ATE367441T1 - Lipophile derivate doppelsträngiger ribonukleinsäure - Google Patents
Lipophile derivate doppelsträngiger ribonukleinsäureInfo
- Publication number
- ATE367441T1 ATE367441T1 AT04704041T AT04704041T ATE367441T1 AT E367441 T1 ATE367441 T1 AT E367441T1 AT 04704041 T AT04704041 T AT 04704041T AT 04704041 T AT04704041 T AT 04704041T AT E367441 T1 ATE367441 T1 AT E367441T1
- Authority
- AT
- Austria
- Prior art keywords
- double stranded
- ribonucleic acid
- stranded ribonucleic
- lipophilic derivatives
- lipophilic
- Prior art date
Links
Classifications
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- C12N15/09—Recombinant DNA-technology
- C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
- C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
- C12N15/1131—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing against viruses
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P31/00—Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
- A61P31/12—Antivirals
- A61P31/14—Antivirals for RNA viruses
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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
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K38/00—Medicinal preparations containing peptides
-
- 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
- C12N2310/11—Antisense
- C12N2310/111—Antisense spanning the whole gene, or a large part of it
-
- 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 nucleic acids [NA]
-
- 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
- C12N2310/3515—Lipophilic moiety, e.g. cholesterol
-
- 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
- C12N2310/50—Physical structure
- C12N2310/53—Physical structure partially self-complementary or closed
-
- 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
- C12N2320/32—Special delivery means, e.g. tissue-specific
-
- 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
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Engineering & Computer Science (AREA)
- Genetics & Genomics (AREA)
- Biomedical Technology (AREA)
- Molecular Biology (AREA)
- Organic Chemistry (AREA)
- Chemical & Material Sciences (AREA)
- Wood Science & Technology (AREA)
- Biotechnology (AREA)
- General Engineering & Computer Science (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Zoology (AREA)
- General Health & Medical Sciences (AREA)
- Biophysics (AREA)
- Microbiology (AREA)
- Plant Pathology (AREA)
- Biochemistry (AREA)
- Physics & Mathematics (AREA)
- Virology (AREA)
- General Chemical & Material Sciences (AREA)
- Oncology (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Communicable Diseases (AREA)
- Medicinal Chemistry (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Pharmacology & Pharmacy (AREA)
- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Saccharide Compounds (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
Applications Claiming Priority (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| DE10302421A DE10302421A1 (de) | 2003-01-21 | 2003-01-21 | Doppelsträngige Ribonukleinsäure mit verbesserter Wirksamkeit |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| ATE367441T1 true ATE367441T1 (de) | 2007-08-15 |
Family
ID=32602867
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| AT04704041T ATE367441T1 (de) | 2003-01-21 | 2004-01-21 | Lipophile derivate doppelsträngiger ribonukleinsäure |
Country Status (10)
| Country | Link |
|---|---|
| US (1) | US20060178324A1 (de) |
| EP (2) | EP1587926B1 (de) |
| AT (1) | ATE367441T1 (de) |
| AU (2) | AU2004206255B2 (de) |
| CA (1) | CA2513809C (de) |
| DE (2) | DE10302421A1 (de) |
| DK (1) | DK1587926T3 (de) |
| ES (1) | ES2289474T3 (de) |
| PT (1) | PT1587926E (de) |
| WO (1) | WO2004065601A2 (de) |
Families Citing this family (92)
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| US7767802B2 (en) | 2001-01-09 | 2010-08-03 | Alnylam Pharmaceuticals, Inc. | Compositions and methods for inhibiting expression of anti-apoptotic genes |
| US7423142B2 (en) | 2001-01-09 | 2008-09-09 | Alnylam Pharmaceuticals, Inc. | Compositions and methods for inhibiting expression of anti-apoptotic genes |
| EP1532248B1 (de) | 2002-07-26 | 2009-04-01 | Novartis Vaccines and Diagnostics, Inc. | Modifizierte kleine irns moleküle und methoden zu deren anwendung |
| US7723509B2 (en) | 2003-04-17 | 2010-05-25 | Alnylam Pharmaceuticals | IRNA agents with biocleavable tethers |
| US8796436B2 (en) | 2003-04-17 | 2014-08-05 | Alnylam Pharmaceuticals, Inc. | Modified iRNA agents |
| US7851615B2 (en) | 2003-04-17 | 2010-12-14 | Alnylam Pharmaceuticals, Inc. | Lipophilic conjugated iRNA agents |
| AU2004233092C9 (en) | 2003-04-17 | 2010-10-28 | Alnylam Pharmaceuticals, Inc. | Modified iRNA agents |
| US8017762B2 (en) | 2003-04-17 | 2011-09-13 | Alnylam Pharmaceuticals, Inc. | Modified iRNA agents |
| US8680063B2 (en) | 2003-09-12 | 2014-03-25 | University Of Massachusetts | RNA interference for the treatment of gain-of-function disorders |
| ES2485848T3 (es) | 2003-09-12 | 2014-08-14 | University Of Massachusetts | ARN de interferencia para el tratamiento de trastornos relacionados con la ganancia de función |
| EP2636739B1 (de) | 2004-03-12 | 2014-12-10 | Alnylam Pharmaceuticals Inc. | iRNA-Wirkstoffe für VEGF-Targeting |
| KR101147147B1 (ko) | 2004-04-01 | 2012-05-25 | 머크 샤프 앤드 돔 코포레이션 | Ｒｎａ 간섭의 오프 타겟 효과 감소를 위한 변형된폴리뉴클레오타이드 |
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| US8765704B1 (en) | 2008-02-28 | 2014-07-01 | Novartis Ag | Modified small interfering RNA molecules and methods of use |
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| JP5101288B2 (ja) | 2004-10-05 | 2012-12-19 | カリフォルニア インスティテュート オブ テクノロジー | アプタマー調節される核酸及びその利用 |
| US7935811B2 (en) | 2004-11-22 | 2011-05-03 | Dharmacon, Inc. | Apparatus and system having dry gene silencing compositions |
| US7923207B2 (en) | 2004-11-22 | 2011-04-12 | Dharmacon, Inc. | Apparatus and system having dry gene silencing pools |
| US7923206B2 (en) | 2004-11-22 | 2011-04-12 | Dharmacon, Inc. | Method of determining a cellular response to a biological agent |
| US20070161591A1 (en) * | 2005-08-18 | 2007-07-12 | University Of Massachusetts | Methods and compositions for treating neurological disease |
| EP1934348B1 (de) * | 2005-10-11 | 2018-05-02 | Ben-Gurion University Of The Negev Research And Development Authority | Zusammensetzung zum silencing der expression von vdac1 und verwendungen davon |
| JP5111385B2 (ja) | 2005-10-28 | 2013-01-09 | 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 | ハンチンチン遺伝子の発現を抑制するための組成物および方法 |
| ATE510545T1 (de) | 2005-11-09 | 2011-06-15 | Alnylam Pharmaceuticals Inc | Zusammensetzungen und verfahren zur hemmung der expression von faktor v leiden mutationsgen |
| RU2008141977A (ru) | 2006-03-24 | 2010-05-27 | Новартис АГ (CH) | КОМПОЗИЦИИ dsPHK И СПОСОБЫ ЛЕЧЕНИЯ HPV-ИНФЕКЦИИ |
| CN101448849B (zh) | 2006-03-31 | 2013-08-21 | 阿尔尼拉姆医药品有限公司 | 抑制Eg5基因表达的组合物和方法 |
| WO2007134161A2 (en) | 2006-05-11 | 2007-11-22 | Alnylam Pharmaceuticals, Inc. | Compositions and methods for inhibiting expression of the pcsk9 gene |
| ATE528008T1 (de) | 2006-05-19 | 2011-10-15 | Alnylam Pharmaceuticals Inc | Rnai-modulation von aha und ihre therapeutische verwendung |
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| WO2008058291A2 (en) | 2006-11-09 | 2008-05-15 | California Institute Of Technology | Modular aptamer-regulated ribozymes |
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| JP2024529025A (ja) | 2021-08-04 | 2024-08-01 | フィオ ファーマシューティカルズ コーポレーション | 化学修飾されたオリゴヌクレオチド |
| WO2023015264A1 (en) | 2021-08-04 | 2023-02-09 | Phio Pharmaceuticals Corp. | Immunotherapy of cancer utilizing natural killer cells treated with chemically modified oligonucleotides |
| CN115707710B (zh) * | 2021-08-18 | 2025-06-06 | 中国科学院上海有机化学研究所 | 样品衍生化试剂及其在检测旋光异构体中的应用 |
| EP4565597A2 (de) | 2022-08-03 | 2025-06-11 | Voyager Therapeutics, Inc. | Zusammensetzungen und verfahren zur durchquerung der blut-hirn-schranke |
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|---|---|---|---|---|
| US4980286A (en) * | 1985-07-05 | 1990-12-25 | Whitehead Institute For Biomedical Research | In vivo introduction and expression of foreign genetic material in epithelial cells |
| IL79289A (en) * | 1985-07-05 | 1992-01-15 | Whitehead Biomedical Inst | Introduction and expression of foreign genetic material into keratinocytes using a recombinant retrovirus |
| US6395492B1 (en) * | 1990-01-11 | 2002-05-28 | Isis Pharmaceuticals, Inc. | Derivatized oligonucleotides having improved uptake and other properties |
| US6054299A (en) * | 1994-04-29 | 2000-04-25 | Conrad; Charles A. | Stem-loop cloning vector and method |
| US6111085A (en) * | 1996-09-13 | 2000-08-29 | Isis Pharmaceuticals, Inc. | Carbamate-derivatized nucleosides and oligonucleosides |
| DE10160151A1 (de) * | 2001-01-09 | 2003-06-26 | Ribopharma Ag | Verfahren zur Hemmung der Expression eines vorgegebenen Zielgens |
| WO2002081628A2 (en) * | 2001-04-05 | 2002-10-17 | Ribozyme Pharmaceuticals, Incorporated | Modulation of gene expression associated with inflammation proliferation and neurite outgrowth, using nucleic acid based technologies |
| US7097856B2 (en) * | 2000-09-29 | 2006-08-29 | The Regents Of The University Of California | Dendrimeric support or carrier macromolecule |
| US10590418B2 (en) * | 2001-07-23 | 2020-03-17 | The Board Of Trustees Of The Leland Stanford Junior University | Methods and compositions for RNAi mediated inhibition of gene expression in mammals |
| EP1532248B1 (de) * | 2002-07-26 | 2009-04-01 | Novartis Vaccines and Diagnostics, Inc. | Modifizierte kleine irns moleküle und methoden zu deren anwendung |
| ATE513843T1 (de) * | 2002-09-25 | 2011-07-15 | Univ Massachusetts | Abstellen von genen in vivo durch chemischmodifizierte und stabile sirna |
| US20040147027A1 (en) * | 2003-01-28 | 2004-07-29 | Troy Carol M. | Complex for facilitating delivery of dsRNA into a cell and uses thereof |
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2003
- 2003-01-21 DE DE10302421A patent/DE10302421A1/de not_active Withdrawn
-
2004
- 2004-01-21 DE DE602004007620T patent/DE602004007620T2/de not_active Expired - Lifetime
- 2004-01-21 PT PT04704041T patent/PT1587926E/pt unknown
- 2004-01-21 DK DK04704041T patent/DK1587926T3/da active
- 2004-01-21 ES ES04704041T patent/ES2289474T3/es not_active Expired - Lifetime
- 2004-01-21 AT AT04704041T patent/ATE367441T1/de active
- 2004-01-21 EP EP04704041A patent/EP1587926B1/de not_active Expired - Lifetime
- 2004-01-21 WO PCT/US2004/001461 patent/WO2004065601A2/en not_active Ceased
- 2004-01-21 CA CA002513809A patent/CA2513809C/en not_active Expired - Lifetime
- 2004-01-21 AU AU2004206255A patent/AU2004206255B2/en not_active Expired
- 2004-01-21 EP EP07010839A patent/EP1852506A3/de not_active Withdrawn
- 2004-01-21 US US10/543,048 patent/US20060178324A1/en not_active Abandoned
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| Publication number | Publication date |
|---|---|
| AU2004206255A1 (en) | 2004-08-05 |
| EP1852506A3 (de) | 2011-08-31 |
| DK1587926T3 (da) | 2007-11-19 |
| WO2004065601A2 (en) | 2004-08-05 |
| DE602004007620T2 (de) | 2008-04-10 |
| EP1587926B1 (de) | 2007-07-18 |
| AU2008203538B2 (en) | 2012-05-17 |
| US20060178324A1 (en) | 2006-08-10 |
| PT1587926E (pt) | 2007-10-26 |
| AU2008203538A1 (en) | 2008-08-28 |
| CA2513809C (en) | 2009-10-06 |
| DE10302421A1 (de) | 2004-07-29 |
| WO2004065601A3 (en) | 2004-11-18 |
| ES2289474T3 (es) | 2008-02-01 |
| AU2004206255B2 (en) | 2008-05-08 |
| EP1587926A2 (de) | 2005-10-26 |
| CA2513809A1 (en) | 2004-08-05 |
| DE602004007620D1 (de) | 2007-08-30 |
| EP1852506A2 (de) | 2007-11-07 |
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