CN104755620B - 具有细胞内穿透能力的诱导rna干扰的核酸分子及其用途 - Google Patents

具有细胞内穿透能力的诱导rna干扰的核酸分子及其用途 Download PDF

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CN104755620B
CN104755620B CN201380038984.6A CN201380038984A CN104755620B CN 104755620 B CN104755620 B CN 104755620B CN 201380038984 A CN201380038984 A CN 201380038984A CN 104755620 B CN104755620 B CN 104755620B
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nucleic acid
strand
acid molecule
lasirna
modification
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CN104755620A (zh
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弘善宇
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Olix Pharmaceuticals Inc
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CN201380038984.6A 2012-05-22 2013-05-21 具有细胞内穿透能力的诱导rna干扰的核酸分子及其用途 Active CN104755620B (zh)

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KR20120053950 2012-05-22
KR10-2012-0053950 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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Families Citing this family (38)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
DK2631291T3 (da) 2010-10-22 2019-06-11 Olix Pharmaceuticals Inc Nukleinsyremolekyler, der inducerer rna-interferens, og anvendelser deraf
KR101567576B1 (ko) 2012-05-22 2015-11-10 올릭스 주식회사 세포 내 관통능을 가지고 ｒｎａ 간섭을 유도하는 핵산 분자 및 그 용도
CA2924343C (en) 2013-09-17 2025-09-16 Bolt Threads, Inc. PROCESSES AND COMPOSITIONS FOR SYNTHESIZING IMPROVED SILK FIBERS
KR102279110B1 (ko) * 2014-04-30 2021-07-20 올릭스 주식회사 LasiRNA를 유효성분으로 포함하는 피부 미백용 조성물
PL3277814T3 (pl) 2015-04-03 2020-11-30 University Of Massachusetts Związki oligonukleotydowe ukierunkowane na mrna huntingtyny
US20160319278A1 (en) 2015-04-03 2016-11-03 University Of Massachusetts Fully stabilized asymmetric sirna
WO2016185934A1 (ja) 2015-05-15 2016-11-24 旭硝子株式会社 化学強化ガラス
EP3331543A4 (en) * 2015-08-06 2019-03-20 City of Hope Cell-pollenizing protein antibody conjugates and methods of use
WO2017030973A1 (en) * 2015-08-14 2017-02-23 University Of Massachusetts Bioactive conjugates for oligonucleotide delivery
JP7027311B2 (ja) 2015-11-16 2022-03-01 オリックス ファーマシューティカルズ，インコーポレーテッド ＭｙＤ８８又はＴＬＲ３を標的とするＲＮＡ複合体を使用した加齢黄斑変性の治療
US10478503B2 (en) 2016-01-31 2019-11-19 University Of Massachusetts Branched oligonucleotides
JP6944942B2 (ja) 2016-02-02 2021-10-06 オリックス ファーマシューティカルズ，インコーポレーテッド ＩＬ４Ｒα、ＴＲＰＡ１、またはＦ２ＲＬ１を標的とするＲＮＡ複合体を用いたアトピー性皮膚炎および喘息の治療
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
JP7049262B2 (ja) * 2016-04-11 2022-04-06 オリックス ファーマシューティカルズ，インコーポレーテッド 結合組織成長因子を標的とするｒｎａ複合体を用いた特発性肺胞線維症の治療
KR101916652B1 (ko) * 2016-06-29 2018-11-08 올릭스 주식회사 작은 간섭 rna의 rna 간섭효과 증진용 화합물 및 이의 용도
CA3033368A1 (en) 2016-08-12 2018-02-15 University Of Massachusetts Conjugated oligonucleotides
EP3580339A4 (en) 2017-02-10 2020-12-23 Research & Business Foundation Sungkyunkwan University LONG DOUBLE STRAND RNA FOR RNA INTERFERENCE
KR102321426B1 (ko) * 2017-02-21 2021-11-05 올릭스 주식회사 남성형 탈모 표적 유전자의 발현을 억제하는 비대칭 siRNA
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
KR20190037166A (ko) * 2017-09-28 2019-04-05 올릭스 주식회사 결합 조직 성장 인자를 표적으로 하는 rna 복합체를 함유하는 노인성 황반변성의 예방 또는 치료용 약학 조성물
HRP20230127T1 (hr) * 2017-11-13 2023-03-31 Silence Therapeutics Gmbh Nukleinske kiseline za inhibiranje ekspresije lpa u stanici
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
WO2020033899A1 (en) 2018-08-10 2020-02-13 University Of Massachusetts Modified oligonucleotides targeting snps
US11279930B2 (en) 2018-08-23 2022-03-22 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
CN113614232A (zh) 2019-01-18 2021-11-05 马萨诸塞大学 动态药代动力学修饰锚
KR102397455B1 (ko) * 2019-05-20 2022-05-13 올릭스 주식회사 PD-1의 발현을 억제하는 비대칭 siRNA
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
WO2021242883A1 (en) 2020-05-26 2021-12-02 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
WO2022124739A1 (ko) * 2020-12-07 2022-06-16 올릭스 주식회사 ROR-beta의 발현을 억제하는 비대칭 RNAi 유도용 핵산 분자
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
CN117677699A (zh) 2021-06-23 2024-03-08 马萨诸塞大学 用于治疗先兆子痫和其他血管生成病症的优化抗flt1寡核苷酸化合物
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
CA2429814C (en) 2000-12-01 2014-02-18 Thomas Tuschl Rna interference mediating small rna molecules
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)
US20050119202A1 (en) 2001-10-26 2005-06-02 Roland Kreutzer Medicament to treat a fibrotic disease
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)
DE60310944T3 (de) * 2002-08-05 2017-08-03 Silence Therapeutics Gmbh Weitere neue formen von interferierende rns moleküle
AU2003295600A1 (en) 2002-11-14 2004-06-15 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
CA2518475C (en) * 2003-03-07 2014-12-23 Alnylam Pharmaceuticals, Inc. Irna agents comprising asymmetrical modifications
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
EP1660657A1 (en) * 2003-08-28 2006-05-31 Novartis AG Interfering rna duplex having blunt-ends and 3'-modifications
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
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
US20060069050A1 (en) 2004-02-17 2006-03-30 University Of Massachusetts Methods and compositions for mediating gene silencing
ES2423060T3 (es) * 2004-03-12 2013-09-17 Alnylam Pharmaceuticals, Inc. Agentes iRNA que tienen como diana al 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
EP2377873B1 (en) * 2004-09-24 2014-08-20 Alnylam Pharmaceuticals, Inc. RNAi modulation of ApoB and uses thereof
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
AU2006235364A1 (en) 2005-04-08 2006-10-19 Marina Biotech, Inc. RNAi therapeutic for respiratory virus infection
EP1891216B1 (en) 2005-05-25 2012-02-22 The University Of York Hybrid interfering 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
EP1937066A4 (en) * 2005-08-18 2008-12-24 Alnylam Pharmaceuticals Inc METHOD AND COMPOSITIONS FOR THE TREATMENT OF NERVOUS DISEASES
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
WO2007089607A2 (en) 2006-01-26 2007-08-09 University Of Massachusetts Rna silencing agents for use in therapy and nanotransporters for efficient delivery of same
PT1981902E (pt) 2006-01-27 2015-11-02 Biogen Ma Inc Antagonistas dos recetores nogo
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
EP2121924A1 (en) 2007-03-02 2009-11-25 MDRNA, Inc. Nucleic acid compounds for inhibiting vegf family gene expression and uses thereof
US20100105134A1 (en) 2007-03-02 2010-04-29 Mdrna, Inc. Nucleic acid compounds for inhibiting gene expression and uses thereof
DK2164967T3 (en) 2007-05-31 2015-10-19 Univ Iowa Res Found Reduction of off-target rna interferenstoksicitet
US20090004668A1 (en) 2007-06-22 2009-01-01 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
EP3889261A1 (en) 2007-08-27 2021-10-06 1Globe Health Institute LLC Compositions of asymmetric interfering rna and uses thereof
EP2195428B1 (en) 2007-09-19 2013-12-11 Applied Biosystems, LLC SiRNA SEQUENCE-INDEPENDENT MODIFICATION FORMATS FOR REDUCING OFF-TARGET PHENOTYPIC EFFECTS IN RNAi, AND STABILIZED FORMS THEREOF
WO2009045457A2 (en) * 2007-10-02 2009-04-09 Rxi Pharmaceuticals Corp. Tripartite rnai constructs
RU2487716C2 (ru) 2007-10-03 2013-07-20 Кварк Фармасьютикалс, Инк. Новые структуры малых интерферирующих рнк (sirna)
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
US20110251258A1 (en) 2008-07-24 2011-10-13 Rxi Pharmaceuticals Corporation Rnai constructs and uses thereof
US8946172B2 (en) * 2008-08-25 2015-02-03 Excaliard Pharmaceuticals, Inc. Method for reducing scarring during wound healing using antisense compounds directed to CTGF
WO2010026660A1 (ja) 2008-09-08 2010-03-11 学校法人福岡大学 Ｚｆａｔ遺伝子発現抑制ｒｎａ
EP2342340A1 (en) * 2008-09-22 2011-07-13 Rxi Pharmaceuticals Corporation Rna interference in skin indications
US20100227920A1 (en) 2008-09-29 2010-09-09 The Regents Of The University Of California Aldehyde dehydrogenase inhibitors as novel depigmenting agents
US9745574B2 (en) 2009-02-04 2017-08-29 Rxi Pharmaceuticals Corporation RNA duplexes with single stranded phosphorothioate nucleotide regions for additional functionality
WO2010094491A1 (en) 2009-02-18 2010-08-26 Silence Therapeutics Ag 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)
CN103555722B (zh) 2009-04-03 2016-08-31 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
CN103200945B (zh) * 2010-03-24 2016-07-06 雷克西制药公司 眼部症候中的rna干扰
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
CA2812046A1 (en) * 2010-09-15 2012-03-22 Alnylam Pharmaceuticals, Inc. Modified irna agents
DK2631291T3 (da) 2010-10-22 2019-06-11 Olix Pharmaceuticals Inc Nukleinsyremolekyler, der inducerer rna-interferens, og anvendelser deraf
AU2011338682B2 (en) 2010-12-06 2017-04-27 Quark Pharmaceuticals, Inc. Double stranded oligonucleotide compounds comprising threose modifications
PT2670411T (pt) * 2011-02-02 2019-06-18 Excaliard Pharmaceuticals Inc Compostos anti sentido visando um fator de crescimento do tecido conetivo (ctfg) para utilização num método de tratamento de queloides ou cicatrizes hipertróficas
CA2828544A1 (en) 2011-03-03 2012-09-07 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
AU2012284223B2 (en) 2011-07-18 2017-06-22 University Of Kentucky Research Foundation Protection of cells from Alu-RNA-induced degenereation and inhibitors for protecting cells
US9707235B1 (en) 2012-01-13 2017-07-18 University Of Kentucky Research Foundation Protection of cells from degeneration and treatment of geographic atrophy
KR101567576B1 (ko) 2012-05-22 2015-11-10 올릭스 주식회사 세포 내 관통능을 가지고 ｒｎａ 간섭을 유도하는 핵산 분자 및 그 용도
US9611473B2 (en) 2012-09-12 2017-04-04 Quark Pharmaceuticals, Inc. Double-stranded nucleic acid compounds
CN110592082A (zh) 2013-07-05 2019-12-20 柏业公司 呼吸疾病相关基因特异性siRNA、含有siRNA的双螺旋寡RNA结构及其用途
EP3027223A1 (en) 2013-07-31 2016-06-08 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
US20160319278A1 (en) 2015-04-03 2016-11-03 University Of Massachusetts Fully stabilized asymmetric sirna
JP7027303B2 (ja) 2015-07-27 2022-03-01 オリックス ファーマシューティカルズ，インコーポレーテッド メラニン産生を抑制するｒｎａ複合体
JP7027311B2 (ja) 2015-11-16 2022-03-01 オリックス ファーマシューティカルズ，インコーポレーテッド ＭｙＤ８８又はＴＬＲ３を標的とするＲＮＡ複合体を使用した加齢黄斑変性の治療
JP6944942B2 (ja) 2016-02-02 2021-10-06 オリックス ファーマシューティカルズ，インコーポレーテッド ＩＬ４Ｒα、ＴＲＰＡ１、またはＦ２ＲＬ１を標的とするＲＮＡ複合体を用いたアトピー性皮膚炎および喘息の治療
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
JP7049262B2 (ja) * 2016-04-11 2022-04-06 オリックス ファーマシューティカルズ，インコーポレーテッド 結合組織成長因子を標的とするｒｎａ複合体を用いた特発性肺胞線維症の治療
KR101916652B1 (ko) 2016-06-29 2018-11-08 올릭스 주식회사 작은 간섭 rna의 rna 간섭효과 증진용 화합물 및 이의 용도
EP3580339A4 (en) 2017-02-10 2020-12-23 Research & Business Foundation Sungkyunkwan University LONG DOUBLE STRAND RNA FOR RNA INTERFERENCE

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