ATE350473T2 - Weitere neue formen von interferierende rns moleküle - Google Patents

Weitere neue formen von interferierende rns moleküle Download PDF

Info

Publication number
ATE350473T2
ATE350473T2 AT03784183T AT03784183T ATE350473T2 AT E350473 T2 ATE350473 T2 AT E350473T2 AT 03784183 T AT03784183 T AT 03784183T AT 03784183 T AT03784183 T AT 03784183T AT E350473 T2 ATE350473 T2 AT E350473T2
Authority
AT
Austria
Prior art keywords
rna molecules
stretch
interfering rna
new forms
strand
Prior art date
Application number
AT03784183T
Other languages
English (en)
Inventor
Klaus Giese
Joerg Kaufmann
Anke Klippel-Giese
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=ATE350473(T2) "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
Application granted granted Critical
Publication of ATE350473T2 publication Critical patent/ATE350473T2/de

Links

Classifications

    • 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
    • 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
    • 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
    • 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
    • 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)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Molecular Biology (AREA)
  • General Engineering & Computer Science (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Biotechnology (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Diabetes (AREA)
  • Hematology (AREA)
  • Epidemiology (AREA)
  • Obesity (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Virology (AREA)
  • Immunology (AREA)
  • Endocrinology (AREA)
  • Urology & Nephrology (AREA)
  • Heart & Thoracic Surgery (AREA)
  • Cardiology (AREA)
  • Analytical Chemistry (AREA)
  • Cell Biology (AREA)
AT03784183T 2002-08-05 2003-08-05 Weitere neue formen von interferierende rns moleküle ATE350473T2 (de)

Applications Claiming Priority (3)

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

Publications (1)

Publication Number Publication Date
ATE350473T2 true ATE350473T2 (de) 2007-01-15

Family

ID=31720998

Family Applications (1)

Application Number Title Priority Date Filing Date
AT03784183T ATE350473T2 (de) 2002-08-05 2003-08-05 Weitere neue formen von interferierende rns moleküle

Country Status (19)

Country Link
US (15) US7452987B2 (de)
EP (6) EP1527176B2 (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) SI3222724T1 (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
US9096636B2 (en) 1996-06-06 2015-08-04 Isis Pharmaceuticals, Inc. Chimeric oligomeric compounds and their use in gene modulation
US5898031A (en) 1996-06-06 1999-04-27 Isis Pharmaceuticals, Inc. Oligoribonucleotides for cleaving RNA
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
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)
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
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)
PT1309726E (pt) 2000-03-30 2010-03-08 Whitehead Biomedical Inst Mediadores de interferência por rna específicos de sequência de rna
EP1416972B1 (de) * 2000-11-09 2012-02-08 Cold Spring Harbor Laboratory Chimäre moleküle zur modulation der genexpression
US20040259247A1 (en) 2000-12-01 2004-12-23 Thomas Tuschl Rna interference mediating small rna molecules
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)
US20030166282A1 (en) 2002-02-01 2003-09-04 David Brown High potency siRNAS for reducing the expression of target genes
US20060009409A1 (en) 2002-02-01 2006-01-12 Woolf Tod M Double-stranded oligonucleotides
EP2213737B1 (de) 2002-02-01 2012-11-07 Life Technologies Corporation Doppelsträngige Oligonukleotide
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)
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
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)
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)
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)
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)
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)
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)
AU2003260370B2 (en) 2002-08-05 2008-05-22 Silence Therapeutics Gmbh Further novel forms of interfering RNA molecules
WO2004014933A1 (en) 2002-08-07 2004-02-19 University Of Massachusetts Compositions for rna interference and methods of use thereof
US7956176B2 (en) * 2002-09-05 2011-06-07 Sirna Therapeutics, Inc. RNA interference mediated inhibition of gene expression using chemically modified short interfering nucleic acid (siNA)
AU2003273336A1 (en) * 2002-09-18 2004-04-08 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
US9827263B2 (en) * 2002-11-05 2017-11-28 Ionis Pharmaceuticals, Inc. 2′-methoxy substituted oligomeric compounds and compositions for use in gene modulations
US9150605B2 (en) * 2002-11-05 2015-10-06 Isis Pharmaceuticals, Inc. Compositions comprising alternating 2′-modified nucleosides for use in gene modulation
US8604183B2 (en) * 2002-11-05 2013-12-10 Isis Pharmaceuticals, Inc. Compositions comprising alternating 2′-modified nucleosides for use in gene modulation
US9150606B2 (en) * 2002-11-05 2015-10-06 Isis Pharmaceuticals, Inc. Compositions comprising alternating 2'-modified nucleosides for use in gene modulation
CA2519860C (en) * 2003-03-21 2018-01-16 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
US7750144B2 (en) 2003-06-02 2010-07-06 University Of Massachusetts Methods and compositions for enhancing the efficacy and specificity of RNA silencing
DK1633767T3 (en) * 2003-06-02 2019-03-25 Univ Massachusetts METHODS AND COMPOSITIONS FOR MANAGING THE EFFECT OF RNA SILENCING
ES2357116T5 (es) 2003-06-02 2021-09-03 Univ Massachusetts Métodos y composiciones para mejorar la eficacia y especificad de FNAi
US8575327B2 (en) 2003-06-12 2013-11-05 Alnylam Pharmaceuticals, Inc. Conserved HBV and HCV sequences useful for gene silencing
EP1644475A4 (de) * 2003-06-20 2009-06-03 Isis Pharmaceuticals Inc Doppelstrang-zusammensetzungen mit einem 3'-endo-modifizierten strang zur verwendung bei der genmodulation
US7683036B2 (en) * 2003-07-31 2010-03-23 Regulus Therapeutics Inc. Oligomeric compounds and compositions for use in modulation of small non-coding RNAs
US20050136437A1 (en) * 2003-08-25 2005-06-23 Nastech Pharmaceutical Company Inc. Nanoparticles for delivery of nucleic acids and stable double-stranded RNA
CN1845993B (zh) 2003-08-28 2010-06-23 诺瓦提斯公司 具有平端和3'修饰的干扰rna双链体
US20050074801A1 (en) * 2003-09-09 2005-04-07 Monia Brett P. Chimeric oligomeric compounds comprising alternating regions of northern and southern conformational geometry
WO2005027980A1 (en) 2003-09-12 2005-03-31 University Of Massachusetts Rna interference for the treatment of gain-of-function disorders
US8680063B2 (en) 2003-09-12 2014-03-25 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
WO2005073378A1 (en) * 2004-01-30 2005-08-11 Santaris Pharma A/S MODIFIED SHORT INTERFERING RNA (MODIFIED siRNA)
JP4755113B2 (ja) 2004-01-30 2011-08-24 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 線維性状態およびその他の疾患の治療のための、オリゴリボヌクレオチドおよびその使用方法
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
EP2290071B1 (de) 2004-05-28 2014-12-31 Asuragen, Inc. Verfahren und Zusammensetzungen mit MicroRNA
US8394947B2 (en) 2004-06-03 2013-03-12 Isis Pharmaceuticals, Inc. Positionally modified siRNA constructs
EP1766052A4 (de) * 2004-06-03 2009-12-16 Isis Pharmaceuticals Inc Chimärische oligomere zusammensetzungen mit gap
AU2005252663B2 (en) * 2004-06-03 2011-07-07 Isis Pharmaceuticals, Inc. Double strand compositions comprising differentially modified strands for use in gene modulation
US20090048192A1 (en) * 2004-06-03 2009-02-19 Isis Pharmaceuticals, Inc. Double Strand Compositions Comprising Differentially Modified Strands for Use in Gene Modulation
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
HRP20171010T8 (hr) 2004-08-23 2017-11-03 Sylentis S.A.U. Liječenje poremećaja očiju koji su karakterizirani povišenim intraokularnim pritiskom pomoću sirnk
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
CA2857881A1 (en) 2004-11-12 2006-12-28 Asuragen, Inc. Methods and compositions involving mirna and mirna inhibitor molecules
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
US7923207B2 (en) 2004-11-22 2011-04-12 Dharmacon, Inc. Apparatus and system having dry gene silencing pools
US20060160110A1 (en) * 2004-12-02 2006-07-20 Takayuki Mizutani Methods of designing small interfering RNAs, antisense polynucleotides, and other hybridizing polynucleotides
US9550990B2 (en) * 2004-12-10 2017-01-24 Ionis Pharmaceuticals, 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
EP2239328A3 (de) * 2005-08-18 2011-01-05 Alnylam Pharmaceuticals Inc. Verfahren und Zusammensetzungen zur Behandlung von neurologischen Erkrankungen
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
CA2628113A1 (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
NL2000439C2 (nl) * 2006-01-20 2009-03-16 Quark Biotech Therapeutische toepassingen van inhibitoren van 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
EP1984499B1 (de) * 2006-01-27 2015-05-27 Isis Pharmaceuticals, Inc. Oligomere verbindungen und zusammensetzungen zur verwendung bei der modulation von mikro-rnas
EP1989307B1 (de) * 2006-02-08 2012-08-08 Quark Pharmaceuticals, Inc. Neue 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
WO2007107162A2 (en) * 2006-03-23 2007-09-27 Santaris Pharma 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
AU2007276388A1 (en) 2006-07-21 2008-01-24 Silence Therapeutics Ag Means for inhibiting the expression of protein kinase 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
EP2076599A2 (de) * 2006-09-19 2009-07-08 Asuragen, Inc. Mir-200-regulierte gene und wege als ziele für einen therapeutischen eingriff
WO2008036841A2 (en) 2006-09-22 2008-03-27 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
WO2008052774A2 (en) 2006-10-31 2008-05-08 Noxxon Pharma Ag Methods for detection of a single- or double-stranded nucleic acid molecule
US7872119B2 (en) 2007-02-26 2011-01-18 Quark Pharmaceuticals, Inc. Inhibitors of RTP801 and their use in disease treatment
US20100292301A1 (en) * 2007-02-28 2010-11-18 Elena Feinstein 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
WO2008152636A2 (en) * 2007-06-15 2008-12-18 Quark Pharmaceuticals, Inc. Compositions and methods for inhibiting nadph oxidase expression
WO2009001359A2 (en) 2007-06-27 2008-12-31 Quark Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of pro-apoptotic genes
US8361714B2 (en) 2007-09-14 2013-01-29 Asuragen, Inc. Micrornas differentially expressed in cervical cancer and uses thereof
DK2548962T3 (en) 2007-09-19 2016-04-11 Applied Biosystems Llc Sirna sequence-independent modification formats to reduce off-target phenotype effects in RNAI and stabilized forms thereof
AU2008306455C1 (en) 2007-10-03 2014-04-17 Quark Pharmaceuticals, Inc. Novel siRNA structures
CN101889087B (zh) * 2007-11-29 2013-04-03 苏州瑞博生物技术有限公司 一种干扰靶基因表达的复合分子及其制备方法
US8071562B2 (en) 2007-12-01 2011-12-06 Mirna Therapeutics, Inc. MiR-124 regulated genes and pathways as targets for therapeutic intervention
US8614311B2 (en) 2007-12-12 2013-12-24 Quark Pharmaceuticals, Inc. RTP801L siRNA compounds and methods of use thereof
US20110105584A1 (en) * 2007-12-12 2011-05-05 Elena Feinstein Rtp80il 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
WO2009090639A2 (en) * 2008-01-15 2009-07-23 Quark Pharmaceuticals, Inc. Sirna compounds and methods of use thereof
US10131904B2 (en) 2008-02-11 2018-11-20 Rxi Pharmaceuticals Corporation Modified RNAi polynucleotides and uses thereof
US8188060B2 (en) 2008-02-11 2012-05-29 Dharmacon, Inc. Duplex oligonucleotides with enhanced functionality in gene regulation
EP2256191A4 (de) * 2008-02-15 2011-07-06 Riken Cyclischer einzelstrangnukleinsäure-komplex und verfahren zur herstellung davon
US20110028531A1 (en) * 2008-03-20 2011-02-03 Elena Feinstein 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
JP5524189B2 (ja) 2008-06-06 2014-06-18 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 耳障害治療のための組成物および方法
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
US8815818B2 (en) 2008-07-18 2014-08-26 Rxi Pharmaceuticals Corporation Phagocytic cell delivery of RNAI
US9433684B2 (en) 2008-08-19 2016-09-06 Nektar Therapeutics Conjugates of small-interfering nucleic acids
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
CA2746527A1 (en) 2008-09-22 2010-03-25 Rxi Pharmaceuticals Corporation Rna interference in skin indications
EA036772B1 (ru) 2008-10-20 2020-12-18 Элнилэм Фармасьютикалз, Инк. Композиции и способы для ингибирования экспрессии транстиретина
ES2563984T3 (es) * 2008-10-22 2016-03-17 Quark Pharmaceuticals, Inc. Métodos para el tratamiento de trastornos oculares
WO2010048549A2 (en) 2008-10-24 2010-04-29 Isis Pharmaceuticals, Inc. 5' and 2' bis-substituted nucleosides and oligomeric compounds prepared therefrom
EP2358398A2 (de) 2008-10-24 2011-08-24 Isis Pharmaceuticals, Inc. Oligomere verbindungen und verfahren
WO2010059226A2 (en) 2008-11-19 2010-05-27 Rxi Pharmaceuticals Corporation Inhibition of map4k4 through rnai
WO2010080452A2 (en) 2008-12-18 2010-07-15 Quark Pharmaceuticals, Inc. siRNA COMPOUNDS AND METHODS OF USE THEREOF
US9493774B2 (en) 2009-01-05 2016-11-15 Rxi Pharmaceuticals Corporation Inhibition of PCSK9 through RNAi
US9745574B2 (en) 2009-02-04 2017-08-29 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
WO2010094491A1 (en) 2009-02-18 2010-08-26 Silence Therapeutics Ag Means for inhibiting the expression of ang2
WO2010107952A2 (en) * 2009-03-19 2010-09-23 Merck Sharp & Dohme Corp. RNA INTERFERENCE MEDIATED INHIBITION OF CONNECTIVE TISSUE GROWTH FACTOR (CTGF) GENE EXPRESSION USING SHORT INTERFERING NUCLEIC ACID (siNA)
CN102378766A (zh) 2009-03-23 2012-03-14 夸克医药公司 治疗癌症和纤维化疾病的化合物组合物和方法
CN102458418B (zh) 2009-06-08 2015-09-16 夸克制药公司 一种寡核苷酸化合物的制药用途
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
WO2011034811A1 (en) 2009-09-17 2011-03-24 Sigma-Aldrich Co. 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
EP2510098B1 (de) 2009-12-09 2015-02-11 Quark Pharmaceuticals, Inc. Verfahren und zusammensetzungen und zur behandlung von erkrankungen, störungen oder läsionen des zns
KR101718534B1 (ko) 2009-12-09 2017-03-22 닛토덴코 가부시키가이샤 Hsp47 발현의 조절
WO2011084193A1 (en) 2010-01-07 2011-07-14 Quark Pharmaceuticals, Inc. Oligonucleotide compounds comprising non-nucleotide overhangs
CN106074591B (zh) 2010-03-24 2020-01-14 菲奥医药公司 眼部症候中的rna干扰
BR112012024049A2 (pt) 2010-03-24 2017-03-01 Rxi Pharmaceuticals Corp interferência de rna em indicações dérmicas e fibróticas
US9080171B2 (en) 2010-03-24 2015-07-14 RXi Parmaceuticals Corporation Reduced size self-delivering RNAi compounds
WO2011139699A2 (en) 2010-04-28 2011-11-10 Isis Pharmaceuticals, Inc. 5' modified nucleosides and oligomeric compounds prepared therefrom
KR101869570B1 (ko) 2010-04-28 2018-06-20 아이오니스 파마수티컬즈, 인코포레이티드 변형된 뉴클레오사이드 및 그로부터 제조된 올리고머 화합물
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
KR101553753B1 (ko) 2010-06-24 2015-09-16 쿠아크 파마수티칼스 인코퍼레이티드 Rhoa에 대한 이중 가닥 rna 및 그의 용도
HUE037500T2 (hu) * 2010-07-08 2018-08-28 Bonac Corp Egyszálú nukleinsav molekula a génexpresszió szabályozására
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
US8691782B2 (en) 2010-08-03 2014-04-08 Bonac Corporation Single-stranded nucleic acid molecule having nitrogen-containing alicyclic skeleton
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
DK2631291T3 (da) 2010-10-22 2019-06-11 Olix Pharmaceuticals Inc Nukleinsyremolekyler, der inducerer rna-interferens, og anvendelser deraf
WO2012058210A1 (en) 2010-10-29 2012-05-03 Merck Sharp & Dohme Corp. RNA INTERFERENCE MEDIATED INHIBITION OF GENE EXPRESSION USING SHORT INTERFERING NUCLEIC ACIDS (siNA)
AU2011338682B2 (en) 2010-12-06 2017-04-27 Quark Pharmaceuticals, Inc. Double stranded oligonucleotide compounds comprising threose modifications
US9796979B2 (en) 2011-03-03 2017-10-24 Quark Pharmaceuticals Inc. Oligonucleotide modulators of the toll-like receptor pathway
CN103562387A (zh) 2011-03-03 2014-02-05 夸克医药公司 Toll样受体途径的寡核苷酸调剂
CN103492572A (zh) 2011-03-03 2014-01-01 夸克医药公司 用于治疗肺疾病和损伤的组合物和方法
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
US10196637B2 (en) 2011-06-08 2019-02-05 Nitto Denko Corporation Retinoid-lipid drug carrier
HRP20181564T1 (hr) 2011-06-30 2018-11-30 Arrowhead Pharmaceuticals, Inc. Pripravci i postupci za inhibiciju ekspresije gena virusa hepatitisa b
WO2013040251A2 (en) 2011-09-13 2013-03-21 Asurgen, Inc. Methods and compositions involving mir-135b for distinguishing pancreatic cancer from benign pancreatic disease
JP6294229B2 (ja) 2011-10-18 2018-03-14 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Dicerna Pharmaceuticals, Inc. アミン陽イオン性脂質およびその使用
US20140323549A1 (en) 2011-11-08 2014-10-30 Quark Pharmaceuticals, Inc. Methods and compositions for treating diseases, disorders or injury of the nervous system
HUE048622T2 (hu) 2011-11-18 2020-08-28 Alnylam Pharmaceuticals Inc RNSi szerek, készítmények és alkalmazási eljárások transztiretin (TTR) asszociált betegségek kezelésére
EP2780454A2 (de) 2011-11-18 2014-09-24 Alnylam Pharmaceuticals, Inc. Modifizierte rnai-mittel
JP2015509085A (ja) 2012-01-01 2015-03-26 キュービーアイ エンタープライゼズ リミテッドQbi Enterprises Ltd. 治療剤および診断剤の選択的送達のためのendo180を標的とする粒子
EP2802657B1 (de) 2012-01-12 2018-05-02 Quark Pharmaceuticals, Inc. Kombinationstherapie zur behandlung von gehör-und gleichgewichtsstörungen
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
CN108148838A (zh) 2012-05-22 2018-06-12 奥利克斯医药有限公司 具有细胞内穿透能力的诱导rna干扰的核酸分子及用途
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
SG11201501385UA (en) 2012-09-05 2015-03-30 Sylentis Sau Sirna and their use in methods and compositions for the treatment and/or prevention of eye conditions
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
US9932578B2 (en) 2012-09-12 2018-04-03 Quark Pharmaceuticals, Inc. Double-stranded oligonucleotide molecules to P53 and methods of use thereof
DK2895608T3 (en) 2012-09-12 2019-01-21 Quark Pharmaceuticals Inc DOUBLE-STRENGTHED OIGONUCLEOTIDE MOLECULES FOR P53 AND PROCEDURES FOR USING IT
WO2014153163A1 (en) 2013-03-14 2014-09-25 Dicerna Pharmaceuticals, Inc. Process for formulating an anionic agent
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
US9889200B2 (en) 2013-07-31 2018-02-13 Qbi Enterprises Ltd. Sphingolipid-polyalkylamine-oligonucleotide compounds
EP3027223A1 (de) 2013-07-31 2016-06-08 QBI Enterprises Ltd. Verfahren zur verwendung von sphingolipid-polyalkylamin-oligonukleotidverbindungen
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
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
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
WO2015084897A2 (en) 2013-12-02 2015-06-11 Mirimmune, Llc Immunotherapy of cancer
CN105899663B (zh) 2013-12-26 2019-07-26 学校法人东京医科大学 用于基因表达控制的人工模拟miRNA及其用途
SG10201913570XA (en) 2013-12-27 2020-03-30 Bonac Corp Artificial match-type mirna for controlling gene expression and use therefor
KR20160108494A (ko) * 2014-01-17 2016-09-19 교와 핫꼬 기린 가부시키가이샤 β2GPI의 발현을 억제하는 핵산
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
WO2016022648A1 (en) * 2014-08-07 2016-02-11 Industrial Technology Research Institute Small interfering rna and pharmaceutical composition and method for inhibiting galectin-12 expression and/or enhancing lipolysis containing the same
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
TWI710633B (zh) 2014-11-10 2020-11-21 美商阿尼拉製藥公司 B型肝炎病毒(HBV)iRNA組成物及其用途方法
WO2016083624A1 (en) 2014-11-28 2016-06-02 Silence Therapeutics Gmbh Means for inhibiting the expression of edn1
EP4372091A3 (de) 2014-12-12 2024-07-31 Tod M. Woolf Zusammensetzungen und verfahren zur bearbeitung von nukleinsäuren in zellen unter verwendung von oligonukleotiden
SG11201705223XA (en) 2014-12-27 2017-07-28 Bonac Corp NATURALLY OCCURING miRNA FOR CONTROLLING GENE EXPRESSION, AND USE OF SAME
US20160272970A1 (en) * 2015-03-17 2016-09-22 Arrowhead Madison Inc. RNA Interference Agents
JP6602847B2 (ja) 2015-03-27 2019-11-06 株式会社ボナック デリバリー機能と遺伝子発現制御能を有する一本鎖核酸分子
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
CN108135923B (zh) 2015-07-06 2021-03-02 菲奥医药公司 靶向超氧化物歧化酶1(sod1)的核酸分子
CN108138182B (zh) 2015-07-31 2022-08-19 阿尔尼拉姆医药品有限公司 甲状腺素运载蛋白(TTR)iRNA组合物及其治疗或预防TTR相关疾病的使用方法
KR20180038465A (ko) 2015-08-07 2018-04-16 애로우헤드 파마슈티컬스 인코포레이티드 B형 간염 바이러스 감염에 대한 rnai 치료법
KR102708889B1 (ko) 2015-09-08 2024-09-25 실렌티스 에스.에이.유. siRNA, 및 NRARP 유전자의 발현을 억제하기 위한 방법 및 조성물에서의 이의 용도
WO2017070151A1 (en) 2015-10-19 2017-04-27 Rxi Pharmaceuticals Corporation Reduced size self-delivering nucleic acid compounds targeting long non-coding rna
US11273151B2 (en) 2015-11-04 2022-03-15 Icahn School Of Medicine At Mount Sinai Methods of treating tumors and cancer, and identifying candidate subjects for such treatment
EP4454637A3 (de) 2015-11-16 2025-01-08 Olix Pharmaceuticals, Inc. Behandlung von altersbedingter makuladegeneration mit auf myd88 oder tlr3 abzielenden rna-komplexen
WO2017131124A1 (ja) 2016-01-26 2017-08-03 日産化学工業株式会社 一本鎖オリゴヌクレオチド
CA3022877A1 (en) 2016-02-02 2017-08-10 Olix Pharmaceuticals, Inc. Treatment of angiogenesis-associated diseases using rna complexes that target angpt2 and pdgfb
KR102825946B1 (ko) 2016-02-02 2025-06-27 올릭스 주식회사 IL4Rα, TRPA1, 또는 F2RL1을 표적화하는 RNA 복합체를 사용한 아토피 피부염 및 천식의 치료
WO2023150553A1 (en) 2022-02-01 2023-08-10 University Of Rochester Gpr17 promoter-based targeting and transduction of glial progenitor cells
JP2019513371A (ja) 2016-04-01 2019-05-30 アビディティー バイオサイエンシーズ エルエルシー 核酸ポリペプチド組成物とその使用
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
US11246868B2 (en) 2016-04-26 2022-02-15 Icahn School Of Medicine At Mount Sinai Treatment of hippo pathway mutant tumors and methods of identifying subjects as candidates for treatment
JP6872560B2 (ja) 2016-05-06 2021-05-19 エム. ウルフ、トッド プログラム可能ヌクレアーゼのあるゲノム編集及びプログラム可能ヌクレアーゼのないゲノム編集のための改善された方法
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
EP4035659A1 (de) 2016-11-29 2022-08-03 PureTech LYT, Inc. Exosome zur ausgabe von therapeutischen wirkstoffen
MX2019008199A (es) 2017-01-06 2019-11-25 Avidity Biosciences Llc Composiciones de acido nucleico polipeptido y metodos de induccion de la omision de exon.
CN110337495B (zh) 2017-02-06 2024-03-29 日产化学株式会社 单链寡核苷酸
US11591600B2 (en) 2017-02-10 2023-02-28 OliX Pharmaceuticals. Inc. Long double-stranded RNA for RNA interference
US11324820B2 (en) 2017-04-18 2022-05-10 Alnylam Pharmaceuticals, Inc. Methods for the treatment of subjects having a hepatitis b virus (HBV) infection
WO2018195355A1 (en) 2017-04-19 2018-10-25 Rxi Pharmaceuticals Corporation Topical delivery of nucleic acid compounds
GB201711809D0 (en) 2017-07-21 2017-09-06 Governors Of The Univ Of Alberta Antisense oligonucleotide
WO2019048645A1 (en) * 2017-09-08 2019-03-14 Mina Therapeutics Limited STABILIZED COMPOSITIONS OF SMALL ACTIVATOR RNA (PARNA) FROM CEBPA AND METHODS OF USE
CA3075205A1 (en) 2017-09-08 2019-03-14 Mina Therapeutics Limited Stabilized hnf4a sarna compositions and methods of use
KR20250046363A (ko) 2017-09-19 2025-04-02 알닐람 파마슈티칼스 인코포레이티드 트랜스타이레틴(ttr) 매개 아밀로이드증을 치료하기 위한 조성물 및 방법
IL274611B2 (en) * 2017-11-13 2024-10-01 Silence Therapeutics Gmbh Nucleic acids for inhibiting expression of a target gene comprising phosphorodithioate linkages
JP7506601B2 (ja) 2017-12-06 2024-06-26 アビディティー バイオサイエンシーズ,インク. 筋萎縮症と筋緊張性ジストロフィーを処置するための組成物および方法
US11648260B2 (en) 2018-03-29 2023-05-16 Technion Research And Development Foundation Limitted Vesicles comprising a PTEN inhibitor and uses of same
CN112566640A (zh) 2018-06-18 2021-03-26 罗切斯特大学 治疗精神分裂症和其他神经精神病症的方法
WO2019246262A2 (en) 2018-06-21 2019-12-26 University Of Rochester Methods of treating or inhibiting onset of huntington's disease
CA3106701A1 (en) 2018-08-13 2020-02-20 Alnylam Pharmaceuticals, Inc. Hepatitis b virus (hbv) dsrna agent compositions and methods of use thereof
CA3122289A1 (en) 2018-12-11 2020-06-18 University Of Rochester Methods of treating schizophrenia and other neuropsychiatric disorders
IL312067B2 (en) 2018-12-21 2025-08-01 Avidity Biosciences Inc Anti-transferrin receptor antibodies and uses thereof
WO2020167822A2 (en) 2019-02-13 2020-08-20 University Of Rochester Gene networks that mediate remyelination of the human brain
US12497615B2 (en) 2019-04-25 2025-12-16 Avidity Biosciences, Inc. Nucleic acid compositions and methods of multi-exon skipping
US20220267737A1 (en) 2019-07-18 2022-08-25 University Of Rochester Cell-type selective immunoprotection of cells
CA3151110A1 (en) 2019-08-16 2021-02-25 Children's Hospital Medical Center Methods of treating a subject with a cdc42-specific inhibitor
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
US11555190B2 (en) 2020-03-19 2023-01-17 Avidity Biosciences, Inc. Compositions and methods of treating Facioscapulohumeral muscular dystrophy
AU2021241682B2 (en) 2020-03-27 2026-02-12 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
WO2022162157A1 (en) 2021-01-30 2022-08-04 E-Therapeutics Plc Conjugated oligonucleotide compounds, methods of making and uses thereof
EP4175964A1 (de) 2021-01-30 2023-05-10 E-Therapeutics plc Konjugierte oligonukleotidverbindungen, verfahren zur herstellung und verwendungen davon
WO2022162155A1 (en) 2021-01-30 2022-08-04 E-Therapeutics Plc Nucleic acids containing abasic nucleotides
CA3204340A1 (en) 2021-01-30 2022-08-04 Ahmad Ali MORTAZAVI Conjugated oligonucleotide compounds, methods of making and uses thereof
CA3227852A1 (en) 2021-08-03 2023-02-09 Alnylam Pharmaceuticals, Inc. Transthyretin (ttr) irna compositions and methods of use thereof
MX2024002726A (es) 2021-09-03 2024-03-20 CureVac SE Nuevas nanoparticulas lipidicas para la administracion de acidos nucleicos.
MX2024003266A (es) 2021-09-16 2024-04-03 Avidity Biosciences Inc Composiciones y metodos de tratamiento de la distrofia muscular facioescapulohumeral.
KR20240163743A (ko) 2022-03-28 2024-11-19 엠피리코 인크. 변형된 올리고뉴클레오티드
US12071621B2 (en) 2022-04-05 2024-08-27 Avidity Biosciences, Inc. Anti-transferrin receptor antibody-PMO conjugates for inducing DMD exon 44 skipping
EP4560019A1 (de) 2023-11-22 2025-05-28 Sylentis S.A.U. Sirna und zusammensetzungen zur prophylaktischen und therapeutischen behandlung von augenerkrankungen der retina
EP4560020A1 (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
ATE269870T1 (de) 1989-10-24 2004-07-15 Isis Pharmaceuticals Inc 2'-modifizierte oligonukleotide
US5955589A (en) * 1991-12-24 1999-09-21 Isis Pharmaceuticals Inc. Gapped 2' modified oligonucleotides
US6399754B1 (en) 1991-12-24 2002-06-04 Isis Pharmaceuticals, Inc. Sugar modified oligonucleotides
US6005087A (en) 1995-06-06 1999-12-21 Isis Pharmaceuticals, Inc. 2'-modified oligonucleotides
US5914396A (en) 1990-01-11 1999-06-22 Isis Pharmaceuticals, Inc. 2'-O-modified nucleosides and phosphoramidites
US6153737A (en) 1990-01-11 2000-11-28 Isis Pharmaceuticals, Inc. Derivatized oligonucleotides having improved uptake and other properties
US5670633A (en) 1990-01-11 1997-09-23 Isis Pharmaceuticals, Inc. Sugar modified oligonucleotides that detect and modulate gene expression
US5872232A (en) 1990-01-11 1999-02-16 Isis Pharmaceuticals Inc. 2'-O-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
JP3257675B2 (ja) 1990-10-12 2002-02-18 マックス−プランク−ゲゼルシャフト ツール フェルデルング デル ビッセンシャフテン エー.ファウ. 修飾リボザイム
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)
NZ255028A (en) 1992-07-02 1997-03-24 Hybridon Inc Antisense oligonucleotides resistant to nucleolytic degradation
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)
GB9425118D0 (en) 1994-12-13 1995-02-08 Henkel Kgaa Two component natural hair colorant
CA2207593A1 (en) 1994-12-13 1996-06-20 John Gustofson Method and reagent for treatment of arthritic conditions, induction of graft tolerance and reversal of immune responses
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
US6129912A (en) 1997-09-26 2000-10-10 Uab Research Foundation Polyethylene glycol-protein compositions which reduce the antigenic display of red blood cells
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
WO1999049029A1 (en) 1998-03-20 1999-09-30 Benitec Australia Ltd Control of gene expression
NZ507093A (en) 1998-04-08 2003-08-29 Commw Scient Ind Res Org Methods and means for reducing the phenotypic expression of a nucleic acid of interest in a plant
AU3751299A (en) 1998-04-20 1999-11-08 Ribozyme Pharmaceuticals, Inc. Nucleic acid molecules with novel chemical compositions capable of modulating gene expression
EP1073732A2 (de) 1998-04-29 2001-02-07 Ribozyme Pharmaceuticals, Inc. Nukleosid-triphosphate und deren einschläusung in ribozyme
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
EP2314700A1 (de) 1999-01-28 2011-04-27 Medical College of Georgia Research Institute, Inc Zusammensetzung und Verfahren zur in vivo und in vitro Abschwächung der Genexpression mittels dobbelsträngiger 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
WO2000063364A2 (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
DE10160151A1 (de) 2001-01-09 2003-06-26 Ribopharma Ag Verfahren zur Hemmung der Expression eines vorgegebenen Zielgens
US7829693B2 (en) 1999-11-24 2010-11-09 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of a target gene
WO2003070918A2 (en) 2002-02-20 2003-08-28 Ribozyme Pharmaceuticals, Incorporated Rna interference by modified short interfering nucleic acid
PT1309726E (pt) 2000-03-30 2010-03-08 Whitehead Biomedical Inst Mediadores de interferência por rna específicos de sequência de rna
AU2001284160A1 (en) 2000-08-19 2002-03-04 Axordia Limited Modulation of 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
US20040259247A1 (en) 2000-12-01 2004-12-23 Thomas Tuschl Rna interference mediating small rna molecules
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)
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)
WO2002097114A2 (en) 2001-05-29 2002-12-05 Sirna Therapeutics, Inc. Nucleic acid treatment of diseases or conditions related to levels of ras, her2 and hiv
ATE487744T1 (de) 2001-06-22 2010-11-15 Borealis Tech Oy Metallocenkatalysatoren mit einer in 4-, 5-, 6- oder 7-stellung mit siloxy oder germyloxy substitutierten indenylgruppe
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
DE60325016D1 (de) 2002-02-01 2009-01-15 Univ Mcgill Oligonukleotide mit alternierenden segmenten und deren verwendungen
US20030166282A1 (en) 2002-02-01 2003-09-04 David Brown 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
AU2003260370B2 (en) 2002-08-05 2008-05-22 Silence Therapeutics Gmbh Further novel forms of interfering RNA molecules
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
EP2072619A1 (de) 2002-10-18 2009-06-24 Silence Therapeutics Aktiengesellschaft Neuer Faktor für Metastase und Verwendungen davon
US7289445B2 (en) * 2002-11-25 2007-10-30 Intel Corporation Managing a protocol control block cache in a network device
AU2004263830B2 (en) 2003-06-13 2008-12-18 Alnylam Pharmaceuticals, Inc. Double-stranded ribonucleic acid with increased effectiveness in an organism
WO2005000320A2 (en) 2003-06-27 2005-01-06 Atugen Ag Use of double-stranded ribonucleic acid for inducing cell lysis
AU2005222902B2 (en) 2004-03-12 2010-06-10 Alnylam Pharmaceuticals, Inc. iRNA agents targeting 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
SI1771206T1 (en) 2004-05-05 2018-06-29 Silence Therapeutics Gmbh Lipids, lipid complexes and use thereof
EP2399924B1 (de) 2004-05-27 2015-01-28 Alnylam Pharmaceuticals, Inc. Nukleaseresistente doppelsträngige Ribonukleinsäure
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
CN101287497B (zh) 2004-12-27 2013-03-06 赛伦斯治疗公司 涂层脂质复合体和它们的用途
US20070027097A1 (en) 2005-07-28 2007-02-01 Lewis David L Small interfering RNA with improved activity
MX2008005508A (es) 2005-10-28 2008-11-18 Topigen Pharmaceuticals Inc Pequeños oligonucleotidos de interferencia que comprenden nucleotidos modificados de arabinosa.
US8101741B2 (en) 2005-11-02 2012-01-24 Protiva Biotherapeutics, Inc. Modified siRNA molecules and uses thereof
NL2000439C2 (nl) 2006-01-20 2009-03-16 Quark Biotech Therapeutische toepassingen van inhibitoren van RTP801.
US7541994B2 (en) 2006-05-17 2009-06-02 Raytheon Company Refractive compact range
AU2008306455C1 (en) 2007-10-03 2014-04-17 Quark Pharmaceuticals, Inc. Novel siRNA structures

Also Published As

Publication number Publication date
US9758784B1 (en) 2017-09-12
DE60310944T3 (de) 2017-08-03
US7893245B2 (en) 2011-02-22
EP3222724B1 (de) 2018-10-31
EP1527176B1 (de) 2007-01-03
IL166546A0 (en) 2006-01-15
US10329568B2 (en) 2019-06-25
EP2258847B2 (de) 2020-07-01
JP2017163995A (ja) 2017-09-21
US20040180351A1 (en) 2004-09-16
US20180223286A1 (en) 2018-08-09
EP1857547A3 (de) 2011-05-18
US10266829B2 (en) 2019-04-23
US10774332B2 (en) 2020-09-15
US20170247698A1 (en) 2017-08-31
AU2008207337A1 (en) 2008-09-04
US9783802B2 (en) 2017-10-10
US20160130587A1 (en) 2016-05-12
JP2016144451A (ja) 2016-08-12
BR0313202A (pt) 2005-06-21
PT1527176E (pt) 2007-04-30
EP3222725A1 (de) 2017-09-27
US20110118456A1 (en) 2011-05-19
CN1675359A (zh) 2005-09-28
US20170247707A1 (en) 2017-08-31
US20170247697A1 (en) 2017-08-31
IL166546A (en) 2014-01-30
EP1857547B2 (de) 2020-12-02
US20190024089A1 (en) 2019-01-24
US9695423B2 (en) 2017-07-04
JP2011024592A (ja) 2011-02-10
US9790505B2 (en) 2017-10-17
DK2258847T3 (en) 2017-06-26
CA2494930A1 (en) 2004-02-19
DK1527176T3 (da) 2007-05-07
EP1857547A2 (de) 2007-11-21
US9222092B2 (en) 2015-12-29
DK1857547T3 (en) 2018-04-23
ES2280826T3 (es) 2007-09-16
CY1120919T1 (el) 2019-12-11
MXPA05001355A (es) 2005-09-30
US20150105545A1 (en) 2015-04-16
CA2494930C (en) 2021-03-09
KR101201664B1 (ko) 2012-11-15
SI1857547T1 (en) 2018-05-31
US20190390201A1 (en) 2019-12-26
JP6069096B2 (ja) 2017-01-25
US20180291381A1 (en) 2018-10-11
DK3222724T3 (en) 2018-12-03
ZA200500459B (en) 2005-08-31
US20210062198A1 (en) 2021-03-04
DE60310944D1 (de) 2007-02-15
KR20050035877A (ko) 2005-04-19
US20130102769A1 (en) 2013-04-25
EP1857547B1 (de) 2018-01-17
US9790501B2 (en) 2017-10-17
EP1527176A2 (de) 2005-05-04
EP3222724A1 (de) 2017-09-27
AU2003260370B2 (en) 2008-05-22
WO2004015107A3 (en) 2004-04-29
US10323246B2 (en) 2019-06-18
JP2013179944A (ja) 2013-09-12
JP5490655B2 (ja) 2014-05-14
AU2003260370A1 (en) 2004-02-25
EP2258847A2 (de) 2010-12-08
EP2258847B1 (de) 2017-03-15
US20170247691A1 (en) 2017-08-31
DK1527176T4 (en) 2017-07-03
SI3222724T1 (sl) 2019-03-29
US8933215B2 (en) 2015-01-13
DE60310944T2 (de) 2007-10-11
EP2258847A3 (de) 2011-05-18
US20090186845A1 (en) 2009-07-23
EP1527176B2 (de) 2017-03-22
US7452987B2 (en) 2008-11-18
CY1120128T1 (el) 2018-12-12
CN100543137C (zh) 2009-09-23
SI2258847T1 (sl) 2017-08-31
US11578328B2 (en) 2023-02-14
US8324370B2 (en) 2012-12-04
EP3222726A1 (de) 2017-09-27
ES2280826T5 (es) 2017-08-03
CY1119021T1 (el) 2018-01-10
BRPI0313202A8 (pt) 2016-08-16
CY1106422T1 (el) 2011-10-12
WO2004015107A2 (en) 2004-02-19

Similar Documents

Publication Publication Date Title
ATE350473T2 (de) Weitere neue formen von interferierende rns moleküle
EA200701901A1 (ru) Нуклеиновые кислоты, связывающие грелин
WO2004007718A3 (en) Rna-interference by single-stranded rna molecules
WO2005014782A3 (en) Double-stranded ribonucleic acid with increased effectiveness in an organism
DE60330842D1 (de) Für den nachweis von nukleinsäuren kompetente biologische rohderivate
ATE367441T1 (de) Lipophile derivate doppelsträngiger ribonukleinsäure
DK2385141T3 (da) Flerkomponentnukleinsyreenzymer og fremgangsmåder til anvendelse heraf
ATE525469T1 (de) Nachweis kleiner nukleinsäuren
ATE425263T1 (de) Multiplex-oligonukleotidaddition und zielamplifikation
BR9913360A (pt) Oxidase de urato
ATE328075T1 (de) Verfahren zur hemmung der expression eine zielgens
WO2006083800A3 (en) Nucleic acid silencing of huntington's disease gene
WO2006110688A3 (en) Rnai therapeutic for respiratory virus infection
WO2004065600A3 (en) Rna interference by palindromic or modified rna molecules
BR9809433A (pt) Moléculas de dna enzimáticas
ATE439048T1 (de) Prozess zur verminderung des acrylamidgehaltes von hitzebehandelten lebensmitteln
WO2005021786A8 (en) A method of sequencing nucleic acids by ligation of labelled oligonucleotides
BRPI0517131A2 (pt) mÉtodos para simplificar Ácidos nuclÉicos microbiais por modificaÇço quÍmica de citosinas
GB2409457B (en) Method to generate or determine nucleic acid tags corresponding to the terminal ends of DNA molecules using sequence analysis of gene expression(terminal SAGE
ATE242261T1 (de) Fluoreszend-konjugaten von nukleosiden und von nukleotiden, ihrer verfahren zur herstellung und ihrer verwendungen
DE602005022443D1 (de) Schnelle synthese von oligonukleotiden
WO2005000320A3 (en) Use of double-stranded ribonucleic acid for inducing cell lysis
JP2006521824A5 (de)
DE69902799D1 (de) Von kreisförmiger Matrize abhängige Kettenreaktion
ATE420975T1 (de) Methode zur linearen nicht-selektiven amplifikation von nukleinsäuren

Legal Events

Date Code Title Description
UEP Publication of translation of european patent specification

Ref document number: 1527176

Country of ref document: EP

EEFA Change of the company name