568A1 (de) 1999-01-30 2000-08-17 Roland Kreutzer Verfahren und Medikament zur Hemmung der Expression eines vorgegebenen Gens
CA2370628A1 (en) 1999-04-21 2000-10-26 American Home Products Corporation Methods and compositions for inhibiting the function of polynucleotide sequences
DE60026313D1 (de) 1999-07-23 2006-04-27 Uutech Ltd Sensibilisierung von roten blutkörperchen gegenüber ultraschall durch einwirkung eines elektrischen feldes
GB9927444D0 (en) 1999-11-19 2000-01-19 Cancer Res Campaign Tech Inhibiting gene expression
US7829693B2 (en) 1999-11-24 2010-11-09 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of a target gene
DE10160151A1 (de) 2001-01-09 2003-06-26 Ribopharma Ag Verfahren zur Hemmung der Expression eines vorgegebenen Zielgens
WO2003070918A2 (en) 2002-02-20 2003-08-28 Ribozyme Pharmaceuticals, Incorporated Rna interference by modified short interfering nucleic acid
ATE450621T2 (de) 2000-03-30 2009-12-15 Whitehead Biomedical Inst Mediatoren von rns-interferenz, die rns- sequenzspezifisch sind
CA2456008A1 (en) 2000-08-19 2002-02-28 Axordia Limited Stem cell differentiation
US20030190635A1 (en) 2002-02-20 2003-10-09 Mcswiggen James A. RNA interference mediated treatment of Alzheimer's disease using short interfering RNA
KR100872437B1 (ko) 2000-12-01 2008-12-05 막스-플랑크-게젤샤프트 츄어 푀르더룽 데어 비쎈샤프텐 에.파우. Rna 간섭을 매개하는 작은 rna 분자
US20060142226A1 (en) 2001-05-18 2006-06-29 Sirna Therapeutics, Inc. RNA interference mediated inhibition of cholesteryl ester transfer protein (CETP) gene expression using short interfering nucleic acid (siNA)
US20030124513A1 (en) 2001-05-29 2003-07-03 Mcswiggen James Enzymatic nucleic acid treatment of diseases or conditions related to levels of HIV
WO2003070912A2 (en) 2001-06-06 2003-08-28 Sirna Therapeutics, Inc. RNA INTERFERENCE MEDIATED INHIBITION OF EPIDERMAL GROWTH FACTOR RECEPTOR GENE EXPRESSION USING SHORT INTERFERING NUCLEIC ACID (siNA)
WO2003000744A1 (en) 2001-06-22 2003-01-03 Borealis Technology Oy Metallocene catalysts containing an idenyl moiety substituted at the 4-, 5-, 6- or 7-position by a siloxy or germyloxy group
DE10163098B4 (de) 2001-10-12 2005-06-02 Alnylam Europe Ag Verfahren zur Hemmung der Replikation von Viren
US20030135033A1 (en) 2002-01-04 2003-07-17 Anke Klippel-Giese Compounds and methods for the identification and/ or validation of a target
DE10202419A1 (de) 2002-01-22 2003-08-07 Ribopharma Ag Verfahren zur Hemmung der Expression eines durch eine Chromosomen-Aberration entstandenen Zielgens
ES2318106T3 (es) 2002-02-01 2009-05-01 Mcgill University Oligonucleotidos que comprenden segmentos alternos y usos de los mismos.
WO2003064621A2 (en) 2002-02-01 2003-08-07 Ambion, Inc. HIGH POTENCY siRNAS FOR REDUCING THE EXPRESSION OF TARGET GENES
AU2003207708A1 (en) * 2002-02-20 2003-09-09 Sirna Therapeutics, Inc. Rna interference mediated inhibition of map kinase genes
US7148342B2 (en) 2002-07-24 2006-12-12 The Trustees Of The University Of Pennyslvania Compositions and methods for sirna inhibition of angiogenesis
ES2280826T5 (es) 2002-08-05 2017-08-03 Silence Therapeutics Gmbh Nuevas formas adicionales de moléculas de ARN de interferencia
EP1393742A1 (de) 2002-08-14 2004-03-03 atugen AG Verwendung von Protein Kinase N beta
WO2004019973A1 (en) 2002-08-14 2004-03-11 Atugen Ag Use of protein kinase n beta
ES2319891T3 (es) 2002-10-18 2009-05-14 Silence Therapeutics Ag Factor implicado en la metastasis y usos del mismo.
US7289445B2 (en) * 2002-11-25 2007-10-30 Intel Corporation Managing a protocol control block cache in a network device
WO2005014782A2 (en) 2003-06-13 2005-02-17 Alnylam Europe Ag., Double-stranded ribonucleic acid with increased effectiveness in an organism
JP2009513487A (ja) 2003-06-27 2009-04-02 サイレンス・セラピューティクス・アーゲー 細胞溶解を誘発するための二本鎖リボ核酸の使用
ES2423060T3 (es) 2004-03-12 2013-09-17 Alnylam Pharmaceuticals, Inc. Agentes iRNA que tienen como diana al VEGF
US20070265220A1 (en) 2004-03-15 2007-11-15 City Of Hope Methods and compositions for the specific inhibition of gene expression by double-stranded RNA
US20050217329A1 (en) * 2004-04-02 2005-10-06 Wen-Kwei Chang Anti-drilling structure of a latch
PT1771206T (pt) 2004-05-05 2018-05-16 Silence Therapeutics Gmbh Lípidos, complexos lipídicos e sua utilização
CA2568013C (en) 2004-05-27 2015-11-24 Alnylam Pharmaceuticals, Inc. Nuclease resistant double-stranded ribonucleic acid
JP4468989B2 (ja) 2004-08-16 2010-05-26 クアーク・ファーマスーティカルス、インコーポレイテッド Rtp801阻害剤の治療への使用
EP2992902A1 (de) 2004-12-27 2016-03-09 Silence Therapeutics GmbH Mit peg beschichtete lipid-komplexe und ihre anwendung
US20070027097A1 (en) 2005-07-28 2007-02-01 Lewis David L Small interfering RNA with improved activity
BRPI0617860A2 (pt) 2005-10-28 2011-08-09 Topigen Pharmaceuticals Inc pequenos oligonucleotìdeos de interferência que compreendem nucleotìdeos modificados com arabinose
WO2007051303A1 (en) 2005-11-02 2007-05-10 Protiva Biotherapeutics, Inc. MODIFIED siRNA MOLECULES AND USES THEREOF
UY30097A1 (es) 2006-01-20 2007-08-31 Atugen Ag Usos terapeuticos de inhibidores de rtp801
US7541994B2 (en) 2006-05-17 2009-06-02 Raytheon Company Refractive compact range
CN103898110A (zh) 2007-10-03 2014-07-02 夸克制药公司 新siRNA结构

Also Published As

Publication number Publication date
US20170247697A1 (en) 2017-08-31
EP3222724A1 (de) 2017-09-27
CY1120128T1 (el) 2018-12-12
IL166546A (en) 2014-01-30
WO2004015107A3 (en) 2004-04-29
EP2258847B1 (de) 2017-03-15
EP3222724B1 (de) 2018-10-31
US8933215B2 (en) 2015-01-13
CY1106422T1 (el) 2011-10-12
US20040180351A1 (en) 2004-09-16
PT1527176E (pt) 2007-04-30
DK1527176T4 (en) 2017-07-03
US9790505B2 (en) 2017-10-17
US9758784B1 (en) 2017-09-12
US9695423B2 (en) 2017-07-04
SI2258847T1 (sl) 2017-08-31
EP1857547B1 (de) 2018-01-17
US10323246B2 (en) 2019-06-18
KR20050035877A (ko) 2005-04-19
US20190390201A1 (en) 2019-12-26
EP2258847A3 (de) 2011-05-18
DK1857547T3 (en) 2018-04-23
SI3222724T1 (sl) 2019-03-29
US20150105545A1 (en) 2015-04-16
JP2017163995A (ja) 2017-09-21
EP3222725A1 (de) 2017-09-27
CY1119021T1 (el) 2018-01-10
EP2258847B2 (de) 2020-07-01
ZA200500459B (en) 2005-08-31
US20190024089A1 (en) 2019-01-24
CN100543137C (zh) 2009-09-23
US20160130587A1 (en) 2016-05-12
CN1675359A (zh) 2005-09-28
US10266829B2 (en) 2019-04-23
US20170247698A1 (en) 2017-08-31
AU2003260370A1 (en) 2004-02-25
AU2003260370B2 (en) 2008-05-22
US8324370B2 (en) 2012-12-04
JP2011024592A (ja) 2011-02-10
US20170247691A1 (en) 2017-08-31
EP1857547A3 (de) 2011-05-18
DE60310944D1 (de) 2007-02-15
US20090186845A1 (en) 2009-07-23
EP1857547B2 (de) 2020-12-02
US20130102769A1 (en) 2013-04-25
DK2258847T3 (en) 2017-06-26
EP1857547A2 (de) 2007-11-21
US20110118456A1 (en) 2011-05-19
EP3222726A1 (de) 2017-09-27
DE60310944T2 (de) 2007-10-11
SI1857547T1 (en) 2018-05-31
US9783802B2 (en) 2017-10-10
CY1120919T1 (el) 2019-12-11
WO2004015107A2 (en) 2004-02-19
ES2280826T3 (es) 2007-09-16
JP2013179944A (ja) 2013-09-12
EP1527176B2 (de) 2017-03-22
CA2494930C (en) 2021-03-09
US7893245B2 (en) 2011-02-22
DK1527176T3 (da) 2007-05-07
ES2280826T5 (es) 2017-08-03
KR101201664B1 (ko) 2012-11-15
US9222092B2 (en) 2015-12-29
US20170247707A1 (en) 2017-08-31
EP1527176B1 (de) 2007-01-03
EP1527176A2 (de) 2005-05-04
US10774332B2 (en) 2020-09-15
ATE350473T2 (de) 2007-01-15
DE60310944T3 (de) 2017-08-03
BR0313202A (pt) 2005-06-21
JP2016144451A (ja) 2016-08-12
US20180291381A1 (en) 2018-10-11
US20180223286A1 (en) 2018-08-09
US20210062198A1 (en) 2021-03-04
JP6069096B2 (ja) 2017-01-25
EP2258847A2 (de) 2010-12-08
US11578328B2 (en) 2023-02-14
DK3222724T3 (en) 2018-12-03
AU2008207337A1 (en) 2008-09-04
JP5490655B2 (ja) 2014-05-14
US7452987B2 (en) 2008-11-18
US9790501B2 (en) 2017-10-17
MXPA05001355A (es) 2005-09-30
US10329568B2 (en) 2019-06-25
BRPI0313202A8 (pt) 2016-08-16
CA2494930A1 (en) 2004-02-19

Similar Documents

Publication Publication Date Title
SI3222724T1 (sl) Nadaljnje nove oblike molekul interferenčne RNA
WO2005014782A3 (en) Double-stranded ribonucleic acid with increased effectiveness in an organism
WO2002026967A3 (en) Targeted gene correction by single-stranded oligodeoxynucleotides
WO2004007718A3 (en) Rna-interference by single-stranded rna molecules
GB2439673A (en) Ghrelin binding nucleic acids
WO2005120230A3 (en) POSITIONALLY MODIFIED siRNA CONSTRUCTS
WO2007031877A3 (en) Modulation of immunostimulatory properties of short interfering ribonucleic acid (sirna) by nucleotide modification
WO2006002038A3 (en) Immunostimulatory oligonucleotide multimers
EP2135950A3 (de) Kurze interferierende Ribonukleinsäure (siRNA) zur oralen Verabreichung
WO2005121372A3 (en) Double strand compositions comprising differentially modified strands for use in gene modulation
WO2006110688A3 (en) Rnai therapeutic for respiratory virus infection
WO2006036739A3 (en) Gene silencing
WO2007051045A3 (en) Compositions and methods for inhibiting expression of huntingtin gene
WO2011056005A3 (ko) 안티센스 가닥에 의한 오프-타겟 효과를 최소화한 신규한 siRNA 구조 및 그 용도
WO2006083800A3 (en) Nucleic acid silencing of huntington's disease gene
WO2004065600A3 (en) Rna interference by palindromic or modified rna molecules
EP2700720A3 (de) Zusammensetzungen und Verfahren zur Optimierung der Spaltung von RNA durch RNase H
WO2004001013A3 (en) Inhibition of gene expression in vertebrates using double-stranded rna (rnai)
WO2005000320A3 (en) Use of double-stranded ribonucleic acid for inducing cell lysis
MX2007004686A (es) Moleculas de arn de multidominio que comprenden al menos un aptamero para suministrar arn de doble hebra a organismos nocivos.
EP1333089A4 (de) Verfahren zur synthese einzelsträngiger nukleinsäure
WO2007056331A3 (en) Compositions and methods for inhibiting expression of factor v leiden mutant gene
WO2004015117A3 (en) Targeted gene modification by single-stranded dna oligonucleotides
WO2008041933A3 (en) Rna interference against wrap53 to treat hyperproliferative diseases
WO2004016757A3 (en) Methods, compositions and kits for producing nucleic and acid sequences