JP6139671B2 - 細胞内貫通能を持ってｒｎａ干渉を誘導する核酸分子およびその用途 - Google Patents

細胞内貫通能を持ってｒｎａ干渉を誘導する核酸分子およびその用途 Download PDF

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JP6139671B2
JP6139671B2 JP2015513901A JP2015513901A JP6139671B2 JP 6139671 B2 JP6139671 B2 JP 6139671B2 JP 2015513901 A JP2015513901 A JP 2015513901A JP 2015513901 A JP2015513901 A JP 2015513901A JP 6139671 B2 JP6139671 B2 JP 6139671B2
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nucleic acid
strand
acid molecule
ctgf
lasirna
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ソンウ ホン
ソンウ ホン
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Olix Pharmaceuticals Inc
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JP2015513901A 2012-05-22 2013-05-21 細胞内貫通能を持ってｒｎａ干渉を誘導する核酸分子およびその用途 Active JP6139671B2 (ja)

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KR10-2012-0053950 2012-05-22
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

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JP2016250474A Division JP6629712B2 (ja) 2012-05-22 2016-12-26 細胞内貫通能を持ってｒｎａ干渉を誘導する核酸分子およびその用途

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JP2015518721A JP2015518721A (ja) 2015-07-06
JP2015518721A5 JP2015518721A5 (enExample) 2016-06-23
JP6139671B2 true JP6139671B2 (ja) 2017-05-31

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JP2016250474A Active JP6629712B2 (ja) 2012-05-22 2016-12-26 細胞内貫通能を持ってｒｎａ干渉を誘導する核酸分子およびその用途
JP2019031549A Active JP6999590B2 (ja) 2012-05-22 2019-02-25 細胞内貫通能を持ってｒｎａ干渉を誘導する核酸分子およびその用途

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JP2019031549A Active JP6999590B2 (ja) 2012-05-22 2019-02-25 細胞内貫通能を持ってｒｎａ干渉を誘導する核酸分子およびその用途

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US (3) US10125362B2 (enExample)
EP (2) EP3514236A1 (enExample)
JP (3) JP6139671B2 (enExample)
KR (2) KR101567576B1 (enExample)
CN (2) CN108148838A (enExample)
DK (1) DK2853597T3 (enExample)
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Families Citing this family (38)

* Cited by examiner, † Cited by third party
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

Family Cites Families (90)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5837258A (en) 1991-08-30 1998-11-17 University Of South Florida Induction of tissue, bone or cartilage formation using connective tissue growth factor
DE10160151A1 (de) 2001-01-09 2003-06-26 Ribopharma Ag Verfahren zur Hemmung der Expression eines vorgegebenen Zielgens
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
KR100909681B1 (ko) 2000-12-01 2009-07-29 막스-플랑크-게젤샤프트 츄어 푀르더룽 데어 비쎈샤프텐 에.파우. Ｒｎａ 간섭을 매개하는 작은 ｒｎａ 분자
US20080188430A1 (en) 2001-05-18 2008-08-07 Sirna Therapeutics, Inc. RNA interference mediated inhibition of hypoxia inducible factor 1 (HIF1) gene expression using short interfering nucleic acid (siNA)
US20050282188A1 (en) 2001-05-18 2005-12-22 Sirna Therapeutics, Inc. RNA interference mediated inhibition of gene expression using short interfering nucleic acid (siNA)
CN1608133A (zh) * 2001-10-26 2005-04-20 里伯药品公司 双链核糖核酸用于治疗正(+)链rna病毒感染的用途
US20040138163A1 (en) 2002-05-29 2004-07-15 Mcswiggen James RNA interference mediated inhibition of vascular edothelial growth factor and vascular edothelial growth factor receptor gene expression using short interfering nucleic acid (siNA)
BRPI0313202A8 (pt) * 2002-08-05 2016-08-16 Atugen Ag Formas adicionais para interferir com as moléculas de rna
US8090542B2 (en) 2002-11-14 2012-01-03 Dharmacon Inc. Functional and hyperfunctional siRNA
WO2006006948A2 (en) * 2002-11-14 2006-01-19 Dharmacon, Inc. METHODS AND COMPOSITIONS FOR SELECTING siRNA OF IMPROVED FUNCTIONALITY
AU2004220556B2 (en) 2003-03-07 2009-05-07 Alnylam Pharmaceuticals, Inc. Therapeutic compositions
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
NZ545360A (en) * 2003-08-28 2009-06-26 Novartis Ag Interfering RNA duplex having blunt-ends and 3'-modifications
WO2005062937A2 (en) 2003-12-22 2005-07-14 University Of Massachusetts Methods and compositions for enhancing the efficacy and specificity of single and double blunt-ended sirna
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
WO2005079533A2 (en) 2004-02-17 2005-09-01 University Of Massachusetts Methods and compositions for mediating gene silencing
WO2005089224A2 (en) * 2004-03-12 2005-09-29 Alnylam Pharmaceuticals, Inc. iRNA AGENTS TARGETING VEGF
KR101147147B1 (ko) 2004-04-01 2012-05-25 머크 샤프 앤드 돔 코포레이션 Ｒｎａ 간섭의 오프 타겟 효과 감소를 위한 변형된폴리뉴클레오타이드
WO2006031901A2 (en) 2004-09-10 2006-03-23 Somagenics, Inc. SMALL INTERFERING RNAs THAT EFFICIENTLY INHIBIT VIRAL GENE EXPRESSION AND METHODS OF USE THEREOF
CN101133074B (zh) * 2004-09-24 2012-05-30 阿尔尼拉姆医药品有限公司 APOB的RNAi调节及其用途
TWI386225B (zh) 2004-12-23 2013-02-21 Alcon Inc 用於治療眼睛病症的結締組織生長因子（ＣＴＧＦ）ＲＮＡ干擾（ＲＮＡｉ）抑制技術
US20060142228A1 (en) 2004-12-23 2006-06-29 Ambion, Inc. Methods and compositions concerning siRNA's as mediators of RNA interference
CN101184839A (zh) * 2005-04-08 2008-05-21 纳斯泰克制药公司 用于呼吸道病毒感染的RNAi治疗
ATE546526T1 (de) 2005-05-25 2012-03-15 Univ York Hybrid-interferenz-rna
CA2612047C (en) 2005-06-24 2015-05-12 Intervet International B.V. Inactivated chimeric vaccines and related methods of use
AU2006272808C1 (en) * 2005-07-21 2010-10-21 Alnylam Pharmaceuticals, Inc. RNAi modulation of the Rho-A gene and uses thereof
WO2007022470A2 (en) * 2005-08-18 2007-02-22 Alnylam Pharmaceuticals, Inc. Methods and compositions for treating neurological disease
FR2890859B1 (fr) 2005-09-21 2012-12-21 Oreal Oligonucleotide d'arn double brin inhibant l'expression de la tyrosinase
WO2007041282A2 (en) 2005-09-29 2007-04-12 The Johns Hopkins University Methods and compositions for treatment of cystic fibrosis
US7825099B2 (en) 2006-01-20 2010-11-02 Quark Pharmaceuticals, Inc. Treatment or prevention of oto-pathologies by inhibition of pro-apoptotic genes
AU2007210034A1 (en) 2006-01-26 2007-08-09 University Of Massachusetts RNA silencing agents for use in therapy and nanotransporters for efficient delivery of same
JP5829373B2 (ja) 2006-01-27 2015-12-09 バイオジェン・エムエイ・インコーポレイテッドＢｉｏｇｅｎ ＭＡ Ｉｎｃ． Ｎｏｇｏレセプターアンタゴニスト
US20070218495A1 (en) 2006-03-16 2007-09-20 Dharmacon, Inc. Methods, libraries and computer program products for gene silencing with reduced off-target effects
US7700541B2 (en) 2006-04-06 2010-04-20 Nitto Denko Corporation Biodegradable cationic polymers
GB0608838D0 (en) * 2006-05-04 2006-06-14 Novartis Ag Organic compounds
US20100105134A1 (en) 2007-03-02 2010-04-29 Mdrna, Inc. Nucleic acid compounds for inhibiting gene expression and uses thereof
EP2121924A1 (en) 2007-03-02 2009-11-25 MDRNA, Inc. Nucleic acid compounds for inhibiting vegf family gene expression and uses thereof
PT2164967E (pt) 2007-05-31 2015-10-27 Univ Iowa Res Found Redução da toxicidade não dirigida do arn de interferência
WO2009002462A1 (en) 2007-06-22 2008-12-31 The Board Of Trustees Of The Leland Stanford Junior University Pre-mirna loop-modulated target regulation
WO2009020344A2 (en) 2007-08-06 2009-02-12 Postech Acad Ind Found Small interfering rnas (sirnas) controlling multiple target genes and method for preparing the same
DK2193140T3 (en) 2007-08-27 2017-01-23 Iglobe Health Inst Llc COMPOSITIONS OF ASYMMETRIC INTERFERRING RNA AND ITS APPLICATIONS
EP2548962B1 (en) 2007-09-19 2016-01-13 Applied Biosystems, LLC Sirna sequence-independent modification formats for reducing off-target phenotypic effects in rnai, and stabilized forms thereof
EP2205740A2 (en) * 2007-10-02 2010-07-14 Rxi Pharmaceuticals Corp. Tripartite rnai constructs
WO2009044392A2 (en) * 2007-10-03 2009-04-09 Quark Pharmaceuticals, Inc. Novel sirna structures
US8614311B2 (en) 2007-12-12 2013-12-24 Quark Pharmaceuticals, Inc. RTP801L 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
EP2318528A1 (en) 2008-07-24 2011-05-11 Rxi Pharmaceuticals Corporation Rnai constructs and uses therof
US8946172B2 (en) * 2008-08-25 2015-02-03 Excaliard Pharmaceuticals, Inc. Method for reducing scarring during wound healing using antisense compounds directed to CTGF
JP5294344B2 (ja) 2008-09-08 2013-09-18 学校法人福岡大学 白血病治療用医薬組成物
US8796443B2 (en) 2008-09-22 2014-08-05 Rxi Pharmaceuticals Corporation Reduced size self-delivering RNAi compounds
US20100227920A1 (en) 2008-09-29 2010-09-09 The Regents Of The University Of California Aldehyde dehydrogenase inhibitors as novel depigmenting agents
WO2010090762A1 (en) 2009-02-04 2010-08-12 Rxi Pharmaceuticals Corporation Rna duplexes with single stranded phosphorothioate nucleotide regions for additional functionality
US8829179B2 (en) 2009-02-18 2014-09-09 Silence Therapeutics Gmbh Means for inhibiting the expression of ANG2
EP2408916A2 (en) * 2009-03-19 2012-01-25 Merck Sharp&Dohme Corp. RNA INTERFERENCE MEDIATED INHIBITION OF CONNECTIVE TISSUE GROWTH FACTOR (CTGF) GENE EXPRESSION USING SHORT INTERFERING NUCLEIC ACID (siNA)
CN102575254B (zh) 2009-04-03 2015-03-04 戴瑟纳制药公司 利用不对称双链rna特异性抑制kras的方法和组合物
WO2011019887A2 (en) 2009-08-14 2011-02-17 Massachusetts Institute Of Technology Waveguide coupler having continuous three-dimensional tapering
KR101237036B1 (ko) * 2009-11-04 2013-02-25 성균관대학교산학협력단 안티센스 가닥에 의한 오프-타겟 효과를 최소화한 신규한 ｓｉＲＮＡ 구조 및 그 용도
KR101207561B1 (ko) 2009-12-15 2012-12-04 주식회사 코리아나화장품 티로시나제의 발현을 저해하는 ｓｉＲＮＡ 올리고뉴클레오타이드 및 이를 함유하는 화장료 조성물
WO2011084193A1 (en) 2010-01-07 2011-07-14 Quark Pharmaceuticals, Inc. Oligonucleotide compounds comprising non-nucleotide overhangs
JP5847700B2 (ja) * 2010-03-05 2016-01-27 株式会社Ｗａｖｅ Ｌｉｆｅ Ｓｃｉｅｎｃｅｓ Ｊａｐａｎ リボヌクレオシドホスホロチオエートの製造方法
US9095504B2 (en) * 2010-03-24 2015-08-04 Rxi Pharmaceuticals Corporation RNA interference in ocular indications
CA2834108A1 (en) 2010-05-12 2011-11-17 Val-Chum, Limited Partnership Screening assays based on mag and/or abhd6 for selecting insulin secretion promoting agents
AU2011302152B2 (en) * 2010-09-15 2015-06-11 Alnylam Pharmaceuticals, Inc. Modified iRNA agents
WO2012053741A2 (ko) 2010-10-22 2012-04-26 성균관대학교산학협력단 Rna 간섭을 유도하는 핵산 분자 및 그 용도
EP2649181B1 (en) 2010-12-06 2016-04-27 Quark Pharmaceuticals, Inc. Double stranded oligonucleotide compounds comprising positional modifications
JP6177692B2 (ja) * 2011-02-02 2017-08-09 エクスカリアード・ファーマシューティカルズ，インク 結合組織増殖因子（ｃｔｇｆ）をターゲティングするアンチセンス化合物を用いた、ケロイドまたは肥厚性瘢痕の治療法
EP2681315B1 (en) 2011-03-03 2017-05-03 Quark Pharmaceuticals, Inc. Oligonucleotide modulators of the toll-like receptor pathway
CN102719432B (zh) 2011-03-29 2013-10-23 南京大学 特异性抑制肿瘤凋亡抑制基因Bcl2的双链不对称小核酸干扰分子asiRNA及其应用
KR101590586B1 (ko) 2011-05-30 2016-02-01 성균관대학교산학협력단 표적 유전자 발현 억제 및 면역 반응을 동시에 유발하는 이중가닥의 긴 간섭 ｒｎａ
CN103889461B (zh) 2011-07-18 2016-11-09 肯塔基大学研究基金会 保护细胞免于alu-rna诱导的变性和用于保护细胞的抑制剂
US9707235B1 (en) 2012-01-13 2017-07-18 University Of Kentucky Research Foundation Protection of cells from degeneration and treatment of geographic atrophy
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
WO2014043291A1 (en) 2012-09-12 2014-03-20 Quark Pharmaceuticals, Inc. Double-stranded nucleic acid compounds
US20160122764A1 (en) 2013-07-05 2016-05-05 Bioneer Corporation Respiratory disease-related gene specific sirna, double-helical oligo rna structure containing sirna, compositon containing same for preventing or treating respiratory disease
WO2015015498A1 (en) 2013-07-31 2015-02-05 Qbi Enterprises Ltd. Methods of use of sphingolipid polyalkylamine oligonucleotide compounds
US9790506B2 (en) 2013-10-02 2017-10-17 The Regents Of The University Of California Diagnostic and screening methods for atopic dermatitis
US8980273B1 (en) 2014-07-15 2015-03-17 Kymab Limited Method of treating atopic dermatitis or asthma using antibody to IL4RA
US20170226507A1 (en) 2014-05-05 2017-08-10 The Brigham And Women's Hospital, Inc. Coordinate control of pathogenic signaling by the mir-130/301 family in pulmonary hypertension and fibroproliferative diseases
US9139648B1 (en) 2014-07-15 2015-09-22 Kymab Limited Precision medicine by targeting human NAV1.9 variants for treatment of pain
WO2016161388A1 (en) 2015-04-03 2016-10-06 University Of Massachusetts Fully stabilized asymmetric sirna
CA3022872C (en) 2015-07-27 2024-01-09 Olix Pharmaceuticals, Inc. Rna complexes that inhibit melanin production
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
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
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
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
WO2018146557A2 (en) 2017-02-10 2018-08-16 Dong Ki Lee Long double-stranded rna for rna interference

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