CN110317810A - Tmprss6 irna组合物及其使用方法 - Google Patents
Tmprss6 irna组合物及其使用方法 Download PDFInfo
- Publication number
- CN110317810A CN110317810A CN201910593463.6A CN201910593463A CN110317810A CN 110317810 A CN110317810 A CN 110317810A CN 201910593463 A CN201910593463 A CN 201910593463A CN 110317810 A CN110317810 A CN 110317810A
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- Prior art keywords
- nucleotides
- double
- stranded rnai
- strand
- rnai agent
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- 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
- C12N15/1138—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 receptors or cell surface proteins
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- A61K31/713—Double-stranded nucleic acids or oligonucleotides
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- 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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- 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
- C12N15/1137—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 enzymes
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- C12N9/48—Hydrolases (3) acting on peptide bonds (3.4)
- C12N9/50—Proteinases, e.g. Endopeptidases (3.4.21-3.4.25)
- C12N9/64—Proteinases, e.g. Endopeptidases (3.4.21-3.4.25) derived from animal tissue
- C12N9/6421—Proteinases, e.g. Endopeptidases (3.4.21-3.4.25) derived from animal tissue from mammals
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- C12N2310/34—Spatial arrangement of the modifications
- C12N2310/343—Spatial arrangement of the modifications having patterns, e.g. ==--==--==--
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Applications Claiming Priority (5)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201361826178P | 2013-05-22 | 2013-05-22 | |
| US61/826,178 | 2013-05-22 | ||
| US201361912988P | 2013-12-06 | 2013-12-06 | |
| US61/912,988 | 2013-12-06 | ||
| CN201480040477.0A CN105452463B (zh) | 2013-05-22 | 2014-05-22 | Tmprss6 irna组合物及其使用方法 |
Related Parent Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| CN201480040477.0A Division CN105452463B (zh) | 2013-05-22 | 2014-05-22 | Tmprss6 irna组合物及其使用方法 |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| CN110317810A true CN110317810A (zh) | 2019-10-11 |
Family
ID=50943629
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| CN201910593463.6A Pending CN110317810A (zh) | 2013-05-22 | 2014-05-22 | Tmprss6 irna组合物及其使用方法 |
| CN201480040477.0A Active CN105452463B (zh) | 2013-05-22 | 2014-05-22 | Tmprss6 irna组合物及其使用方法 |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| CN201480040477.0A Active CN105452463B (zh) | 2013-05-22 | 2014-05-22 | Tmprss6 irna组合物及其使用方法 |
Country Status (20)
| Country | Link |
|---|---|
| US (6) | US9783806B2 (2) |
| EP (3) | EP2999786A1 (2) |
| JP (5) | JP2016520593A (2) |
| KR (3) | KR102234623B1 (2) |
| CN (2) | CN110317810A (2) |
| AP (1) | AP2015008862A0 (2) |
| AU (4) | AU2014268529C1 (2) |
| BR (1) | BR112015029276B1 (2) |
| CA (3) | CA2912834C (2) |
| CL (2) | CL2015003440A1 (2) |
| EA (1) | EA201592225A1 (2) |
| HK (1) | HK1221978A1 (2) |
| IL (6) | IL324706A (2) |
| MY (1) | MY181888A (2) |
| RU (1) | RU2015154738A (2) |
| SG (2) | SG10202103166XA (2) |
| TW (3) | TW202342750A (2) |
| UY (1) | UY35583A (2) |
| WO (1) | WO2014190157A1 (2) |
| ZA (1) | ZA202110854B (2) |
Cited By (1)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CN117136236A (zh) * | 2022-09-29 | 2023-11-28 | 广州必贝特医药股份有限公司 | 用于抑制TMPRSS6基因表达的siRNA或其盐、药物及其应用 |
Families Citing this family (38)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2011149962A1 (en) | 2010-05-24 | 2011-12-01 | The Trustees Of Columbia University In The City Of New York | Mutant ngal proteins and uses thereof |
| PH12013501969B1 (en) | 2011-03-29 | 2018-08-31 | Alnylam Pharmaceuticals Inc | Compositions and methods for inhibiting expression of tmprss6 gene |
| SI3366775T2 (sl) | 2011-11-18 | 2025-12-31 | Alnylam Pharmaceuticals, Inc. | Modificirana sredstva RNAI |
| EP2925337B1 (en) * | 2012-11-21 | 2019-07-03 | The Trustees of Columbia University in the City of New York | Mutant ngal proteins and uses thereof |
| KR102234623B1 (ko) | 2013-05-22 | 2021-04-02 | 알닐람 파마슈티칼스 인코포레이티드 | Tmprss6 조성물 및 이의 사용 방법 |
| WO2015099122A1 (ja) | 2013-12-26 | 2015-07-02 | 学校法人東京医科大学 | 遺伝子発現制御のための人工ミミックmiRNAおよびその用途 |
| CN106068324B (zh) | 2013-12-27 | 2020-12-29 | 株式会社博纳克 | 控制基因表达的人工匹配型miRNA及其用途 |
| IL316808A (en) | 2014-08-20 | 2025-01-01 | Alnylam Pharmaceuticals Inc | Modified double-stranded RNA materials and their uses |
| WO2016085852A1 (en) | 2014-11-24 | 2016-06-02 | Alnylam Pharmaceuticals, Inc. | Tmprss6 irna compositions and methods of use thereof |
| KR20170098914A (ko) | 2014-12-27 | 2017-08-30 | 가부시키가이샤 보낙 | 유전자 발현 제어를 위한 천연형 miRNA 및 그의 용도 |
| WO2016158809A1 (ja) * | 2015-03-27 | 2016-10-06 | 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 |
| US20160319278A1 (en) | 2015-04-03 | 2016-11-03 | University Of Massachusetts | Fully stabilized asymmetric sirna |
| MX2017012426A (es) * | 2015-04-03 | 2018-01-26 | Ionis Pharmaceuticals Inc | Composiciones y metodos para modular la expresion de tmprss6. |
| JP6695902B2 (ja) * | 2015-05-06 | 2020-05-20 | アルナイラム ファーマシューティカルズ, インコーポレイテッドAlnylam Pharmaceuticals, Inc. | 第XII因子(ハーゲマン因子)(F12)、カリクレインB、血漿(フレッチャー因子)1(KLKB1)、及びキニノーゲン1(KNG1)iRNA組成物及びその使用方法 |
| WO2017030973A1 (en) | 2015-08-14 | 2017-02-23 | University Of Massachusetts | Bioactive conjugates for oligonucleotide delivery |
| US10478503B2 (en) | 2016-01-31 | 2019-11-19 | University Of Massachusetts | Branched oligonucleotides |
| CN108342386B (zh) * | 2017-01-22 | 2022-04-15 | 广州市锐博生物科技有限公司 | 一种多聚寡核酸分子及其在多靶标干扰中的应用 |
| EP3385380A1 (en) * | 2017-04-05 | 2018-10-10 | Silence Therapeutics GmbH | Products and compositions |
| DK3607069T3 (da) * | 2017-04-05 | 2022-11-21 | Silence Therapeutics Gmbh | Produkter og sammensætninger |
| SI3607068T1 (sl) * | 2017-04-05 | 2023-02-28 | Silence Therapeutics Gmbh | Z RNA interferenco posredovano inhibiranje TMPRSS6 |
| JP7406793B2 (ja) | 2017-06-23 | 2023-12-28 | ユニバーシティー オブ マサチューセッツ | 2テイル自己デリバリー型siRNAおよび関連方法 |
| AU2019239971B2 (en) | 2018-03-21 | 2025-09-11 | Regeneron Pharmaceuticals, Inc. | 17beta-hydroxysteroid dehydrogenase type 13 (HSD17b13) iRNA compositions and methods of use thereof |
| KR102857148B1 (ko) * | 2018-04-05 | 2025-09-11 | 사일런스 테라퓨틱스 게엠베하 | 안티센스 가닥의 5' 단부에 비닐포스포네이트를 갖는 siRNA |
| TWI851574B (zh) * | 2018-05-14 | 2024-08-11 | 美商阿尼拉製藥公司 | 血管收縮素原(AGT)iRNA組成物及其使用方法 |
| EP3598995A1 (en) * | 2018-07-26 | 2020-01-29 | Silence Therapeutics GmbH | Products and compositions |
| WO2020061295A1 (en) | 2018-09-19 | 2020-03-26 | Modernatx, Inc. | High-purity peg lipids and uses thereof |
| WO2020097044A1 (en) | 2018-11-09 | 2020-05-14 | Alnylam Pharmaceuticals, Inc. | Modified double stranded oligonucleotides |
| CN111973619B (zh) * | 2019-05-23 | 2024-01-30 | 苏州瑞博生物技术股份有限公司 | 核酸、含有该核酸的药物组合物与siRNA缀合物及制备方法和用途 |
| KR20220047989A (ko) | 2019-08-09 | 2022-04-19 | 유니버시티 오브 매사추세츠 | Snp를 표적화하는 화학적으로 변형된 올리고뉴클레오타이드 |
| WO2021242883A1 (en) | 2020-05-26 | 2021-12-02 | University Of Massachusetts | Synthetic oligonucleotides having regions of block and cluster modifications |
| US20220228141A1 (en) * | 2020-11-23 | 2022-07-21 | University Of Massachusetts | Oligonucleotides for dgat2 modulation |
| EP4330392A1 (en) * | 2021-04-26 | 2024-03-06 | Alnylam Pharmaceuticals, Inc. | Transmembrane protease, serine 6 (tmprss6) irna compositions and methods of use thereof |
| AU2022265493A1 (en) | 2021-04-27 | 2023-09-21 | Silence Therapeutics Gmbh | Sirna targeting tmprss6 for the treatment of myeloproliferative disorders |
| TWI868755B (zh) * | 2022-06-24 | 2025-01-01 | 丹麥商諾佛 儂迪克股份有限公司 | 抑制跨膜絲胺酸蛋白酶6(tmprss6)表現的組成物及方法 |
| WO2024060649A1 (zh) * | 2022-09-29 | 2024-03-28 | 广州必贝特医药股份有限公司 | 用于抑制TMPRSS6基因表达的siRNA或其盐、药物及其应用 |
| CN120981247A (zh) | 2023-03-27 | 2025-11-18 | 赛伦斯治疗有限责任公司 | 用于干细胞移植的化合物和组合物 |
| WO2024254589A2 (en) * | 2023-06-09 | 2024-12-12 | Sirnaomics, Inc. | Products and compositions |
| WO2025148896A1 (en) * | 2024-01-08 | 2025-07-17 | Shanghai Argo Biopharmaceutical Co., Ltd. | Compositions and methods for inhibiting expression of transmembrane serine protease 6 (tmprss6) |
Citations (5)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO1998013526A1 (en) * | 1996-09-26 | 1998-04-02 | Oligos Etc. Inc. | Three component chimeric antisense oligonucleotides |
| WO2009073809A2 (en) * | 2007-12-04 | 2009-06-11 | Alnylam Pharmaceuticals, Inc. | Carbohydrate conjugates as delivery agents for oligonucleotides |
| WO2010033246A1 (en) * | 2008-09-22 | 2010-03-25 | Rxi Pharmaceuticals Corporation | Rna interference in skin indications |
| WO2010148013A2 (en) * | 2009-06-15 | 2010-12-23 | Alnylam Pharmaceuticals, Inc. | Lipid formulated dsrna targeting the pcsk9 gene |
| WO2012135246A2 (en) * | 2011-03-29 | 2012-10-04 | Alnylam Pharmaceuticals, Inc. | Compositions and methods for inhibiting expression of tmprss6 gene |
Family Cites Families (202)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US564562A (en) | 1896-07-21 | Joseph p | ||
| US513030A (en) | 1894-01-16 | Machine for waxing or coating paper | ||
| US1861608A (en) | 1929-12-21 | 1932-06-07 | Emerson Electric Mfg Co | Fan and means for directing the air current therethrough |
| US1861108A (en) | 1930-01-24 | 1932-05-31 | Eugene O Brace | Integral clutch and transmission control |
| FR874253A (fr) | 1941-03-25 | 1942-08-03 | Nouvel emboîtage d'horlogerie | |
| US3687808A (en) | 1969-08-14 | 1972-08-29 | Univ Leland Stanford Junior | Synthetic polynucleotides |
| US3974808A (en) | 1975-07-02 | 1976-08-17 | Ford Motor Company | Air intake duct assembly |
| US4469863A (en) | 1980-11-12 | 1984-09-04 | Ts O Paul O P | Nonionic nucleic acid alkyl and aryl phosphonates and processes for manufacture and use thereof |
| US5023243A (en) | 1981-10-23 | 1991-06-11 | Molecular Biosystems, Inc. | Oligonucleotide therapeutic agent and method of making same |
| US4476301A (en) | 1982-04-29 | 1984-10-09 | Centre National De La Recherche Scientifique | Oligonucleotides, a process for preparing the same and their application as mediators of the action of interferon |
| US4708708A (en) | 1982-12-06 | 1987-11-24 | International Paper Company | Method and apparatus for skiving and hemming |
| US5118800A (en) | 1983-12-20 | 1992-06-02 | California Institute Of Technology | Oligonucleotides possessing a primary amino group in the terminal nucleotide |
| US5550111A (en) | 1984-07-11 | 1996-08-27 | Temple University-Of The Commonwealth System Of Higher Education | Dual action 2',5'-oligoadenylate antiviral derivatives and uses thereof |
| FR2567892B1 (fr) | 1984-07-19 | 1989-02-17 | Centre Nat Rech Scient | Nouveaux oligonucleotides, leur procede de preparation et leurs applications comme mediateurs dans le developpement des effets des interferons |
| US5367066A (en) | 1984-10-16 | 1994-11-22 | Chiron Corporation | Oligonucleotides with selectably cleavable and/or abasic sites |
| US4897355A (en) | 1985-01-07 | 1990-01-30 | Syntex (U.S.A.) Inc. | N[ω,(ω-1)-dialkyloxy]- and N-[ω,(ω-1)-dialkenyloxy]-alk-1-yl-N,N,N-tetrasubstituted ammonium lipids and uses therefor |
| FR2575751B1 (fr) | 1985-01-08 | 1987-04-03 | Pasteur Institut | Nouveaux nucleosides de derives de l'adenosine, leur preparation et leurs applications biologiques |
| US5405938A (en) | 1989-12-20 | 1995-04-11 | Anti-Gene Development Group | Sequence-specific binding polymers for duplex nucleic acids |
| US5166315A (en) | 1989-12-20 | 1992-11-24 | Anti-Gene Development Group | Sequence-specific binding polymers for duplex nucleic acids |
| US5034506A (en) | 1985-03-15 | 1991-07-23 | Anti-Gene Development Group | Uncharged morpholino-based polymers having achiral intersubunit linkages |
| US5235033A (en) | 1985-03-15 | 1993-08-10 | Anti-Gene Development Group | Alpha-morpholino ribonucleoside derivatives and polymers thereof |
| US5185444A (en) | 1985-03-15 | 1993-02-09 | Anti-Gene Deveopment Group | Uncharged morpolino-based polymers having phosphorous containing chiral intersubunit linkages |
| US4683202A (en) | 1985-03-28 | 1987-07-28 | Cetus Corporation | Process for amplifying nucleic acid sequences |
| US5139941A (en) | 1985-10-31 | 1992-08-18 | University Of Florida Research Foundation, Inc. | AAV transduction vectors |
| US4837028A (en) | 1986-12-24 | 1989-06-06 | Liposome Technology, Inc. | Liposomes with enhanced circulation time |
| US4920016A (en) | 1986-12-24 | 1990-04-24 | Linear Technology, Inc. | Liposomes with enhanced circulation time |
| US5264423A (en) | 1987-03-25 | 1993-11-23 | The United States Of America As Represented By The Department Of Health And Human Services | Inhibitors for replication of retroviruses and for the expression of oncogene products |
| US5276019A (en) | 1987-03-25 | 1994-01-04 | The United States Of America As Represented By The Department Of Health And Human Services | Inhibitors for replication of retroviruses and for the expression of oncogene products |
| EP0366685B1 (en) | 1987-06-24 | 1994-10-19 | Howard Florey Institute Of Experimental Physiology And Medicine | Nucleoside derivatives |
| US4924624A (en) | 1987-10-22 | 1990-05-15 | Temple University-Of The Commonwealth System Of Higher Education | 2,',5'-phosphorothioate oligoadenylates and plant antiviral uses thereof |
| US5188897A (en) | 1987-10-22 | 1993-02-23 | Temple University Of The Commonwealth System Of Higher Education | Encapsulated 2',5'-phosphorothioate oligoadenylates |
| JPH03503894A (ja) | 1988-03-25 | 1991-08-29 | ユニバーシィティ オブ バージニア アランミ パテンツ ファウンデイション | オリゴヌクレオチド n‐アルキルホスホラミデート |
| US5278302A (en) | 1988-05-26 | 1994-01-11 | University Patents, Inc. | Polynucleotide phosphorodithioates |
| US5216141A (en) | 1988-06-06 | 1993-06-01 | Benner Steven A | Oligonucleotide analogs containing sulfur linkages |
| US5175273A (en) | 1988-07-01 | 1992-12-29 | Genentech, Inc. | Nucleic acid intercalating agents |
| FR2645866B1 (fr) | 1989-04-17 | 1991-07-05 | Centre Nat Rech Scient | Nouvelles lipopolyamines, leur preparation et leur emploi |
| US5143854A (en) | 1989-06-07 | 1992-09-01 | Affymax Technologies N.V. | Large scale photolithographic solid phase synthesis of polypeptides and receptor binding screening thereof |
| US5744101A (en) | 1989-06-07 | 1998-04-28 | Affymax Technologies N.V. | Photolabile nucleoside protecting groups |
| NL8901881A (nl) | 1989-07-20 | 1991-02-18 | Rockwool Grodan Bv | Drainagekoppelelement. |
| US5134066A (en) | 1989-08-29 | 1992-07-28 | Monsanto Company | Improved probes using nucleosides containing 3-dezauracil analogs |
| US5436146A (en) | 1989-09-07 | 1995-07-25 | The Trustees Of Princeton University | Helper-free stocks of recombinant adeno-associated virus vectors |
| US5591722A (en) | 1989-09-15 | 1997-01-07 | Southern Research Institute | 2'-deoxy-4'-thioribonucleosides and their antiviral activity |
| US5399676A (en) | 1989-10-23 | 1995-03-21 | Gilead Sciences | Oligonucleotides with inverted polarity |
| DE69034150T2 (de) | 1989-10-24 | 2005-08-25 | Isis Pharmaceuticals, Inc., Carlsbad | 2'-Modifizierte Oligonukleotide |
| US5264564A (en) | 1989-10-24 | 1993-11-23 | Gilead Sciences | Oligonucleotide analogs with novel linkages |
| US5177198A (en) | 1989-11-30 | 1993-01-05 | University Of N.C. At Chapel Hill | Process for preparing oligoribonucleoside and oligodeoxyribonucleoside boranophosphates |
| CA2029273A1 (en) | 1989-12-04 | 1991-06-05 | Christine L. Brakel | Modified nucleotide compounds |
| US5681941A (en) | 1990-01-11 | 1997-10-28 | Isis Pharmaceuticals, Inc. | Substituted purines and oligonucleotide cross-linking |
| US5852188A (en) | 1990-01-11 | 1998-12-22 | Isis Pharmaceuticals, Inc. | Oligonucleotides having chiral phosphorus linkages |
| US5646265A (en) | 1990-01-11 | 1997-07-08 | Isis Pharmceuticals, Inc. | Process for the preparation of 2'-O-alkyl purine phosphoramidites |
| US5670633A (en) | 1990-01-11 | 1997-09-23 | Isis Pharmaceuticals, Inc. | Sugar modified oligonucleotides that detect and modulate gene expression |
| US5459255A (en) | 1990-01-11 | 1995-10-17 | Isis Pharmaceuticals, Inc. | N-2 substituted purines |
| US5587470A (en) | 1990-01-11 | 1996-12-24 | Isis Pharmaceuticals, Inc. | 3-deazapurines |
| US5587361A (en) | 1991-10-15 | 1996-12-24 | Isis Pharmaceuticals, Inc. | Oligonucleotides having phosphorothioate linkages of high chiral purity |
| US5321131A (en) | 1990-03-08 | 1994-06-14 | Hybridon, Inc. | Site-specific functionalization of oligodeoxynucleotides for non-radioactive labelling |
| US5470967A (en) | 1990-04-10 | 1995-11-28 | The Dupont Merck Pharmaceutical Company | Oligonucleotide analogs with sulfamate linkages |
| US5264618A (en) | 1990-04-19 | 1993-11-23 | Vical, Inc. | Cationic lipids for intracellular delivery of biologically active molecules |
| GB9009980D0 (en) | 1990-05-03 | 1990-06-27 | Amersham Int Plc | Phosphoramidite derivatives,their preparation and the use thereof in the incorporation of reporter groups on synthetic oligonucleotides |
| ES2116977T3 (es) | 1990-05-11 | 1998-08-01 | Microprobe Corp | Soportes solidos para ensayos de hibridacion de acidos nucleicos y metodos para inmovilizar oligonucleotidos de modo covalente. |
| US5981276A (en) | 1990-06-20 | 1999-11-09 | Dana-Farber Cancer Institute | Vectors containing HIV packaging sequences, packaging defective HIV vectors, and uses thereof |
| US5541307A (en) | 1990-07-27 | 1996-07-30 | Isis Pharmaceuticals, Inc. | Backbone modified oligonucleotide analogs and solid phase synthesis thereof |
| US5602240A (en) | 1990-07-27 | 1997-02-11 | Ciba Geigy Ag. | Backbone modified oligonucleotide analogs |
| US5608046A (en) | 1990-07-27 | 1997-03-04 | Isis Pharmaceuticals, Inc. | Conjugated 4'-desmethyl nucleoside analog compounds |
| US5489677A (en) | 1990-07-27 | 1996-02-06 | Isis Pharmaceuticals, Inc. | Oligonucleoside linkages containing adjacent oxygen and nitrogen atoms |
| US5677437A (en) | 1990-07-27 | 1997-10-14 | Isis Pharmaceuticals, Inc. | Heteroatomic oligonucleoside linkages |
| US5610289A (en) | 1990-07-27 | 1997-03-11 | Isis Pharmaceuticals, Inc. | Backbone modified oligonucleotide analogues |
| US5618704A (en) | 1990-07-27 | 1997-04-08 | Isis Pharmacueticals, Inc. | Backbone-modified oligonucleotide analogs and preparation thereof through radical coupling |
| JPH0874B2 (ja) | 1990-07-27 | 1996-01-10 | アイシス・ファーマシューティカルス・インコーポレーテッド | 遺伝子発現を検出および変調するヌクレアーゼ耐性、ピリミジン修飾オリゴヌクレオチド |
| US5623070A (en) | 1990-07-27 | 1997-04-22 | Isis Pharmaceuticals, Inc. | Heteroatomic oligonucleoside linkages |
| MY107332A (en) | 1990-08-03 | 1995-11-30 | Sterling Drug Inc | Compounds and methods for inhibiting gene expression. |
| US5214134A (en) | 1990-09-12 | 1993-05-25 | Sterling Winthrop Inc. | Process of linking nucleosides with a siloxane bridge |
| US5561225A (en) | 1990-09-19 | 1996-10-01 | Southern Research Institute | Polynucleotide analogs containing sulfonate and sulfonamide internucleoside linkages |
| JPH06505704A (ja) | 1990-09-20 | 1994-06-30 | ギリアド サイエンシズ,インコーポレイテッド | 改変ヌクレオシド間結合 |
| US5432272A (en) | 1990-10-09 | 1995-07-11 | Benner; Steven A. | Method for incorporating into a DNA or RNA oligonucleotide using nucleotides bearing heterocyclic bases |
| GB9100304D0 (en) | 1991-01-08 | 1991-02-20 | Ici Plc | Compound |
| US7015315B1 (en) | 1991-12-24 | 2006-03-21 | Isis Pharmaceuticals, Inc. | Gapped oligonucleotides |
| US5714331A (en) | 1991-05-24 | 1998-02-03 | Buchardt, Deceased; Ole | Peptide nucleic acids having enhanced binding affinity, sequence specificity and solubility |
| US5539082A (en) | 1993-04-26 | 1996-07-23 | Nielsen; Peter E. | Peptide nucleic acids |
| US5719262A (en) | 1993-11-22 | 1998-02-17 | Buchardt, Deceased; Ole | Peptide nucleic acids having amino acid side chains |
| US5571799A (en) | 1991-08-12 | 1996-11-05 | Basco, Ltd. | (2'-5') oligoadenylate analogues useful as inhibitors of host-v5.-graft response |
| US5283185A (en) | 1991-08-28 | 1994-02-01 | University Of Tennessee Research Corporation | Method for delivering nucleic acids into cells |
| EP0538194B1 (de) | 1991-10-17 | 1997-06-04 | Novartis AG | Bicyclische Nukleoside, Oligonukleotide, Verfahren zu deren Herstellung und Zwischenprodukte |
| US5594121A (en) | 1991-11-07 | 1997-01-14 | Gilead Sciences, Inc. | Enhanced triple-helix and double-helix formation with oligomers containing modified purines |
| US5252479A (en) | 1991-11-08 | 1993-10-12 | Research Corporation Technologies, Inc. | Safe vector for gene therapy |
| CA2124087C (en) | 1991-11-22 | 2002-10-01 | James L. Winkler | Combinatorial strategies for polymer synthesis |
| US5484908A (en) | 1991-11-26 | 1996-01-16 | Gilead Sciences, Inc. | Oligonucleotides containing 5-propynyl pyrimidines |
| US6235887B1 (en) | 1991-11-26 | 2001-05-22 | Isis Pharmaceuticals, Inc. | Enhanced triple-helix and double-helix formation directed by oligonucleotides containing modified pyrimidines |
| US5359044A (en) | 1991-12-13 | 1994-10-25 | Isis Pharmaceuticals | Cyclobutyl oligonucleotide surrogates |
| US6277603B1 (en) | 1991-12-24 | 2001-08-21 | Isis Pharmaceuticals, Inc. | PNA-DNA-PNA chimeric macromolecules |
| JP3131222B2 (ja) | 1991-12-24 | 2001-01-31 | アイシス・ファーマシューティカルス・インコーポレーテッド | ギャップを有する2′修飾オリゴヌクレオチド |
| FR2687679B1 (fr) | 1992-02-05 | 1994-10-28 | Centre Nat Rech Scient | Oligothionucleotides. |
| DE4203923A1 (de) | 1992-02-11 | 1993-08-12 | Henkel Kgaa | Verfahren zur herstellung von polycarboxylaten auf polysaccharid-basis |
| US5633360A (en) | 1992-04-14 | 1997-05-27 | Gilead Sciences, Inc. | Oligonucleotide analogs capable of passive cell membrane permeation |
| US5434257A (en) | 1992-06-01 | 1995-07-18 | Gilead Sciences, Inc. | Binding compentent oligomers containing unsaturated 3',5' and 2',5' linkages |
| US5587308A (en) | 1992-06-02 | 1996-12-24 | The United States Of America As Represented By The Department Of Health & Human Services | Modified adeno-associated virus vector capable of expression from a novel promoter |
| EP0646178A1 (en) | 1992-06-04 | 1995-04-05 | The Regents Of The University Of California | expression cassette with regularoty regions functional in the mammmlian host |
| AU4541093A (en) | 1992-06-18 | 1994-01-24 | Genpharm International, Inc. | Methods for producing transgenic non-human animals harboring a yeast artificial chromosome |
| EP0577558A2 (de) | 1992-07-01 | 1994-01-05 | Ciba-Geigy Ag | Carbocyclische Nukleoside mit bicyclischen Ringen, Oligonukleotide daraus, Verfahren zu deren Herstellung, deren Verwendung und Zwischenproduckte |
| EP0786522A2 (en) | 1992-07-17 | 1997-07-30 | Ribozyme Pharmaceuticals, Inc. | Enzymatic RNA molecules for treatment of stenotic conditions |
| US6346614B1 (en) | 1992-07-23 | 2002-02-12 | Hybridon, Inc. | Hybrid oligonucleotide phosphorothioates |
| US5783701A (en) | 1992-09-08 | 1998-07-21 | Vertex Pharmaceuticals, Incorporated | Sulfonamide inhibitors of aspartyl protease |
| EP1024198A3 (en) | 1992-12-03 | 2002-05-29 | Genzyme Corporation | Pseudo-adenoviral vectors for the gene therapy of haemophiliae |
| US5478745A (en) | 1992-12-04 | 1995-12-26 | University Of Pittsburgh | Recombinant viral vector system |
| US5476925A (en) | 1993-02-01 | 1995-12-19 | Northwestern University | Oligodeoxyribonucleotides including 3'-aminonucleoside-phosphoramidate linkages and terminal 3'-amino groups |
| KR100283601B1 (ko) | 1993-02-19 | 2001-03-02 | 아만 히데아키 | 글리세롤 유도체, 디바이스 및 의약 조성물 |
| GB9304618D0 (en) | 1993-03-06 | 1993-04-21 | Ciba Geigy Ag | Chemical compounds |
| CA2159631A1 (en) | 1993-03-30 | 1994-10-13 | Sanofi | Acyclic nucleoside analogs and oligonucleotide sequences containing them |
| HU9501974D0 (en) | 1993-03-31 | 1995-09-28 | Sterling Winthrop Inc | Oligonucleotides with amide linkages replacing phosphodiester linkages |
| DE4311944A1 (de) | 1993-04-10 | 1994-10-13 | Degussa | Umhüllte Natriumpercarbonatpartikel, Verfahren zu deren Herstellung und sie enthaltende Wasch-, Reinigungs- und Bleichmittelzusammensetzungen |
| US6191105B1 (en) | 1993-05-10 | 2001-02-20 | Protein Delivery, Inc. | Hydrophilic and lipophilic balanced microemulsion formulations of free-form and/or conjugation-stabilized therapeutic agents such as insulin |
| US5955591A (en) | 1993-05-12 | 1999-09-21 | Imbach; Jean-Louis | Phosphotriester oligonucleotides, amidites and method of preparation |
| US6015886A (en) | 1993-05-24 | 2000-01-18 | Chemgenes Corporation | Oligonucleotide phosphate esters |
| US5502177A (en) | 1993-09-17 | 1996-03-26 | Gilead Sciences, Inc. | Pyrimidine derivatives for labeled binding partners |
| CA2176256A1 (en) | 1993-11-16 | 1995-05-26 | Lyle John Arnold, Jr. | Synthetic oligomers having chirally pure phosphonate internucleosidyl linkages mixed with non-phosphonate internucleosidyl linkages |
| US5457187A (en) | 1993-12-08 | 1995-10-10 | Board Of Regents University Of Nebraska | Oligonucleotides containing 5-fluorouracil |
| US5446137B1 (en) | 1993-12-09 | 1998-10-06 | Behringwerke Ag | Oligonucleotides containing 4'-substituted nucleotides |
| US5519134A (en) | 1994-01-11 | 1996-05-21 | Isis Pharmaceuticals, Inc. | Pyrrolidine-containing monomers and oligomers |
| US5599922A (en) | 1994-03-18 | 1997-02-04 | Lynx Therapeutics, Inc. | Oligonucleotide N3'-P5' phosphoramidates: hybridization and nuclease resistance properties |
| US5596091A (en) | 1994-03-18 | 1997-01-21 | The Regents Of The University Of California | Antisense oligonucleotides comprising 5-aminoalkyl pyrimidine nucleotides |
| US5627053A (en) | 1994-03-29 | 1997-05-06 | Ribozyme Pharmaceuticals, Inc. | 2'deoxy-2'-alkylnucleotide containing nucleic acid |
| US5625050A (en) | 1994-03-31 | 1997-04-29 | Amgen Inc. | Modified oligonucleotides and intermediates useful in nucleic acid therapeutics |
| US6054299A (en) | 1994-04-29 | 2000-04-25 | Conrad; Charles A. | Stem-loop cloning vector and method |
| US5525711A (en) | 1994-05-18 | 1996-06-11 | The United States Of America As Represented By The Secretary Of The Department Of Health And Human Services | Pteridine nucleotide analogs as fluorescent DNA probes |
| US5543152A (en) | 1994-06-20 | 1996-08-06 | Inex Pharmaceuticals Corporation | Sphingosomes for enhanced drug delivery |
| US5597909A (en) | 1994-08-25 | 1997-01-28 | Chiron Corporation | Polynucleotide reagents containing modified deoxyribose moieties, and associated methods of synthesis and use |
| US5556752A (en) | 1994-10-24 | 1996-09-17 | Affymetrix, Inc. | Surface-bound, unimolecular, double-stranded DNA |
| US6608035B1 (en) | 1994-10-25 | 2003-08-19 | Hybridon, Inc. | Method of down-regulating gene expression |
| US5665557A (en) | 1994-11-14 | 1997-09-09 | Systemix, Inc. | Method of purifying a population of cells enriched for hematopoietic stem cells populations of cells obtained thereby and methods of use thereof |
| JP3269301B2 (ja) | 1994-12-28 | 2002-03-25 | 豊田合成株式会社 | ガラスラン用ゴム配合物 |
| US6166197A (en) | 1995-03-06 | 2000-12-26 | Isis Pharmaceuticals, Inc. | Oligomeric compounds having pyrimidine nucleotide (S) with 2'and 5 substitutions |
| WO1996027606A1 (en) | 1995-03-06 | 1996-09-12 | Isis Pharmaceuticals, Inc. | Improved process for the synthesis of 2'-o-substituted pyrimidines and oligomeric compounds therefrom |
| CA2220950A1 (en) | 1995-05-26 | 1996-11-28 | Somatix Therapy Corporation | Delivery vehicles comprising stable lipid/nucleic acid complexes |
| US5981501A (en) | 1995-06-07 | 1999-11-09 | Inex Pharmaceuticals Corp. | Methods for encapsulating plasmids in lipid bilayers |
| US5545531A (en) | 1995-06-07 | 1996-08-13 | Affymax Technologies N.V. | Methods for making a device for concurrently processing multiple biological chip assays |
| US5976567A (en) | 1995-06-07 | 1999-11-02 | Inex Pharmaceuticals Corp. | Lipid-nucleic acid particles prepared via a hydrophobic lipid-nucleic acid complex intermediate and use for gene transfer |
| US7422902B1 (en) | 1995-06-07 | 2008-09-09 | The University Of British Columbia | Lipid-nucleic acid particles prepared via a hydrophobic lipid-nucleic acid complex intermediate and use for gene transfer |
| US5858397A (en) | 1995-10-11 | 1999-01-12 | University Of British Columbia | Liposomal formulations of mitoxantrone |
| CA2234931C (en) | 1995-10-16 | 2010-01-19 | Dana-Farber Cancer Institute | Novel expression vectors and methods of use |
| US6160109A (en) | 1995-10-20 | 2000-12-12 | Isis Pharmaceuticals, Inc. | Preparation of phosphorothioate and boranophosphate oligomers |
| US5854033A (en) | 1995-11-21 | 1998-12-29 | Yale University | Rolling circle replication reporter systems |
| US5858401A (en) | 1996-01-22 | 1999-01-12 | Sidmak Laboratories, Inc. | Pharmaceutical composition for cyclosporines |
| US5994316A (en) | 1996-02-21 | 1999-11-30 | The Immune Response Corporation | Method of preparing polynucleotide-carrier complexes for delivery to cells |
| US6444423B1 (en) | 1996-06-07 | 2002-09-03 | Molecular Dynamics, Inc. | Nucleosides comprising polydentate ligands |
| US6639062B2 (en) | 1997-02-14 | 2003-10-28 | Isis Pharmaceuticals, Inc. | Aminooxy-modified nucleosidic compounds and oligomeric compounds prepared therefrom |
| US6172209B1 (en) | 1997-02-14 | 2001-01-09 | Isis Pharmaceuticals Inc. | Aminooxy-modified oligonucleotides and methods for making same |
| US6034135A (en) | 1997-03-06 | 2000-03-07 | Promega Biosciences, Inc. | Dimeric cationic lipids |
| JP3756313B2 (ja) | 1997-03-07 | 2006-03-15 | 武 今西 | 新規ビシクロヌクレオシド及びオリゴヌクレオチド類縁体 |
| CA2294988C (en) | 1997-07-01 | 2015-11-24 | Isis Pharmaceuticals Inc. | Compositions and methods for the delivery of oligonucleotides via the alimentary canal |
| US6794499B2 (en) | 1997-09-12 | 2004-09-21 | Exiqon A/S | Oligonucleotide analogues |
| US6528640B1 (en) | 1997-11-05 | 2003-03-04 | Ribozyme Pharmaceuticals, Incorporated | Synthetic ribonucleic acids with RNAse activity |
| US6617438B1 (en) | 1997-11-05 | 2003-09-09 | Sirna Therapeutics, Inc. | Oligoribonucleotides with enzymatic activity |
| US6436989B1 (en) | 1997-12-24 | 2002-08-20 | Vertex Pharmaceuticals, Incorporated | Prodrugs of aspartyl protease inhibitors |
| EA200000702A1 (ru) | 1997-12-24 | 2000-12-25 | Вертекс Фармасьютикалз Инкорпорейтед | Пролекарства ингибиторов аспартилпротеаз |
| US7273933B1 (en) | 1998-02-26 | 2007-09-25 | Isis Pharmaceuticals, Inc. | Methods for synthesis of oligonucleotides |
| US7045610B2 (en) | 1998-04-03 | 2006-05-16 | Epoch Biosciences, Inc. | Modified oligonucleotides for mismatch discrimination |
| US6531590B1 (en) | 1998-04-24 | 2003-03-11 | Isis Pharmaceuticals, Inc. | Processes for the synthesis of oligonucleotide compounds |
| US6867294B1 (en) | 1998-07-14 | 2005-03-15 | Isis Pharmaceuticals, Inc. | Gapped oligomers having site specific chiral phosphorothioate internucleoside linkages |
| AU747597B2 (en) | 1998-07-20 | 2002-05-16 | Inex Pharmaceuticals Corporation | Liposomal encapsulated nucleic acid-complexes |
| JP2002527061A (ja) | 1998-10-09 | 2002-08-27 | インジーン・インコーポレイテッド | ssDNAの酵素的合成 |
| MXPA01003643A (es) | 1998-10-09 | 2003-07-21 | Ingene Inc | Produccion de adnss in vivo. |
| US6465628B1 (en) | 1999-02-04 | 2002-10-15 | Isis Pharmaceuticals, Inc. | Process for the synthesis of oligomeric compounds |
| TNSN00027A1 (fr) | 1999-02-12 | 2005-11-10 | Vertex Pharma | Inhibiteurs de l'aspartyle protease |
| EP1156812A4 (en) | 1999-02-23 | 2004-09-29 | Isis Pharmaceuticals Inc | MULTIPARTICULAR FORMULATION |
| US7084125B2 (en) | 1999-03-18 | 2006-08-01 | Exiqon A/S | Xylo-LNA analogues |
| US7053207B2 (en) | 1999-05-04 | 2006-05-30 | Exiqon A/S | L-ribo-LNA analogues |
| US6593466B1 (en) | 1999-07-07 | 2003-07-15 | Isis Pharmaceuticals, Inc. | Guanidinium functionalized nucleotides and precursors thereof |
| US6147200A (en) | 1999-08-19 | 2000-11-14 | Isis Pharmaceuticals, Inc. | 2'-O-acetamido modified monomers and oligomers |
| AU2001227965A1 (en) | 2000-01-21 | 2001-07-31 | Geron Corporation | 2'-arabino-fluorooligonucleotide n3'-p5'phosphoramidates: their synthesis and use |
| US7700341B2 (en) * | 2000-02-03 | 2010-04-20 | Dendreon Corporation | Nucleic acid molecules encoding transmembrane serine proteases, the encoded proteins and methods based thereon |
| IT1318539B1 (it) | 2000-05-26 | 2003-08-27 | Italfarmaco Spa | Composizioni farmaceutiche a rilascio prolungato per lasomministrazione parenterale di sostanze idrofile biologicamente |
| JP4413493B2 (ja) | 2000-10-04 | 2010-02-10 | サンタリス ファーマ アー/エス | プリンlna類似体の改善された合成方法 |
| AU2002323151A1 (en) | 2001-08-13 | 2003-03-03 | University Of Pittsburgh | Application of lipid vehicles and use for drug delivery |
| US7795422B2 (en) | 2002-02-20 | 2010-09-14 | Sirna Therapeutics, Inc. | RNA interference mediated inhibition of hypoxia inducible factor 1 (HIF1) gene expression using short interfering nucleic acid (siNA) |
| US8101348B2 (en) | 2002-07-10 | 2012-01-24 | Max-Planck-Gesellschaft Zur Foerderung Der Wissenschaften E.V. | RNA-interference by single-stranded RNA molecules |
| US6878805B2 (en) | 2002-08-16 | 2005-04-12 | Isis Pharmaceuticals, Inc. | Peptide-conjugated oligomeric compounds |
| NZ540779A (en) | 2002-11-01 | 2008-05-30 | Univ Pennsylvania | Compositions and methods for siRNA inhibition of HIF-1 alpha |
| AU2003295600A1 (en) | 2002-11-14 | 2004-06-15 | Dharmacon, Inc. | Functional and hyperfunctional sirna |
| EP1661905B9 (en) | 2003-08-28 | 2012-12-19 | IMANISHI, Takeshi | Novel artificial nucleic acids of n-o bond crosslinkage type |
| ATE452188T1 (de) | 2004-02-10 | 2010-01-15 | Sirna Therapeutics Inc | Rna-interferenz-vermittelte hemmung der genexpression unter verwendung multifunktioneller sina (short interfering nucleic acid) |
| WO2005116204A1 (ja) | 2004-05-11 | 2005-12-08 | Rnai Co., Ltd. | Rna干渉を生じさせるポリヌクレオチド、および、これを用いた遺伝子発現抑制方法 |
| JP2008537551A (ja) | 2005-03-31 | 2008-09-18 | カランド ファーマシューティカルズ, インコーポレイテッド | リボヌクレオチドレダクターゼサブユニット2の阻害剤およびその使用 |
| JP5219819B2 (ja) | 2005-10-21 | 2013-06-26 | カタリスト・バイオサイエンシーズ・インコーポレイテッド | 補体活性化を阻害する修飾プロテアーゼ |
| CN101370818A (zh) * | 2005-11-01 | 2009-02-18 | 阿尔尼拉姆医药品有限公司 | RNAi抑制流感病毒的复制 |
| WO2007090071A2 (en) | 2006-01-27 | 2007-08-09 | Isis Pharmaceuticals, Inc. | 6-modified bicyclic nucleic acid analogs |
| EP1989307B1 (en) | 2006-02-08 | 2012-08-08 | Quark Pharmaceuticals, Inc. | NOVEL TANDEM siRNAS |
| KR101221589B1 (ko) | 2006-04-07 | 2013-01-15 | 이데라 파마슈티칼즈, 인코포레이티드 | Tlr7 및 tlr8에 대한 안정화된 면역 조절성 rna〔simra〕 화합물 |
| CA2927045A1 (en) | 2006-10-03 | 2008-04-10 | Muthiah Manoharan | Lipid containing formulations |
| EP2357231A2 (en) | 2007-07-09 | 2011-08-17 | Idera Pharmaceuticals, Inc. | Stabilized immune modulatory RNA (SIMRA) compounds |
| CA2721333C (en) | 2008-04-15 | 2020-12-01 | Protiva Biotherapeutics, Inc. | Novel lipid formulations for nucleic acid delivery |
| WO2010141511A2 (en) | 2009-06-01 | 2010-12-09 | Halo-Bio Rnai Therapeutics, Inc. | Polynucleotides for multivalent rna interference, compositions and methods of use thereof |
| KR101766408B1 (ko) | 2009-06-10 | 2017-08-10 | 알닐람 파마슈티칼스 인코포레이티드 | 향상된 지질 조성물 |
| US9512164B2 (en) | 2009-07-07 | 2016-12-06 | Alnylam Pharmaceuticals, Inc. | Oligonucleotide end caps |
| WO2011005860A2 (en) | 2009-07-07 | 2011-01-13 | Alnylam Pharmaceuticals, Inc. | 5' phosphate mimics |
| EP2470656B1 (en) | 2009-08-27 | 2015-05-06 | Idera Pharmaceuticals, Inc. | Composition for inhibiting gene expression and uses thereof |
| US8501930B2 (en) * | 2010-12-17 | 2013-08-06 | Arrowhead Madison Inc. | Peptide-based in vivo siRNA delivery system |
| EP2723758B1 (en) * | 2011-06-21 | 2018-06-20 | Alnylam Pharmaceuticals, Inc. | Angiopoietin-like 3 (angptl3) irna compostions and methods of use thereof |
| HRP20191883T1 (hr) * | 2011-11-07 | 2019-12-27 | Ionis Pharmaceuticals, Inc. | Modulacija ekspresije tmprss6 |
| SI3366775T2 (sl) | 2011-11-18 | 2025-12-31 | Alnylam Pharmaceuticals, Inc. | Modificirana sredstva RNAI |
| KR102234623B1 (ko) | 2013-05-22 | 2021-04-02 | 알닐람 파마슈티칼스 인코포레이티드 | Tmprss6 조성물 및 이의 사용 방법 |
| WO2016085852A1 (en) | 2014-11-24 | 2016-06-02 | Alnylam Pharmaceuticals, Inc. | Tmprss6 irna compositions and methods of use thereof |
| US10799808B2 (en) | 2018-09-13 | 2020-10-13 | Nina Davis | Interactive storytelling kit |
| KR102318555B1 (ko) | 2020-03-19 | 2021-10-29 | 한국과학기술연구원 | 광소자용 역나노콘과 그 제조방법 |
-
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- 2014-05-22 IL IL285780A patent/IL285780B/en unknown
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Patent Citations (5)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO1998013526A1 (en) * | 1996-09-26 | 1998-04-02 | Oligos Etc. Inc. | Three component chimeric antisense oligonucleotides |
| WO2009073809A2 (en) * | 2007-12-04 | 2009-06-11 | Alnylam Pharmaceuticals, Inc. | Carbohydrate conjugates as delivery agents for oligonucleotides |
| WO2010033246A1 (en) * | 2008-09-22 | 2010-03-25 | Rxi Pharmaceuticals Corporation | Rna interference in skin indications |
| WO2010148013A2 (en) * | 2009-06-15 | 2010-12-23 | Alnylam Pharmaceuticals, Inc. | Lipid formulated dsrna targeting the pcsk9 gene |
| WO2012135246A2 (en) * | 2011-03-29 | 2012-10-04 | Alnylam Pharmaceuticals, Inc. | Compositions and methods for inhibiting expression of tmprss6 gene |
Cited By (2)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CN117136236A (zh) * | 2022-09-29 | 2023-11-28 | 广州必贝特医药股份有限公司 | 用于抑制TMPRSS6基因表达的siRNA或其盐、药物及其应用 |
| CN117136236B (zh) * | 2022-09-29 | 2024-03-15 | 广州必贝特医药股份有限公司 | 用于抑制TMPRSS6基因表达的siRNA或其盐、药物及其应用 |
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