ES2716818T3 - Molécula de ácido nucleico inductora de interferencias de arn capaz de penetrar en las células y uso de la misma - Google Patents
Molécula de ácido nucleico inductora de interferencias de arn capaz de penetrar en las células y uso de la misma Download PDFInfo
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- ES2716818T3 ES2716818T3 ES13794539T ES13794539T ES2716818T3 ES 2716818 T3 ES2716818 T3 ES 2716818T3 ES 13794539 T ES13794539 T ES 13794539T ES 13794539 T ES13794539 T ES 13794539T ES 2716818 T3 ES2716818 T3 ES 2716818T3
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- rna
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- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- C12N15/09—Recombinant DNA-technology
- C12N15/11—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
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P1/00—Drugs for disorders of the alimentary tract or the digestive system
- A61P1/16—Drugs for disorders of the alimentary tract or the digestive system for liver or gallbladder disorders, e.g. hepatoprotective agents, cholagogues, litholytics
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- A—HUMAN NECESSITIES
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- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P11/00—Drugs for disorders of the respiratory system
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- A61P13/00—Drugs for disorders of the urinary system
- A61P13/12—Drugs for disorders of the urinary system of the kidneys
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- A61P17/00—Drugs for dermatological disorders
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- A61P17/00—Drugs for dermatological disorders
- A61P17/02—Drugs for dermatological disorders for treating wounds, ulcers, burns, scars, keloids, or the like
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- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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- A61P19/00—Drugs for skeletal disorders
- A61P19/02—Drugs for skeletal disorders for joint disorders, e.g. arthritis, arthrosis
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P19/00—Drugs for skeletal disorders
- A61P19/08—Drugs for skeletal disorders for bone diseases, e.g. rachitism, Paget's disease
- A61P19/10—Drugs for skeletal disorders for bone diseases, e.g. rachitism, Paget's disease for osteoporosis
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P25/00—Drugs for disorders of the nervous system
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P3/00—Drugs for disorders of the metabolism
- A61P3/08—Drugs for disorders of the metabolism for glucose homeostasis
- A61P3/10—Drugs for disorders of the metabolism for glucose homeostasis for hyperglycaemia, e.g. antidiabetics
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P35/00—Antineoplastic agents
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- A—HUMAN NECESSITIES
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- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P9/00—Drugs for disorders of the cardiovascular system
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P9/00—Drugs for disorders of the cardiovascular system
- A61P9/10—Drugs 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
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P9/00—Drugs for disorders of the cardiovascular system
- A61P9/12—Antihypertensives
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- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- C12N15/09—Recombinant DNA-technology
- C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
- C12N15/111—General methods applicable to biologically active non-coding nucleic acids
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- C12N15/09—Recombinant DNA-technology
- C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- 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]
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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
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/31—Chemical structure of the backbone
- C12N2310/313—Phosphorodithioates
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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
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/31—Chemical structure of the backbone
- C12N2310/315—Phosphorothioates
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- 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
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- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2320/00—Applications; Uses
- C12N2320/30—Special therapeutic applications
- C12N2320/32—Special delivery means, e.g. tissue-specific
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- Engineering & Computer Science (AREA)
- Life Sciences & Earth Sciences (AREA)
- Genetics & Genomics (AREA)
- Chemical & Material Sciences (AREA)
- Organic Chemistry (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Biomedical Technology (AREA)
- General Health & Medical Sciences (AREA)
- Wood Science & Technology (AREA)
- Biotechnology (AREA)
- General Engineering & Computer Science (AREA)
- Zoology (AREA)
- Pharmacology & Pharmacy (AREA)
- Medicinal Chemistry (AREA)
- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Molecular Biology (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Microbiology (AREA)
- Plant Pathology (AREA)
- Biochemistry (AREA)
- Biophysics (AREA)
- Physics & Mathematics (AREA)
- Physical Education & Sports Medicine (AREA)
- Heart & Thoracic Surgery (AREA)
- Rheumatology (AREA)
- Diabetes (AREA)
- Cardiology (AREA)
- Orthopedic Medicine & Surgery (AREA)
- Dermatology (AREA)
- Urology & Nephrology (AREA)
- Epidemiology (AREA)
- Obesity (AREA)
- Neurology (AREA)
- Neurosurgery (AREA)
- Hematology (AREA)
Applications Claiming Priority (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| KR20120053950 | 2012-05-22 | ||
| PCT/KR2013/004463 WO2013176477A1 (ko) | 2012-05-22 | 2013-05-21 | 세포 내 관통능을 가지는 rna 간섭을 유도하는 핵산 분자 및 그 용도 |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| ES2716818T3 true ES2716818T3 (es) | 2019-06-17 |
Family
ID=49624096
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| ES13794539T Active ES2716818T3 (es) | 2012-05-22 | 2013-05-21 | Molécula de ácido nucleico inductora de interferencias de arn capaz de penetrar en las células y uso de la misma |
Country Status (8)
| Country | Link |
|---|---|
| US (3) | US10125362B2 (enExample) |
| EP (2) | EP3514236A1 (enExample) |
| JP (3) | JP6139671B2 (enExample) |
| KR (2) | KR101567576B1 (enExample) |
| CN (2) | CN108148838A (enExample) |
| DK (1) | DK2853597T3 (enExample) |
| ES (1) | ES2716818T3 (enExample) |
| WO (1) | WO2013176477A1 (enExample) |
Families Citing this family (38)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2012053741A2 (ko) | 2010-10-22 | 2012-04-26 | 성균관대학교산학협력단 | Rna 간섭을 유도하는 핵산 분자 및 그 용도 |
| US10125362B2 (en) | 2012-05-22 | 2018-11-13 | Olix Pharmaceuticals, Inc. | RNA-interference-inducing nucleic acid molecule able to penetrate into cells, and use therefor |
| ES2887366T3 (es) | 2013-09-17 | 2021-12-22 | Bolt Threads Inc | Métodos y composiciones para sintetizar fibras de seda mejoradas |
| KR102279110B1 (ko) * | 2014-04-30 | 2021-07-20 | 올릭스 주식회사 | LasiRNA를 유효성분으로 포함하는 피부 미백용 조성물 |
| WO2016161388A1 (en) | 2015-04-03 | 2016-10-06 | University Of Massachusetts | Fully stabilized asymmetric sirna |
| CN107980062B (zh) | 2015-04-03 | 2022-11-25 | 马萨诸塞大学 | 用于靶向亨廷汀mRNA的寡核苷酸化合物 |
| CN110372230B (zh) | 2015-05-15 | 2020-05-19 | Agc株式会社 | 化学增强玻璃 |
| CN114306627A (zh) * | 2015-08-06 | 2022-04-12 | 希望之城 | 细胞穿透蛋白-抗体缀合物及其使用方法 |
| EP3334499A4 (en) * | 2015-08-14 | 2019-04-17 | University of Massachusetts | BIOACTIVE CONJUGATES FOR THE RELEASE OF OLIGONUCLEOTIDES |
| CN108699556B (zh) * | 2015-11-16 | 2023-02-17 | 奥利克斯医药有限公司 | 使用靶向myd88或tlr3的rna复合物治疗老年性黄斑变性 |
| US10478503B2 (en) | 2016-01-31 | 2019-11-19 | University Of Massachusetts | Branched oligonucleotides |
| CN108779464B (zh) | 2016-02-02 | 2022-05-17 | 奥利克斯医药有限公司 | 使用靶向angpt2和pdgfb的rna复合物治疗血管生成相关性疾病 |
| CA3022874A1 (en) | 2016-02-02 | 2017-08-10 | Olix Pharmaceuticals, Inc. | Treatment of atopic dermatitis and asthma using rna complexes that target il4ra, trpa1, or f2rl1 |
| CN109072238B (zh) * | 2016-04-11 | 2022-05-24 | 奥利克斯医药有限公司 | 使用靶向结缔组织生长因子的rna复合物治疗特发性肺纤维化 |
| KR101916652B1 (ko) * | 2016-06-29 | 2018-11-08 | 올릭스 주식회사 | 작은 간섭 rna의 rna 간섭효과 증진용 화합물 및 이의 용도 |
| WO2018031933A2 (en) | 2016-08-12 | 2018-02-15 | University Of Massachusetts | Conjugated oligonucleotides |
| WO2018146557A2 (en) | 2017-02-10 | 2018-08-16 | Dong Ki Lee | Long double-stranded rna for rna interference |
| KR102321426B1 (ko) * | 2017-02-21 | 2021-11-05 | 올릭스 주식회사 | 남성형 탈모 표적 유전자의 발현을 억제하는 비대칭 siRNA |
| CN110799647A (zh) | 2017-06-23 | 2020-02-14 | 马萨诸塞大学 | 双尾自递送sirna及相关的方法 |
| WO2019066519A1 (ko) * | 2017-09-28 | 2019-04-04 | 올릭스 주식회사 | 결합 조직 성장 인자를 표적으로 하는 rna 복합체를 함유하는 노인성 황반변성의 예방 또는 치료용 약학 조성물 |
| IL274503B2 (en) * | 2017-11-13 | 2024-10-01 | Silence Therapeutics Gmbh | Nucleic acids for inhibiting expression of lpa in a cell |
| CN111527206A (zh) * | 2017-11-13 | 2020-08-11 | 赛伦斯治疗有限责任公司 | 用于抑制靶标基因表达的包含二硫代磷酸酯键的核酸 |
| AU2019316640B2 (en) | 2018-08-10 | 2026-03-12 | University Of Massachusetts | Modified oligonucleotides targeting SNPs |
| EP3840759A4 (en) | 2018-08-23 | 2022-06-01 | University Of Massachusetts | O-METHYL RICH FULLY STABILIZED OLIGONUCLEOTIDES |
| WO2020069055A1 (en) * | 2018-09-28 | 2020-04-02 | Alnylam Pharmaceuticals, Inc. | Transthyretin (ttr) irna compositions and methods of use thereof for treating or preventing ttr-associated ocular diseases |
| CN109402127B (zh) * | 2018-09-29 | 2021-12-10 | 复旦大学附属眼耳鼻喉科医院 | 一组与结缔组织生长因子特异性结合的高亲和力核酸适配体及其应用 |
| WO2020149702A1 (ko) * | 2019-01-18 | 2020-07-23 | 올릭스 주식회사 | Nrl(neural retina leucine zipper)의 발현을 억제하는 비대칭 sirna |
| AU2020207935A1 (en) | 2019-01-18 | 2021-08-26 | University Of Massachusetts | Dynamic pharmacokinetic-modifying anchors |
| US20220380774A1 (en) * | 2019-05-20 | 2022-12-01 | Olix Pharmaceuticals, Inc. | Asymmetric sirna inhibiting expression of pd-1 |
| KR20220047989A (ko) | 2019-08-09 | 2022-04-19 | 유니버시티 오브 매사추세츠 | Snp를 표적화하는 화학적으로 변형된 올리고뉴클레오타이드 |
| US12365894B2 (en) | 2019-09-16 | 2025-07-22 | University Of Massachusetts | Branched lipid conjugates of siRNA for specific tissue delivery |
| KR102757180B1 (ko) * | 2019-11-22 | 2025-01-21 | (주)바이오니아 | Ctgf 유전자 특이적 이중가닥 올리고뉴클레오티드 및 이를 포함하는 섬유증 관련 질환 및 호흡기 관련 질환 예방 및 치료용 조성물 |
| US12534724B2 (en) | 2020-05-26 | 2026-01-27 | University Of Massachusetts | Synthetic oligonucleotides having regions of block and cluster modifications |
| KR102259402B1 (ko) * | 2020-06-04 | 2021-06-01 | 주식회사 아임뉴런바이오사이언스 | 개선된 안정성을 갖는 핵산 분자 및 이의 용도 |
| US20240024492A1 (en) * | 2020-12-07 | 2024-01-25 | Olix Pharmaceuticals, Inc. | Nucleic acid molecule for induction of asymmetric rnai for inhibiting expression of ror-beta |
| CN114807127A (zh) * | 2021-01-19 | 2022-07-29 | 陈璞 | 用于结缔组织生长因子的小干扰rna及其应用 |
| IL309334A (en) | 2021-06-23 | 2024-02-01 | Univ Massachusetts | Anti-FLT1 optimized oligonucleotide compounds for the treatment of preeclampsia and other angiogenic disorders |
| CN119074938B (zh) * | 2024-08-29 | 2025-05-27 | 上海科若纳生命科技有限公司 | 一种用于mRNA递送和表达的核酸组合物及其应用 |
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| BRPI0313202A8 (pt) * | 2002-08-05 | 2016-08-16 | Atugen Ag | Formas adicionais para interferir com as moléculas de rna |
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| WO2006006948A2 (en) * | 2002-11-14 | 2006-01-19 | Dharmacon, Inc. | METHODS AND COMPOSITIONS FOR SELECTING siRNA OF IMPROVED FUNCTIONALITY |
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| AU2003225410A1 (en) * | 2003-03-21 | 2004-10-11 | Academisch Ziekenhuis Leiden | Modulation of exon recognition in pre-mrna by interfering with the secondary rna structure |
| US20040198640A1 (en) * | 2003-04-02 | 2004-10-07 | Dharmacon, Inc. | Stabilized polynucleotides for use in RNA interference |
| US20050136437A1 (en) | 2003-08-25 | 2005-06-23 | Nastech Pharmaceutical Company Inc. | Nanoparticles for delivery of nucleic acids and stable double-stranded RNA |
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| JP2019122379A (ja) | 2019-07-25 |
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