CN101346393B - 修饰的siRNA分子及其应用 - Google Patents
修饰的siRNA分子及其应用 Download PDFInfo
- Publication number
- CN101346393B CN101346393B CN200680049146.9A CN200680049146A CN101346393B CN 101346393 B CN101346393 B CN 101346393B CN 200680049146 A CN200680049146 A CN 200680049146A CN 101346393 B CN101346393 B CN 101346393B
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- Prior art keywords
- sirna
- modification
- nucleotide
- lipid
- nucleic acid
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- C12N15/09—Recombinant DNA-technology
- C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
- C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K31/00—Medicinal preparations containing organic active ingredients
- A61K31/70—Carbohydrates; Sugars; Derivatives thereof
- A61K31/7088—Compounds having three or more nucleosides or nucleotides
- A61K31/713—Double-stranded nucleic acids or oligonucleotides
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K9/00—Medicinal preparations characterised by special physical form
- A61K9/10—Dispersions; Emulsions
- A61K9/127—Liposomes
- A61K9/1271—Non-conventional liposomes, e.g. PEGylated liposomes, liposomes coated with polymers
- A61K9/1272—Non-conventional liposomes, e.g. PEGylated liposomes, liposomes coated with polymers with substantial amounts of non-phosphatidyl, i.e. non-acylglycerophosphate, surfactants as bilayer-forming substances, e.g. cationic lipids
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- C12N15/09—Recombinant DNA-technology
- C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
- C12N15/111—General methods applicable to biologically active non-coding nucleic acids
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/10—Type of nucleic acid
- C12N2310/14—Type of nucleic acid interfering N.A.
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/35—Nature of the modification
- C12N2310/351—Conjugate
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2320/00—Applications; Uses
- C12N2320/30—Special therapeutic applications
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2320/00—Applications; Uses
- C12N2320/50—Methods for regulating/modulating their activity
- C12N2320/51—Methods for regulating/modulating their activity modulating the chemical stability, e.g. nuclease-resistance
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- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Engineering & Computer Science (AREA)
- Genetics & Genomics (AREA)
- Chemical & Material Sciences (AREA)
- Biomedical Technology (AREA)
- Molecular Biology (AREA)
- Biotechnology (AREA)
- General Health & Medical Sciences (AREA)
- Organic Chemistry (AREA)
- Zoology (AREA)
- Wood Science & Technology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- General Engineering & Computer Science (AREA)
- Biophysics (AREA)
- Biochemistry (AREA)
- Veterinary Medicine (AREA)
- Animal Behavior & Ethology (AREA)
- Pharmacology & Pharmacy (AREA)
- Medicinal Chemistry (AREA)
- Epidemiology (AREA)
- Public Health (AREA)
- Microbiology (AREA)
- Physics & Mathematics (AREA)
- Plant Pathology (AREA)
- Dispersion Chemistry (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Medicinal Preparation (AREA)
- Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
- Saccharide Compounds (AREA)
Abstract
Description
Claims (29)
Applications Claiming Priority (5)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
US73296405P | 2005-11-02 | 2005-11-02 | |
US60/732,964 | 2005-11-02 | ||
US81793306P | 2006-06-30 | 2006-06-30 | |
US60/817,933 | 2006-06-30 | ||
PCT/CA2006/001801 WO2007051303A1 (en) | 2005-11-02 | 2006-11-02 | MODIFIED siRNA MOLECULES AND USES THEREOF |
Publications (2)
Publication Number | Publication Date |
---|---|
CN101346393A CN101346393A (zh) | 2009-01-14 |
CN101346393B true CN101346393B (zh) | 2015-07-22 |
Family
ID=38005393
Family Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
CN200680049146.9A Expired - Fee Related CN101346393B (zh) | 2005-11-02 | 2006-11-02 | 修饰的siRNA分子及其应用 |
Country Status (9)
Country | Link |
---|---|
US (4) | US8101741B2 (zh) |
EP (2) | EP1948674A4 (zh) |
JP (1) | JP5336853B2 (zh) |
CN (1) | CN101346393B (zh) |
AU (1) | AU2006308765B2 (zh) |
CA (1) | CA2628300C (zh) |
HK (1) | HK1126785A1 (zh) |
IL (1) | IL191192A (zh) |
WO (1) | WO2007051303A1 (zh) |
Cited By (1)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
CN105452465A (zh) * | 2013-07-31 | 2016-03-30 | 奇比艾企业有限公司 | 鞘脂-聚烷基胺-寡核苷酸化合物 |
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Cited By (2)
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CN105452465A (zh) * | 2013-07-31 | 2016-03-30 | 奇比艾企业有限公司 | 鞘脂-聚烷基胺-寡核苷酸化合物 |
CN105452465B (zh) * | 2013-07-31 | 2019-06-21 | 奇比艾企业有限公司 | 鞘脂-聚烷基胺-寡核苷酸化合物 |
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US20090270481A1 (en) | 2009-10-29 |
EP2395012A3 (en) | 2012-03-14 |
EP2395012A2 (en) | 2011-12-14 |
EP2395012B1 (en) | 2018-04-18 |
CN101346393A (zh) | 2009-01-14 |
JP2009513151A (ja) | 2009-04-02 |
CA2628300A1 (en) | 2007-05-10 |
US9074208B2 (en) | 2015-07-07 |
CA2628300C (en) | 2018-04-17 |
US20120328668A1 (en) | 2012-12-27 |
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US8101741B2 (en) | 2012-01-24 |
US8513403B2 (en) | 2013-08-20 |
US8188263B2 (en) | 2012-05-29 |
US20140179756A1 (en) | 2014-06-26 |
WO2007051303A1 (en) | 2007-05-10 |
AU2006308765B2 (en) | 2013-09-05 |
HK1126785A1 (zh) | 2009-09-11 |
IL191192A (en) | 2012-06-28 |
AU2006308765A8 (en) | 2008-06-05 |
JP5336853B2 (ja) | 2013-11-06 |
EP1948674A4 (en) | 2009-02-04 |
EP2395012B8 (en) | 2018-06-06 |
US20070135372A1 (en) | 2007-06-14 |
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