CA2749312A1 - Sugar-modified gapped oligonucleotides - Google Patents

Sugar-modified gapped oligonucleotides Download PDF

Info

Publication number
CA2749312A1
CA2749312A1 CA2749312A CA2749312A CA2749312A1 CA 2749312 A1 CA2749312 A1 CA 2749312A1 CA 2749312 A CA2749312 A CA 2749312A CA 2749312 A CA2749312 A CA 2749312A CA 2749312 A1 CA2749312 A1 CA 2749312A1
Authority
CA
Canada
Prior art keywords
oligonucleotide
oligonucleotides
nucleoside
subsequence
deoxy
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.)
Abandoned
Application number
CA2749312A
Other languages
English (en)
French (fr)
Inventor
Phillip D. Cook
Brett P. Monia
Karl-Heinz Altmann
Pierre Martin
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Novartis AG
Ionis Pharmaceuticals Inc
Original Assignee
Novartis AG
Isis Pharmaceuticals Inc
Priority date (The priority date 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 date listed.)
Filing date
Publication date
Application filed by Novartis AG, Isis Pharmaceuticals Inc filed Critical Novartis AG
Publication of CA2749312A1 publication Critical patent/CA2749312A1/en
Abandoned legal-status Critical Current

Links

Classifications

    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07HSUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
    • C07H21/00Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
    • C07H21/04Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids with deoxyribosyl as saccharide radical
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07HSUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
    • C07H21/00Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00Antineoplastic agents

Landscapes

  • Chemical & Material Sciences (AREA)
  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Organic Chemistry (AREA)
  • Molecular Biology (AREA)
  • Biochemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Biotechnology (AREA)
  • Genetics & Genomics (AREA)
  • Public Health (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Medicinal Chemistry (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Animal Behavior & Ethology (AREA)
  • Saccharide Compounds (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
CA2749312A 1996-02-14 1997-02-07 Sugar-modified gapped oligonucleotides Abandoned CA2749312A1 (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US1162096P 1996-02-14 1996-02-14
US60/011,620 1996-02-14
CA2246229A CA2246229C (en) 1996-02-14 1997-02-07 Sugar-modified gapped oligonucleotides

Related Parent Applications (1)

Application Number Title Priority Date Filing Date
CA2246229A Division CA2246229C (en) 1996-02-14 1997-02-07 Sugar-modified gapped oligonucleotides

Publications (1)

Publication Number Publication Date
CA2749312A1 true CA2749312A1 (en) 1997-08-21

Family

ID=21751248

Family Applications (2)

Application Number Title Priority Date Filing Date
CA2749312A Abandoned CA2749312A1 (en) 1996-02-14 1997-02-07 Sugar-modified gapped oligonucleotides
CA2246229A Expired - Lifetime CA2246229C (en) 1996-02-14 1997-02-07 Sugar-modified gapped oligonucleotides

Family Applications After (1)

Application Number Title Priority Date Filing Date
CA2246229A Expired - Lifetime CA2246229C (en) 1996-02-14 1997-02-07 Sugar-modified gapped oligonucleotides

Country Status (22)

Country Link
US (1) US6451991B1 (cg-RX-API-DMAC10.html)
EP (1) EP0882061B1 (cg-RX-API-DMAC10.html)
JP (1) JP4293636B2 (cg-RX-API-DMAC10.html)
KR (1) KR19990082476A (cg-RX-API-DMAC10.html)
CN (1) CN1214688A (cg-RX-API-DMAC10.html)
AT (1) ATE267207T1 (cg-RX-API-DMAC10.html)
AU (1) AU725262B2 (cg-RX-API-DMAC10.html)
BR (1) BR9707529A (cg-RX-API-DMAC10.html)
CA (2) CA2749312A1 (cg-RX-API-DMAC10.html)
CZ (1) CZ243498A3 (cg-RX-API-DMAC10.html)
DE (1) DE69729179T2 (cg-RX-API-DMAC10.html)
DK (1) DK0882061T3 (cg-RX-API-DMAC10.html)
ES (1) ES2221039T3 (cg-RX-API-DMAC10.html)
HU (1) HUP9901118A3 (cg-RX-API-DMAC10.html)
IL (1) IL125759A0 (cg-RX-API-DMAC10.html)
NO (1) NO983718L (cg-RX-API-DMAC10.html)
NZ (1) NZ331217A (cg-RX-API-DMAC10.html)
PL (1) PL328563A1 (cg-RX-API-DMAC10.html)
SK (1) SK109198A3 (cg-RX-API-DMAC10.html)
TR (1) TR199801581T2 (cg-RX-API-DMAC10.html)
WO (1) WO1997030067A1 (cg-RX-API-DMAC10.html)
ZA (1) ZA971208B (cg-RX-API-DMAC10.html)

Families Citing this family (122)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US6346614B1 (en) * 1992-07-23 2002-02-12 Hybridon, Inc. Hybrid oligonucleotide phosphorothioates
NZ513757A (en) 1999-02-26 2004-12-24 Univ British Columbia Antisense oligonucleotides to inhibit TRPM-2 expression and delay androgen independance in prostatic tumour cells
US7569551B2 (en) 2000-02-25 2009-08-04 The University Of British Columbia Chemo- and radiation-sensitization of cancer by antisense TRPM-2 oligodeoxynucleotides
PT1309726E (pt) 2000-03-30 2010-03-08 Whitehead Biomedical Inst Mediadores de interferência por rna específicos de sequência de rna
EP1191097A1 (en) 2000-09-21 2002-03-27 Leids Universitair Medisch Centrum Induction of exon skipping in eukaryotic cells
HU230458B1 (hu) 2000-12-01 2016-07-28 Europäisches Laboratorium für Molekularbiologie (EMBL) Az RNS interferenciát közvetítő kis RNS molekulák
ES2307942T3 (es) 2002-01-17 2008-12-01 The University Of British Columbia Oligonucleotidos antisentido biespecificos que inhiben igfbp-2 e igfbp-5 y metodos de uso.
CA2524255C (en) 2003-03-21 2014-02-11 Academisch Ziekenhuis Leiden Modulation of exon recognition in pre-mrna by interfering with the secondary rna structure
EP1667731B1 (en) * 2003-10-01 2013-05-22 The University Of British Columbia Bispecific oligonucleotide for the treatment of cns malignancies
KR101147147B1 (ko) 2004-04-01 2012-05-25 머크 샤프 앤드 돔 코포레이션 Rna 간섭의 오프 타겟 효과 감소를 위한 변형된폴리뉴클레오타이드
US8710020B2 (en) * 2004-04-02 2014-04-29 The University Of British Columbia Clusterin antisense therapy for treatment of cancer
EP4272748A3 (en) 2004-06-28 2024-03-27 The University Of Western Australia Antisense oligonucleotides for inducing exon skipping and methods of use thereof
USRE48960E1 (en) 2004-06-28 2022-03-08 The University Of Western Australia Antisense oligonucleotides for inducing exon skipping and methods of use thereof
US20060166234A1 (en) 2004-11-22 2006-07-27 Barbara Robertson Apparatus and system having dry control gene silencing compositions
US7923207B2 (en) 2004-11-22 2011-04-12 Dharmacon, Inc. Apparatus and system having dry gene silencing pools
US7935811B2 (en) 2004-11-22 2011-05-03 Dharmacon, Inc. Apparatus and system having dry gene silencing compositions
EP1814595B1 (en) * 2004-11-23 2014-01-08 The University Of British Columbia Treatment of cancer with a combination of an agent that perturbs the egf signaling pathway and an oligonucleotide that reduces clusterin levels
EP1976567B1 (en) 2005-12-28 2020-05-13 The Scripps Research Institute Natural antisense and non-coding rna transcripts as drug targets
WO2007123391A1 (en) 2006-04-20 2007-11-01 Academisch Ziekenhuis Leiden Therapeutic intervention in a genetic disease in an individual by modifying expression of an aberrantly expressed gene.
EP1857548A1 (en) 2006-05-19 2007-11-21 Academisch Ziekenhuis Leiden Means and method for inducing exon-skipping
US20070275029A1 (en) * 2006-05-26 2007-11-29 Ico Therapeutics Inc. Therapeutic drug combinations and delivery systems comprising c-raf kinase antisense polynucleotides for treating ocular diseases and disorders
DK2049664T3 (da) 2006-08-11 2012-01-02 Prosensa Technologies Bv Enkeltstrengede oligonukleotider, som er komplementære til repetitive elementer, til behandling af med DNA-repetitiv-ustabilitet associerede lidelser
EP2081949B1 (en) 2006-09-22 2014-12-10 GE Healthcare Dharmacon, Inc. Tripartite oligonucleotide complexes and methods for gene silencing by rna interference
EP2505211B1 (en) 2007-07-12 2020-04-08 BioMarin Technologies B.V. Molecules for targeting compounds to various selected organs or tissues
EP2203173B1 (en) 2007-10-26 2015-12-23 Academisch Ziekenhuis Leiden Means and methods for counteracting muscle disorders
USRE48468E1 (en) 2007-10-26 2021-03-16 Biomarin Technologies B.V. Means and methods for counteracting muscle disorders
EP2249874A1 (en) 2008-02-08 2010-11-17 ProSensa Holding BV Methods and means for treating dna repeat instability associated genetic disorders
US8188060B2 (en) 2008-02-11 2012-05-29 Dharmacon, Inc. Duplex oligonucleotides with enhanced functionality in gene regulation
EP2119783A1 (en) 2008-05-14 2009-11-18 Prosensa Technologies B.V. Method for efficient exon (44) skipping in Duchenne Muscular Dystrophy and associated means
WO2010040112A2 (en) 2008-10-03 2010-04-08 Curna, Inc. Treatment of apolipoprotein-a1 related diseases by inhibition of natural antisense transcript to apolipoprotein-a1
SI3133160T1 (sl) 2008-10-24 2019-05-31 Sarepta Therapeutics, Inc. Sestavki, ki preskakujejo ekson za DMD
CA2741294C (en) * 2008-10-24 2018-04-24 Isis Pharmaceuticals, Inc. 5' and 2' bis-substituted nucleosides and oligomeric compounds prepared therefrom
RU2620970C2 (ru) 2008-12-04 2017-05-30 КьюРНА,Инк., Лечение связанных с эритропоэтином (еро) заболеваний путем ингибирования природного антисмыслового транскрипта к еро
MX358603B (es) 2008-12-04 2018-08-28 Curna Inc Tratamiento de enfermedades relacionadas con un gen supresor de tumor mediante inhibicion del transcrito antisentido natural para el gen.
EP2370581B1 (en) 2008-12-04 2016-08-03 CuRNA, Inc. Treatment of vascular endothelial growth factor (vegf) related diseases by inhibition of natural antisense transcript to vegf
EP3009150B1 (en) 2009-02-12 2019-11-13 CuRNA, Inc. Treatment of brain derived neurotrophic factor (bdnf) related diseases by inhibition of natural antisense transcript to bdnf
WO2010107733A2 (en) 2009-03-16 2010-09-23 Curna, Inc. Treatment of nuclear factor (erythroid-derived 2)-like 2 (nrf2) related diseases by inhibition of natural antisense transcript to nrf2
ES2627763T3 (es) 2009-03-17 2017-07-31 Curna, Inc. Tratamiento de enfermedades relacionadas con el homólogo tipo delta 1 (dlk1) por inhibición de transcrito antisentido natural a dlk1
EP2421971B1 (en) 2009-04-24 2016-07-06 BioMarin Technologies B.V. Oligonucleotide comprising an inosine for treating dmd
EP2427552B1 (en) 2009-05-06 2016-11-16 CuRNA, Inc. Treatment of tristetraproline (ttp) related diseases by inhibition of natural antisense transcript to ttp
US9155754B2 (en) 2009-05-06 2015-10-13 Curna, Inc. Treatment of ABCA1 gene related diseases by inhibition of a natural antisense transcript to ABCA1
CA2761248C (en) 2009-05-08 2023-03-14 Joseph Collard Treatment of dystrophin family related diseases by inhibition of natural antisense transcript to dmd family
DK2432881T3 (en) 2009-05-18 2018-02-26 Curna Inc TREATMENT OF REPROGRAMMING FACTOR-RELATED DISEASES BY INHIBITING NATURAL ANTISENSE TRANSCRIPTS TO A REPROGRAMMING FACTOR
WO2010135695A2 (en) 2009-05-22 2010-11-25 Curna, Inc. TREATMENT OF TRANSCRIPTION FACTOR E3 (TFE3) and INSULIN RECEPTOR SUBSTRATE 2 (IRS2) RELATED DISEASES BY INHIBITION OF NATURAL ANTISENSE TRANSCRIPT TO TFE3
ES2618576T3 (es) 2009-05-28 2017-06-21 Curna, Inc. Tratamiento de enfermedades relacionadas con un gen antivírico mediante la inhibición de una transcripción antisentido natural a un gen antivírico
KR101702689B1 (ko) 2009-06-16 2017-02-06 큐알엔에이, 인크. Pon1에 대한 천연 안티센스 전사체의 억제에 의한 파라옥소나제 1(pon1) 관련된 질환의 치료
KR101801404B1 (ko) 2009-06-16 2017-12-20 큐알엔에이, 인크. 콜라겐 유전자에 대한 천연 안티센스 전사체의 억제에 의한 콜라겐 유전자 관련된 질환의 치료
JP6073133B2 (ja) 2009-06-24 2017-02-01 クルナ・インコーポレーテッド 腫瘍壊死因子受容体2(tnfr2)に対する天然アンチセンス転写物の抑制によるtnfr2関連疾患の治療
ES2583691T3 (es) 2009-06-26 2016-09-21 Curna, Inc. Tratamiento de enfermedades relacionadas con un gen del síndrome de Down mediante la inhibición de una transcripción antisentido natural a un gen del síndrome de Down
WO2011005779A2 (en) 2009-07-07 2011-01-13 University Of Southern California Biomarkers for the early detection of autoimmune diseases
US20120252869A1 (en) 2009-07-24 2012-10-04 Opko Curna, Llc Treatment of sirtuin (sirt) related diseases by inhibition of natural antisense transcript to a sirtuin (sirt)
KR101802536B1 (ko) 2009-08-05 2017-11-28 큐알엔에이, 인크. 인슐린 유전자 (ins)에 대한 자연 안티센스 전사체의 저해에 의한 인슐린 유전자 (ins) 관련된 질환의 치료
WO2011019815A2 (en) 2009-08-11 2011-02-17 Curna, Inc. Treatment of adiponectin (adipoq) related diseases by inhibition of natural antisense transcript to an adiponectin (adipoq)
CN102482670B (zh) 2009-08-21 2018-06-15 库尔纳公司 通过抑制‘hsp70-相互作用蛋白的c末端’(chip)的天然反义转录物而治疗chip相关疾病
CA2771172C (en) 2009-08-25 2021-11-30 Opko Curna, Llc Treatment of 'iq motif containing gtpase activating protein' (iqgap) related diseases by inhibition of natural antisense transcript to iqgap
ES2664591T3 (es) 2009-09-25 2018-04-20 Curna, Inc. Tratamiento de enfermedades relacionadas con la filagrina (flg) mediante la modulación de la expresión y actividad del gen FLG
SMT201800579T1 (it) 2009-11-12 2019-01-11 Univ Western Australia Molecole antisenso e metodi per trattare patologie
NO2513310T3 (cg-RX-API-DMAC10.html) 2009-12-16 2018-03-31
EP2515947B1 (en) 2009-12-23 2021-10-06 CuRNA, Inc. Treatment of uncoupling protein 2 (ucp2) related diseases by inhibition of natural antisense transcript to ucp2
JP5934106B2 (ja) 2009-12-23 2016-06-15 カッパーアールエヌエー,インコーポレイテッド 肝細胞増殖因子(hgf)に対する天然アンチセンス転写物の阻害によるhgf関連性疾患の治療
EP2519634B1 (en) 2009-12-29 2016-06-01 CuRNA, Inc. TREATMENT OF TUMOR PROTEIN 63 (p63) RELATED DISEASES BY INHIBITION OF NATURAL ANTISENSE TRANSCRIPT TO p63
JP5993744B2 (ja) 2009-12-29 2016-09-14 カッパーアールエヌエー,インコーポレイテッド 核呼吸因子1(nrf1)に対する天然アンチセンス転写物の阻害による核呼吸因子1関連疾患の治療
US20120289583A1 (en) 2009-12-31 2012-11-15 Curna, Inc. Treatment of insulin receptor substrate 2 (irs2) related diseases by inhibition of natural antisense transcript to irs2 and transcription factor e3 (tfe3)
NO2521784T3 (cg-RX-API-DMAC10.html) 2010-01-04 2018-05-05
CA2786056C (en) 2010-01-06 2023-03-14 Curna, Inc. Treatment of pancreatic developmental gene related diseases by inhibition of natural antisense transcript to a pancreatic developmental gene
EP2524039B1 (en) 2010-01-11 2017-11-29 CuRNA, Inc. Treatment of sex hormone binding globulin (shbg) related diseases by inhibition of natural antisense transcript to shbg
WO2011091390A2 (en) 2010-01-25 2011-07-28 Opko Curna, Llc Treatment of rnase h1 related diseases by inhibition of natural antisense transcript to rnase h1
WO2011103528A2 (en) 2010-02-22 2011-08-25 Opko Curna Llc Treatment of pyrroline-5-carboxylate reductase 1 (pycr1) related diseases by inhibition of natural antisense transcript to pycr1
ES2557407T3 (es) 2010-03-01 2016-01-25 Tau Therapeutics Llc Procedimiento de imaginología de una enfermedad
WO2011112516A1 (en) * 2010-03-08 2011-09-15 Ico Therapeutics Inc. Treating and preventing hepatitis c virus infection using c-raf kinase antisense oligonucleotides
EP2553098B1 (en) 2010-04-02 2017-10-11 CuRNA, Inc. Treatment of colony-stimulating factor 3 (csf3) related diseases by inhibition of natural antisense transcript to csf3
RU2610661C2 (ru) 2010-04-09 2017-02-14 Курна, Инк. Лечение заболеваний, связанных с фактором роста фибробластов 21 (fgf21), путем ингибирования природного антисмыслового транскрипта к fgf21
KR101915115B1 (ko) 2010-05-03 2018-11-05 큐알엔에이, 인크. 시르투인 (sirt)에 대한 자연 안티센스 전사체의 저해에 의한 시르투인 (sirt) 관련된 질환의 치료
TWI586356B (zh) 2010-05-14 2017-06-11 可娜公司 藉由抑制par4天然反股轉錄本治療par4相關疾病
DK2576783T3 (en) 2010-05-26 2018-03-12 Curna Inc TREATMENT OF ATONAL HOMOLOGY 1- (ATOH1) RELATED DISEASES BY INHIBITION OF NATURAL ANTISENCE TRANSCRIPTS AT ATOH1
ES2664585T3 (es) 2010-05-26 2018-04-20 Curna, Inc. Tratamiento de enfermedades relacionadas con metionina sulfóxido reductasa (MSRA) mediante inhibición del transcrito antisentido natural a MSRA
JP6023705B2 (ja) 2010-06-23 2016-11-09 カッパーアールエヌエー,インコーポレイテッド ナトリウムチャネル、電位依存性、αサブユニット(SCNA)に対する天然アンチセンス転写物の阻害によるSCNA関連疾患の治療
CN103068982B (zh) 2010-07-14 2017-06-09 库尔纳公司 通过抑制盘状大同系物(dlg)的天然反义转录物而治疗dlg相关疾病
JP5986998B2 (ja) 2010-10-06 2016-09-06 カッパーアールエヌエー,インコーポレイテッド シアリダーゼ4(neu4)への天然アンチセンス転写物の阻害によるneu4関連疾患の治療
WO2012054723A2 (en) 2010-10-22 2012-04-26 Opko Curna Llc Treatment of alpha-l-iduronidase (idua) related diseases by inhibition of natural antisense transcript to idua
EP3260540A1 (en) 2010-11-12 2017-12-27 The General Hospital Corporation Polycomb-associated non-coding rnas
US9920317B2 (en) 2010-11-12 2018-03-20 The General Hospital Corporation Polycomb-associated non-coding RNAs
US10000752B2 (en) 2010-11-18 2018-06-19 Curna, Inc. Antagonat compositions and methods of use
ES2657590T3 (es) 2010-11-23 2018-03-06 Curna, Inc. Tratamiento de enfermedades relacionadas con nanog mediante inhibición del transcrito antisentido natural a nanog
EP2718439B1 (en) 2011-06-09 2017-08-09 CuRNA, Inc. Treatment of frataxin (fxn) related diseases by inhibition of natural antisense transcript to fxn
KR101991980B1 (ko) 2011-09-06 2019-06-21 큐알엔에이, 인크. 소형 분자로 전압-개폐된 나트륨 채널 (SCNxA)의 알파 아단위에 관련된 질환의 치료
HK1200120A1 (en) 2011-09-12 2015-07-31 Tau Therapeutics Llc Antagonists of products of the hs.459642 unigene cluster for the inhibition of proliferation, development or differentiation of stem cells including cancer stem cells
WO2013040429A1 (en) 2011-09-14 2013-03-21 Rana Therapeutics Inc. Multimeric oligonucleotide compounds
ES2907250T3 (es) 2012-01-27 2022-04-22 Biomarin Tech Bv Oligonucleótidos de modulación de ARN con características mejoradas para el tratamiento de la distrofia muscular de Duchenne y de Becker
CN110438125A (zh) 2012-03-15 2019-11-12 科纳公司 通过抑制脑源神经营养因子(bdnf)的天然反义转录物治疗bdnf相关疾病
WO2013173608A1 (en) 2012-05-16 2013-11-21 Rana Therapeutics, Inc. Compositions and methods for modulating mecp2 expression
WO2013173599A1 (en) 2012-05-16 2013-11-21 Rana Therapeutics, Inc. Compositions and methods for modulating hemoglobin gene family expression
KR20160074368A (ko) 2012-05-16 2016-06-28 라나 테라퓨틱스, 인크. Utrn 발현을 조절하기 위한 조성물 및 방법
US10174323B2 (en) 2012-05-16 2019-01-08 The General Hospital Corporation Compositions and methods for modulating ATP2A2 expression
US10837014B2 (en) 2012-05-16 2020-11-17 Translate Bio Ma, Inc. Compositions and methods for modulating SMN gene family expression
CN104540947A (zh) 2012-05-16 2015-04-22 Rana医疗有限公司 用于调节smn基因家族表达的组合物和方法
WO2014043544A1 (en) 2012-09-14 2014-03-20 Rana Therapeutics, Inc. Multimeric oligonucleotide compounds
US10024844B2 (en) 2012-12-20 2018-07-17 Hospital For Special Surgery Identification of an inhibitor of iRhom1 or an inhibitor of iRhom2
CN105378081B (zh) 2013-03-14 2019-06-14 萨勒普塔医疗公司 用于治疗肌营养不良的外显子跳跃组合物
HK1220154A1 (zh) 2013-03-15 2017-04-28 Sarepta Therapeutics, Inc. 改进的用於治疗肌营养不良的组合物
JP2016531570A (ja) 2013-08-16 2016-10-13 ラナ セラピューティクス インコーポレイテッド ユークロマチン領域を標的とするオリゴヌクレオチド
WO2015048531A1 (en) 2013-09-26 2015-04-02 Beth Israel Deaconess Medical Center, Inc. Inhibition of sgk1 in the treatment of heart conditions
US9486428B2 (en) 2014-03-20 2016-11-08 Cymabay Therapeutics, Inc. Treatment of intrahepatic cholestatic diseases
US10272058B2 (en) 2014-03-20 2019-04-30 Cymabay Therapeutics, Inc. Treatment of intrahepatic cholestatic diseases
HRP20192299T1 (hr) 2014-04-11 2020-03-06 Cymabay Therapeutics, Inc. Liječenje nafld i nash
GB201410693D0 (en) 2014-06-16 2014-07-30 Univ Southampton Splicing modulation
US9976143B2 (en) 2014-10-03 2018-05-22 Cold Spring Harbor Laboratory Targeted augmentation of nuclear gene output
EP3212824A4 (en) 2014-10-30 2018-08-08 The General Hospital Corporation Methods for modulating atrx-dependent gene repression
WO2016130943A1 (en) 2015-02-13 2016-08-18 Rana Therapeutics, Inc. Hybrid oligonucleotides and uses thereof
WO2016149455A2 (en) 2015-03-17 2016-09-22 The General Hospital Corporation The rna interactome of polycomb repressive complex 1 (prc1)
CN108603230A (zh) 2015-10-09 2018-09-28 南安普敦大学 基因表达的调节与蛋白质表达失调的筛选
FI3364993T3 (fi) 2015-10-22 2023-01-13 Menetelmiä angelmanin oireyhtymän hoitamiseksi
WO2017106377A1 (en) 2015-12-14 2017-06-22 Cold Spring Harbor Laboratory Antisense oligomers for treatment of autosomal dominant mental retardation-5 and dravet syndrome
US11096956B2 (en) 2015-12-14 2021-08-24 Stoke Therapeutics, Inc. Antisense oligomers and uses thereof
JOP20200228A1 (ar) 2015-12-21 2017-06-16 Novartis Ag تركيبات وطرق لخفض تعبير البروتين tau
JP7134178B2 (ja) 2017-02-15 2022-09-09 カビオン・インコーポレイテッド カルシウムチャネル阻害剤
JP7321097B2 (ja) 2017-04-26 2023-08-04 カビオン・インコーポレイテッド 記憶および認知を改善する、ならびに記憶および認知障害を処置するための方法
CN107460219B (zh) * 2017-07-24 2020-04-10 武汉大学 一种差异核酸酶切方法及其在LC-MS法检测mRNA内部修饰中的应用
GB2594767B (en) 2017-08-25 2022-06-22 Stoke Therapeutics Inc Antisense oligomers for treatment of conditions and diseases
CA3099280A1 (en) 2018-05-04 2019-11-07 Stoke Therapeutics, Inc. Methods and compositions for treatment of cholesteryl ester storage disease
WO2020072773A1 (en) 2018-10-03 2020-04-09 Cavion, Inc. Treating essential tremor using (r)-2-(4-isopropylphenyl)-n-(1-(5-(2,2,2-trifluoroethoxy)pyridin-2-yl)ethyl)acetamide
KR20230022409A (ko) 2020-05-11 2023-02-15 스톡 테라퓨틱스, 인크. 병태 및 질환의 치료를 위한 opa1 안티센스 올리고머

Family Cites Families (14)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US3687808A (en) 1969-08-14 1972-08-29 Univ Leland Stanford Junior Synthetic polynucleotides
EP0260032B1 (en) 1986-09-08 1994-01-26 Ajinomoto Co., Inc. Compounds for the cleavage at a specific position of RNA, oligomers employed for the formation of said compounds, and starting materials for the synthesis of said oligomers
DE68929304T2 (de) 1988-04-27 2002-04-25 Isis Pharmaceutical, Inc. Oligoribonukleotid-Derivate und Verwendung davon als Antiviral-Arzneimittel
US5256775A (en) 1989-06-05 1993-10-26 Gilead Sciences, Inc. Exonuclease-resistant oligonucleotides
US5149797A (en) 1990-02-15 1992-09-22 The Worcester Foundation For Experimental Biology Method of site-specific alteration of rna and production of encoded polypeptides
DK0549615T3 (da) 1990-08-13 2006-07-03 Isis Pharmaceuticals Inc Sukkermodificerede oligonukleotider der påviser og modulerer genekspression
AU672175B2 (en) 1991-06-14 1996-09-26 Isis Pharmaceuticals, Inc. Antisense oligonucleotide inhibition of the ras gene
DE4216134A1 (de) * 1991-06-20 1992-12-24 Europ Lab Molekularbiolog Synthetische katalytische oligonukleotidstrukturen
US5948898A (en) * 1992-03-16 1999-09-07 Isis Pharmaceuticals, Inc. Methoxyethoxy oligonucleotides for modulation of protein kinase C expression
ATE256143T1 (de) 1992-07-23 2003-12-15 Isis Pharmaceuticals Inc 2'-0-alkyl-nukleoside und-phosphoramidite, verfahren zu ihrer herstellung und ihre verwendungen
US5652355A (en) 1992-07-23 1997-07-29 Worcester Foundation For Experimental Biology Hybrid oligonucleotide phosphorothioates
TW244371B (cg-RX-API-DMAC10.html) 1992-07-23 1995-04-01 Tri Clover Inc
ATE177430T1 (de) * 1993-05-12 1999-03-15 Novartis Erfind Verwalt Gmbh Nukleoside und oligonukleotide mit 2'- ethergruppen
US5627053A (en) * 1994-03-29 1997-05-06 Ribozyme Pharmaceuticals, Inc. 2'deoxy-2'-alkylnucleotide containing nucleic acid

Also Published As

Publication number Publication date
AU1955297A (en) 1997-09-02
WO1997030067A1 (en) 1997-08-21
CA2246229A1 (en) 1997-08-21
CZ243498A3 (cs) 1999-09-15
NZ331217A (en) 2000-02-28
PL328563A1 (en) 1999-02-01
AU725262B2 (en) 2000-10-12
US6451991B1 (en) 2002-09-17
BR9707529A (pt) 2000-01-04
ATE267207T1 (de) 2004-06-15
EP0882061B1 (en) 2004-05-19
EP0882061A4 (en) 2000-12-13
NO983718D0 (no) 1998-08-13
EP0882061A1 (en) 1998-12-09
TR199801581T2 (xx) 1998-10-21
IL125759A0 (en) 1999-04-11
DE69729179D1 (de) 2004-06-24
ZA971208B (en) 1997-10-23
CN1214688A (zh) 1999-04-21
HUP9901118A2 (hu) 1999-07-28
DE69729179T2 (de) 2004-12-30
JP2000504725A (ja) 2000-04-18
CA2246229C (en) 2011-08-23
JP4293636B2 (ja) 2009-07-08
DK0882061T3 (da) 2004-09-27
HUP9901118A3 (en) 2000-06-28
SK109198A3 (en) 1999-06-11
KR19990082476A (ko) 1999-11-25
ES2221039T3 (es) 2004-12-16
NO983718L (no) 1998-10-13

Similar Documents

Publication Publication Date Title
CA2246229C (en) Sugar-modified gapped oligonucleotides
US5955589A (en) Gapped 2' modified oligonucleotides
US7015315B1 (en) Gapped oligonucleotides
US5700922A (en) PNA-DNA-PNA chimeric macromolecules
US5859221A (en) 2'-modified oligonucleotides
US5872232A (en) 2'-O-modified oligonucleotides
US7138517B2 (en) Sugar modified oligonucleotides
US6531584B1 (en) 2'modified oligonucleotides
US6172209B1 (en) Aminooxy-modified oligonucleotides and methods for making same
US6277603B1 (en) PNA-DNA-PNA chimeric macromolecules
US6146829A (en) Gapped 2' modified oligonucleotides
US5856455A (en) Gapped 2'-modified oligonucleotides
US5623065A (en) Gapped 2' modified oligonucleotides
KR100354610B1 (ko) 키메라 및 교호결합 올리고뉴클레오티드를 갖는 ras 유전자의 안티센스 저해
US20060270624A1 (en) Gapped 2' modified oligonucleotides
EP0813539A1 (en) Improved process for the synthesis of 2'-o-substituted pyrimidines and oligomeric compounds therefrom

Legal Events

Date Code Title Description
EEER Examination request
FZDE Discontinued

Effective date: 20130614