JP2016522674A5 - - Google Patents

Download PDF

Info

Publication number
JP2016522674A5
JP2016522674A5 JP2015512857A JP2015512857A JP2016522674A5 JP 2016522674 A5 JP2016522674 A5 JP 2016522674A5 JP 2015512857 A JP2015512857 A JP 2015512857A JP 2015512857 A JP2015512857 A JP 2015512857A JP 2016522674 A5 JP2016522674 A5 JP 2016522674A5
Authority
JP
Japan
Prior art keywords
oligonucleotide
nucleotide
nucleotides
sequence
stranded oligonucleotide
Prior art date
Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
Pending
Application number
JP2015512857A
Other languages
English (en)
Japanese (ja)
Other versions
JP2016522674A (ja
Filing date
Publication date
Application filed filed Critical
Priority claimed from PCT/US2013/041437 external-priority patent/WO2013173637A1/en
Publication of JP2016522674A publication Critical patent/JP2016522674A/ja
Publication of JP2016522674A5 publication Critical patent/JP2016522674A5/ja
Pending legal-status Critical Current

Links

JP2015512857A 2012-05-16 2013-05-16 遺伝子発現を調節するための組成物及び方法 Pending JP2016522674A (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201261648077P 2012-05-16 2012-05-16
US61/648,077 2012-05-16
PCT/US2013/041437 WO2013173637A1 (en) 2012-05-16 2013-05-16 Compositions and methods for modulating gene expression

Publications (2)

Publication Number Publication Date
JP2016522674A JP2016522674A (ja) 2016-08-04
JP2016522674A5 true JP2016522674A5 (enExample) 2016-09-15

Family

ID=49584304

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2015512857A Pending JP2016522674A (ja) 2012-05-16 2013-05-16 遺伝子発現を調節するための組成物及び方法

Country Status (4)

Country Link
US (1) US20150141320A1 (enExample)
EP (1) EP2850184A4 (enExample)
JP (1) JP2016522674A (enExample)
WO (1) WO2013173637A1 (enExample)

Families Citing this family (94)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP3489360A3 (en) 2010-07-19 2019-08-28 Ionis Pharmaceuticals, Inc. Modulation of nuclear-retained rna
US9920317B2 (en) 2010-11-12 2018-03-20 The General Hospital Corporation Polycomb-associated non-coding RNAs
AU2011325956B2 (en) 2010-11-12 2016-07-14 The General Hospital Corporation Polycomb-associated non-coding RNAs
AU2013262709A1 (en) 2012-05-16 2015-01-22 Rana Therapeutics, Inc. Compositions and methods for modulating MECP2 expression
KR20150030205A (ko) 2012-05-16 2015-03-19 라나 테라퓨틱스, 인크. Smn 유전자 패밀리 발현을 조절하기 위한 조성물 및 방법
EA201492121A1 (ru) 2012-05-16 2015-10-30 Рана Терапьютикс, Инк. Композиции и способы для модулирования экспрессии семейства генов гемоглобина
US10837014B2 (en) 2012-05-16 2020-11-17 Translate Bio Ma, Inc. Compositions and methods for modulating SMN gene family expression
JP2015523853A (ja) 2012-05-16 2015-08-20 ラナ セラピューティクス インコーポレイテッド Atp2a2発現を調節するための組成物及び方法
SG11201407486PA (en) 2012-05-16 2014-12-30 Rana Therapeutics Inc Compositions and methods for modulating utrn expression
WO2014210097A2 (en) * 2013-06-26 2014-12-31 Novartis Animal Health Us. Inc. Markers to predict macrocyclic lactone drug resistance in dirofilaria immitis, the causative agent of heartworm disease
TW201536329A (zh) 2013-08-09 2015-10-01 Isis Pharmaceuticals Inc 用於調節失養性肌強直蛋白質激酶(dmpk)表現之化合物及方法
KR102275504B1 (ko) * 2013-09-05 2021-07-09 사렙타 쎄러퓨틱스, 인코퍼레이티드 산성 알파-글루코시다제 내 안티센스-유도된 엑손2 포함
JP2016534035A (ja) 2013-10-04 2016-11-04 ラナ セラピューティクス インコーポレイテッド 筋萎縮性側索硬化症を治療するための組成物及び方法
US11162096B2 (en) 2013-10-14 2021-11-02 Ionis Pharmaceuticals, Inc Methods for modulating expression of C9ORF72 antisense transcript
WO2015143245A1 (en) 2014-03-19 2015-09-24 Isis Pharmaceuticals, Inc. Methods for modulating ataxin 2 expression
CN116970607A (zh) 2014-03-19 2023-10-31 Ionis制药公司 用于调节共济失调蛋白2表达的组合物
MY192634A (en) 2014-04-01 2022-08-29 Biogen Ma Inc Compositions for modulating sod-1 expression
EP3207138B1 (en) * 2014-10-17 2020-07-15 Alnylam Pharmaceuticals, Inc. Polynucleotide agents targeting aminolevulinic acid synthase-1 (alas1) and uses thereof
US10858650B2 (en) 2014-10-30 2020-12-08 The General Hospital Corporation Methods for modulating ATRX-dependent gene repression
WO2016112132A1 (en) * 2015-01-06 2016-07-14 Ionis Pharmaceuticals, Inc. Compositions for modulating expression of c9orf72 antisense transcript
US10426789B2 (en) 2015-02-26 2019-10-01 Ionis Pharmaceuticals, Inc. Allele specific modulators of P23H rhodopsin
GB201504124D0 (en) 2015-03-11 2015-04-22 Proqr Therapeutics B V Oligonucleotides
WO2016149455A2 (en) 2015-03-17 2016-09-22 The General Hospital Corporation The rna interactome of polycomb repressive complex 1 (prc1)
WO2016167780A1 (en) 2015-04-16 2016-10-20 Ionis Pharmaceuticals, Inc. Compositions for modulating expression of c9orf72 antisense transcript
US10801024B2 (en) * 2015-05-20 2020-10-13 Indiana University Research And Technology Corporation Inhibition of lncRNA HOTAIR and related materials and methods
EP3124619B1 (en) * 2015-07-31 2019-03-06 Menicon Co., Ltd Reagents, method and kit for across and within dog breed glaucoma diagnosis
JP2018538287A (ja) * 2015-12-14 2018-12-27 コールド スプリング ハーバー ラボラトリー 多発性嚢胞腎の処置のためのアンチセンスオリゴマー
CA3006015A1 (en) 2015-12-31 2017-07-06 Ionis Pharmaceuticals, Inc. Methods for reducing ataxin-2 expression
JP6853193B2 (ja) 2016-01-29 2021-03-31 協和キリン株式会社 核酸複合体
WO2017134252A1 (en) * 2016-02-05 2017-08-10 INSERM (Institut National de la Santé et de la Recherche Médicale) Antisense oligonucleotides effective to reduce the expression of menin in cancer cells of a subject
MY200252A (en) 2016-04-18 2023-12-16 Sarepta Therapeutics Inc Antisense oligomers and methods of using the same for treating diseases associated with the acid alpha-glucosidase gene
CN109477097A (zh) 2016-04-20 2019-03-15 Jbs科学公司 检测CTNNBl和HTERT突变的试剂盒和方法及其在HCC检测和疾病管理中的用途
CA3023514A1 (en) 2016-06-17 2017-12-21 Ionis Pharmaceuticals, Inc. Modulation of gys1 expression
EP3498724B1 (en) 2016-06-30 2023-06-21 Kyowa Kirin Co., Ltd. Nucleic acid complex
EP3478839A1 (en) 2016-07-01 2019-05-08 H. Hoffnabb-La Roche Ag Antisense oligonucleotides for modulating htra1 expression
US11655470B2 (en) * 2016-07-05 2023-05-23 The Usa, As Represented By The Secretary, Dept. Of Health And Human Services Diagnosing COL6-related disorders and methods for treating same
JOP20190104A1 (ar) 2016-11-10 2019-05-07 Ionis Pharmaceuticals Inc مركبات وطرق لتقليل التعبير عن atxn3
US11118182B2 (en) 2016-11-17 2021-09-14 Ramot At Tel-Aviv University Ltd. Modulators of human KAI1 metastasis suppressor gene, methods and uses thereof
AU2017368050A1 (en) 2016-11-29 2019-06-20 Puretech Lyt, Inc. Exosomes for delivery of therapeutic agents
US11352625B2 (en) 2017-01-12 2022-06-07 Duke University Compositions and methods for disrupting the molecular mechanisms associated with mitochondrial dysfunction and neurodegenerative disease
WO2018180005A1 (ja) * 2017-03-31 2018-10-04 国立研究開発法人日本医療研究開発機構 コンドロイチン硫酸生合成を阻害するアンチセンス核酸
GB201706009D0 (en) * 2017-04-13 2017-05-31 Proqr Therapeutics Ii Bv Antisense oligonucleotides for the treatment of stargardt disease
US20190055564A1 (en) * 2017-06-01 2019-02-21 F. Hoffmann-La Roche Ag Antisense oligonucleotides for modulating htra1 expression
EP4219715A3 (en) 2017-09-08 2023-09-06 MiNA Therapeutics Limited Stabilized cebpa sarna compositions and methods of use
CA3075205A1 (en) 2017-09-08 2019-03-14 Mina Therapeutics Limited Stabilized hnf4a sarna compositions and methods of use
US20230201357A1 (en) * 2017-11-08 2023-06-29 Aptamer Diagnostic, Inc. D-dimer-specific aptamers and methods of use in diagnostics, therapeutic and theranostic purposes
TWI809004B (zh) 2017-11-09 2023-07-21 美商Ionis製藥公司 用於降低snca表現之化合物及方法
WO2019109001A1 (en) * 2017-12-01 2019-06-06 The Texas A&M University System Angelman syndrome antisense treatment
RU2020125769A (ru) * 2018-02-09 2022-03-10 Дженентек, Инк. Олигонуклеотиды для модуляции экспрессии tmem106b
CA3091789A1 (en) * 2018-02-21 2019-08-29 Bristol-Myers Squibb Company Camk2d antisense oligonucleotides and uses thereof
US11732260B2 (en) 2018-03-02 2023-08-22 Ionis Pharmaceuticals, Inc. Compounds and methods for the modulation of amyloid-β precursor protein
TWI840345B (zh) 2018-03-02 2024-05-01 美商Ionis製藥公司 Irf4表現之調節劑
CA3094020A1 (en) 2018-04-11 2019-10-17 Ionis Pharmaceuticals, Inc. Modulators of ezh2 expression
SG11202010215TA (en) 2018-05-09 2020-11-27 Ionis Pharmaceuticals Inc Compounds and methods for reducing atxn3 expression
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
WO2020007772A1 (en) * 2018-07-02 2020-01-09 Roche Innovation Center Copenhagen A/S Antisense oligonucleotides targeting gbp-1
WO2020007700A1 (en) * 2018-07-02 2020-01-09 Roche Innovation Center Copenhagen A/S Antisense oligonucleotides targeting spi1
WO2020011653A1 (en) * 2018-07-09 2020-01-16 Roche Innovation Center Copenhagen A/S Antisense oligonucleotides targeting kynu
EP3824086A1 (en) * 2018-07-19 2021-05-26 Stichting Katholieke Universiteit Antisense oligonucleotides rescue aberrant splicing of abca4
CA3106986A1 (en) 2018-07-25 2020-01-30 Ionis Pharmaceuticals, Inc. Compounds and methods for reducing atxn2 expression
CN112912112B (zh) * 2018-10-26 2024-12-17 Vrije布鲁塞尔大学 肝特异性核酸调节元件以及其方法及用途
TW202028222A (zh) 2018-11-14 2020-08-01 美商Ionis製藥公司 Foxp3表現之調節劑
MX2021008918A (es) 2019-01-31 2021-08-24 Ionis Pharmaceuticals Inc Moduladores de expresion yap1.
AU2020239987A1 (en) * 2019-03-15 2021-11-04 University Of Massachusetts Oligonucleotides for tissue specific ApoE modulation
CA3135180A1 (en) 2019-03-29 2020-10-08 Ionis Pharmaceuticals, Inc. Compounds and methods for modulating ube3a-ats
US11739324B2 (en) 2019-06-25 2023-08-29 Stichting Katholieke Universiteit Antisense oligonucleotides rescue aberrant splicing of ABCA4
EP4017539A1 (en) 2019-08-19 2022-06-29 MiNA Therapeutics Limited Oligonucleotide conjugate compositions and methods of use
US20220290147A1 (en) * 2019-08-30 2022-09-15 Baylor College Of Medicine System for regulating gene expression
EP3822370A1 (en) * 2019-11-15 2021-05-19 Curiosity Diagnostics Sp. z o.o. Method of determining the presence of a hyper-virulent clostridioides difficile strain of the b1/nap1/027 group in a sample
WO2021126997A1 (en) * 2019-12-18 2021-06-24 Petdx, Inc. Methods and compositions for cancer detection, characterization or management in companion animals
US20230127437A1 (en) * 2020-01-31 2023-04-27 Sanwa Kagaku Kenkyusho Co., Ltd. Antisense oligonucleotide of atn1
WO2021163281A1 (en) * 2020-02-12 2021-08-19 Accutar Biotechnology Inc. Antisense oligonucleotides and their use for treating pendred syndrome
JPWO2021177418A1 (enExample) * 2020-03-04 2021-09-10
WO2021222654A1 (en) * 2020-04-29 2021-11-04 Saliogen Therapeutics, Inc. Compositions and methods for treatment of inherited macular degeneration
IL298530A (en) 2020-06-29 2023-01-01 Ionis Pharmaceuticals Inc Compounds and methods for modulating plp1
BR112023000988A2 (pt) * 2020-07-22 2023-03-28 The Florey Inst Of Neuroscience And Mental Health Composições e métodos para o tratamento de distúrbios associados a mutações de perda de função em scn2a
KR20230043914A (ko) 2020-07-28 2023-03-31 아이오니스 파마수티컬즈, 인코포레이티드 App 발현을 감소시키기 위한 화합물 및 방법
WO2022032017A2 (en) * 2020-08-07 2022-02-10 The General Hospital Corporation Human xist antisense oligonucleotides for x reactivation therapy
EP4015040A1 (en) * 2020-12-16 2022-06-22 Fundación para la Formación e Investigación Sanitarias de la Región de Murcia Method for treating blood diseases
US12018261B2 (en) 2020-12-18 2024-06-25 Ionis Pharmaceuticals, Inc. Compounds and methods for modulating factor XII
US20220370599A1 (en) * 2021-04-30 2022-11-24 Greenlight Biosciences, Inc. Messenger rna therapeutics and compositions
EP4355759A4 (en) 2021-06-18 2026-05-13 Ionis Pharmaceuticals Inc COMPOUNDS AND METHODS FOR REDUCING THE EXPRESSION OF IFNAR1
MX2024000996A (es) 2021-07-21 2024-06-28 AcuraStem Incorporated Oligonucleótido antisentido unc13a.
US11833221B2 (en) 2021-09-01 2023-12-05 Ionis Pharmaceuticals, Inc. Oligomeric compounds for reducing DMPK expression
CN118541484A (zh) * 2021-12-17 2024-08-23 豪夫迈·罗氏有限公司 反义寡核苷酸
WO2023139360A1 (en) * 2022-01-19 2023-07-27 Autolus Limited Nucleic acid construct
EP4590841A2 (en) * 2022-09-20 2025-07-30 Aldevron, LLC Best1 vectors and uses thereof
UY40699A (es) 2023-04-05 2024-08-30 Ionis Pharmaceuticals Inc Compuestos y métodos para reducir la expresión de pln
GB202312098D0 (en) * 2023-08-08 2023-09-20 Ucl Business Ltd Therapeutic molecules
AU2024339330A1 (en) * 2023-09-13 2026-04-02 Haya Therapeutics Sa Compositions and methods for modulating wisp2 super-enhancer-associated rna
WO2025072672A2 (en) * 2023-09-27 2025-04-03 Judo Bio, Inc. Slc6a19-targeting modulatory nucleic acid agents
GB202401412D0 (en) * 2024-02-02 2024-03-20 Harness Therapeutics Ltd Functional nucleic acid
WO2026033502A1 (en) * 2024-08-09 2026-02-12 Associação Biopolis Kit, method, and uses thereof
WO2026074434A1 (en) * 2024-10-01 2026-04-09 Fondazione Istituto Italiano Di Tecnologia Rad51/brca2 complex aptamers and uses thereof

Family Cites Families (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP3514779B2 (ja) * 1991-04-17 2004-03-31 株式会社アズウェル アポリポプロテインe遺伝子タイプの検査方法及びその検査に好適なプライマー及びプローブ
WO2008025069A1 (en) * 2006-08-28 2008-03-06 The Walter And Eliza Hall Institute Of Medical Research Methods of modulating cellular activity and compositions therefor
AU2008206168B2 (en) * 2007-01-19 2012-01-19 The Regents Of The University Of Michigan ADRB2 cancer markers
WO2008103761A2 (en) * 2007-02-20 2008-08-28 Sequenom, Inc. Methods and compositions for cancer diagnosis and treatment based on nucleic acid methylation
CN104557914A (zh) * 2008-09-26 2015-04-29 新加坡科技研究局 3-脱氮瓶菌素衍生物
US20120004278A1 (en) * 2010-06-18 2012-01-05 The Board Of Trustees Of The Leland Stanford Junior University Linc rnas in cancer diagnosis and treatment

Similar Documents

Publication Publication Date Title
JP2015523853A5 (enExample)
JP2015518712A5 (enExample)
JP2015519057A5 (enExample)
JP2015518710A5 (enExample)
JP2015523855A5 (enExample)
JP2015518713A5 (enExample)
JP2016521556A5 (enExample)
JP2015518711A5 (enExample)
JP2015523854A5 (enExample)
HRP20201200T1 (hr) OLIGONUKLEOTIDNI SPOJEVI ZA CILJANJE HUNTINGTIN mRNK
EP4455285A3 (en) Nucleic acid, composition and conjugate comprising the same, and preparation method and use thereof
HRP20210612T1 (hr) Postupci i pripravci za specifičnu inhibiciju glikolat oksidaze (hao1) pomoću dvolančane rnk
JP2015518714A5 (enExample)
JP2016520310A5 (enExample)
JP2013226147A5 (enExample)
JP2018529732A5 (enExample)
JP2016531570A (ja) ユークロマチン領域を標的とするオリゴヌクレオチド
EP3033114A1 (en) Heterochromatin forming non-coding rnas
JP2016502858A5 (enExample)
JP2014527401A5 (enExample)
IL300119A (en) Oligonucleotides to induce paternal UBE3A expression
JP2018507711A5 (enExample)
JP2016116520A5 (enExample)
JP2008523094A5 (enExample)
JP2016513976A5 (enExample)