ATE267207T1 - Kohlenhydratmodifizierte luckenhafte oligonukleotide - Google Patents

Kohlenhydratmodifizierte luckenhafte oligonukleotide

Info

Publication number
ATE267207T1
ATE267207T1 AT97907581T AT97907581T ATE267207T1 AT E267207 T1 ATE267207 T1 AT E267207T1 AT 97907581 T AT97907581 T AT 97907581T AT 97907581 T AT97907581 T AT 97907581T AT E267207 T1 ATE267207 T1 AT E267207T1
Authority
AT
Austria
Prior art keywords
oligonucleotides
carbohydrate modified
modified latch
ooligonucleotide
erythro
Prior art date
Application number
AT97907581T
Other languages
English (en)
Inventor
Phillip D Cook
Brett P Monia
Karl-Heinz Altmann
Pierre Martin
Original Assignee
Isis Pharmaceuticals Inc
Novartis Ag
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 Isis Pharmaceuticals Inc, Novartis Ag filed Critical Isis Pharmaceuticals Inc
Application granted granted Critical
Publication of ATE267207T1 publication Critical patent/ATE267207T1/de

Links

Classifications

    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
    • C07H21/00—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
    • C07H21/04—Compounds 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
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
    • C07H21/00—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00—Antineoplastic agents

Landscapes

  • Chemical & Material Sciences (AREA)
  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Organic Chemistry (AREA)
  • Biochemistry (AREA)
  • Molecular Biology (AREA)
  • General Health & Medical Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Biotechnology (AREA)
  • Genetics & Genomics (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Saccharide Compounds (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
AT97907581T 1996-02-14 1997-02-07 Kohlenhydratmodifizierte luckenhafte oligonukleotide ATE267207T1 (de)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US1162096P 1996-02-14 1996-02-14
PCT/US1997/002043 WO1997030067A1 (en) 1996-02-14 1997-02-07 Sugar-modified gapped oligonucleotides

Publications (1)

Publication Number Publication Date
ATE267207T1 true ATE267207T1 (de) 2004-06-15

Family

ID=21751248

Family Applications (1)

Application Number Title Priority Date Filing Date
AT97907581T ATE267207T1 (de) 1996-02-14 1997-02-07 Kohlenhydratmodifizierte luckenhafte oligonukleotide

Country Status (22)

Country Link
US (1) US6451991B1 (de)
EP (1) EP0882061B1 (de)
JP (1) JP4293636B2 (de)
KR (1) KR19990082476A (de)
CN (1) CN1214688A (de)
AT (1) ATE267207T1 (de)
AU (1) AU725262B2 (de)
BR (1) BR9707529A (de)
CA (2) CA2246229C (de)
CZ (1) CZ243498A3 (de)
DE (1) DE69729179T2 (de)
DK (1) DK0882061T3 (de)
ES (1) ES2221039T3 (de)
HU (1) HUP9901118A3 (de)
IL (1) IL125759A0 (de)
NO (1) NO983718L (de)
NZ (1) NZ331217A (de)
PL (1) PL328563A1 (de)
SK (1) SK109198A3 (de)
TR (1) TR199801581T2 (de)
WO (1) WO1997030067A1 (de)
ZA (1) ZA971208B (de)

Families Citing this family (121)

* 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
IL144975A0 (en) 1999-02-26 2002-06-30 Univ British Columbia A composition containing an antisense oligonucleotide
US7569551B2 (en) 2000-02-25 2009-08-04 The University Of British Columbia Chemo- and radiation-sensitization of cancer by antisense TRPM-2 oligodeoxynucleotides
DK2796553T3 (da) 2000-03-30 2019-09-30 Whitehead Inst Biomedical Res Rna-sekvensspecifikke formidlere af rna-interferens
EP1191097A1 (de) 2000-09-21 2002-03-27 Leids Universitair Medisch Centrum Induktion von "exon-skipping" in eukaryotischen Zellen
DK1407044T4 (en) 2000-12-01 2017-12-04 Max-Planck-Gesellschaft Zur Förderung Der Wss E V RNA interference mediating small RNA molecules
KR101265180B1 (ko) * 2002-01-17 2013-05-29 더 유니버시티 오브 브리티쉬 콜롬비아 아이지에프비피-2 및 아이지에프비피-5를 억제하는 양특이성 안티센스 올리고뉴클레오티드, 이를 이용하여 약학적 조성물을 제조하는 방법 및 그 약학적 조성물
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
CA2539727C (en) * 2003-10-01 2016-11-01 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
WO2005094899A1 (en) 2004-04-02 2005-10-13 The University Of British Columbia Clusterin antisense therapy for treatment of cancer
PT1766010E (pt) 2004-06-28 2011-05-25 Univ Western Australia Oligonucle?tidos antisense para a indu??o de skipping de ex?es e seus m?todos de utiliza??o
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
DK1814595T3 (da) * 2004-11-23 2014-03-31 Univ British Columbia Behandling af cancer med en kombination af et middel, som forstyrrer EGF-signalvejen og et oligonucleotid, som reducerer clusterinniveauerne
CN103301475B (zh) 2005-12-28 2016-08-03 斯克里普斯研究所 药物组合物和表达载体以及调节基因表达的方法和核酸分子的应用
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 (de) 2006-05-19 2007-11-21 Academisch Ziekenhuis Leiden Mittel und Verfahren zur Induktion von Exon-skipping
WO2007139943A2 (en) * 2006-05-26 2007-12-06 Ico Therapeutics Inc. Therapeutic drug combinations and delivery systems comprising c-raf kinase antisense polynucleotides for treating ocular diseases and disorders
CA2660523C (en) 2006-08-11 2019-03-19 Prosensa Technologies B.V. Methods and means for treating dna repeat instability associated genetic disorders
US8809514B2 (en) 2006-09-22 2014-08-19 Ge Healthcare Dharmacon, Inc. Tripartite oligonucleotide complexes and methods for gene silencing by RNA interference
CA2693048C (en) 2007-07-12 2016-10-18 Prosensa Technologies B.V. Molecules for targeting compounds to various selected organs or tissues
USRE48468E1 (en) 2007-10-26 2021-03-16 Biomarin Technologies B.V. Means and methods for counteracting muscle disorders
CN105641700B (zh) 2007-10-26 2021-01-01 莱顿教学医院 对抗肌肉病症的方式和方法
NZ587178A (en) 2008-02-08 2011-11-25 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 (de) 2008-05-14 2009-11-18 Prosensa Technologies B.V. Verfahren für effizientes Exon (44)-Skipping bei Duchenne-Muskeldystrophie und entsprechende Mittel
KR101770435B1 (ko) 2008-10-03 2017-09-05 큐알엔에이, 인크. 자연 안티센스 전사체가 아포리포단백질-ａ1로 억제에 의해 아포리포단백-ａ1 관련된 질환의 치료
WO2010048552A2 (en) * 2008-10-24 2010-04-29 Isis Pharmaceuticals, Inc. Methods of using oligomeric compounds comprising 2'-substituted nucleosides
BRPI0920276B1 (pt) 2008-10-24 2021-06-08 Sarepta Therapeutics, Inc oligonucleotídeo antissenso, composição que compreende o mesmo e uso do dito oligonucleotídeo para tratar distrofia muscular
EP2370582B1 (de) 2008-12-04 2017-05-10 CuRNA, Inc. Behandlung von tumorsuppressorgen-bedingten erkrankungen mittels hemmung des natürlichen antisense-transkripts gegen das gen
EP2370579B1 (de) 2008-12-04 2017-03-29 CuRNA, Inc. Behandlung von erythropoetin (epo)-bedingten erkrankungen mittels hemmung des natürlichen antisense-transkripts gegen epo
CN102341498B (zh) 2008-12-04 2017-12-19 库尔纳公司 通过抑制血管内皮生长因子(vegf)的天然反义转录子治疗vegf相关的疾病
ES2560107T3 (es) 2009-02-12 2016-02-17 Curna, Inc. Tratamiento de enfermedades relacionadas con el factor neurotrófico derivado de cerebro (BDNF) por inhibición de transcrito antisentido natural para BDNF
CA2755409C (en) 2009-03-16 2019-04-30 Joseph Collard Treatment of nuclear factor (erythroid-derived 2)-like 2 (nrf2) related diseases by inhibition of natural antisense transcript to nrf2
EP2408920B1 (de) 2009-03-17 2017-03-08 CuRNA, Inc. Behandlung von erkrankungen im zusammenhang mit dem delta-like-1-homolog (dlk1) mittels hemmung des natürlichen antisense-transkripts gegen dlk1
WO2010123369A1 (en) 2009-04-24 2010-10-28 Prosensa Technologies B.V. Oligonucleotide comprising an inosine for treating dmd
ES2609655T3 (es) 2009-05-06 2017-04-21 Curna, Inc. Tratamiento de enfermedades relacionadas con tristetraprolina (TTP) mediante inhibición de transcrito antisentido natural para 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
CN102459597B (zh) 2009-05-08 2020-06-30 库尔纳公司 通过针对dmd家族的天然反义转录物的抑制治疗肌营养蛋白家族相关疾病
CA2762369C (en) 2009-05-18 2021-12-28 Joseph Collard Treatment of reprogramming factor related diseases by inhibition of natural antisense transcript to a reprogramming factor
CN102549158B (zh) 2009-05-22 2017-09-26 库尔纳公司 通过抑制针对转录因子e3(tfe3)的天然反义转录物来治疗tfe3和胰岛素受体底物蛋白2(irs2)相关的疾病
JP5960049B2 (ja) 2009-05-28 2016-08-02 クルナ・インコーポレーテッド 抗ウイルス遺伝子に対する天然アンチセンス転写物の抑制による抗ウイルス遺伝子関連疾患の治療
US8951981B2 (en) 2009-06-16 2015-02-10 Curna, Inc. Treatment of paraoxonase 1 (PON1) related diseases by inhibition of natural antisense transcript to PON1
EP2443237B1 (de) 2009-06-16 2017-02-22 CuRNA, Inc. Behandlung von krankheiten im zusammenhang mit dem kollagengen durch hemmung des natürlichen antisense-transkripts zu einem kollagengens
WO2010151671A2 (en) 2009-06-24 2010-12-29 Curna, Inc. Treatment of tumor necrosis factor receptor 2 (tnfr2) related diseases by inhibition of natural antisense transcript to tnfr2
WO2010151674A2 (en) 2009-06-26 2010-12-29 Curna, Inc. Treatment of down syndrome gene related diseases by inhibition of natural antisense transcript to a down syndrome gene
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)
JP6128848B2 (ja) 2009-08-05 2017-05-17 クルナ・インコーポレーテッド インスリン遺伝子（ｉｎｓ）に対する天然アンチセンス転写物の抑制によるインスリン遺伝子（ｉｎｓ）関連疾患の治療
CN102625841A (zh) 2009-08-11 2012-08-01 欧科库尔纳有限责任公司 通过抑制脂连蛋白(adipoq)的天然反义转录物治疗脂连蛋白(adipoq)相关疾病
US8791087B2 (en) 2009-08-21 2014-07-29 Curna, Inc. Treatment of ‘C terminus of HSP70-interacting protein’ (CHIP)related diseases by inhibition of natural antisense transcript to CHIP
WO2011031482A2 (en) 2009-08-25 2011-03-17 Curna, Inc. Treatment of 'iq motif containing gtpase activating protein' (iqgap) related diseases by inhibition of natural antisense transcript to iqgap
CN102791861B (zh) 2009-09-25 2018-08-07 库尔纳公司 通过调节聚丝蛋白(flg)的表达和活性而治疗flg相关疾病
RS58079B1 (sr) 2009-11-12 2019-02-28 Univ Western Australia Antisens molekuli i postupci za tretman patologija
EP2513310B1 (de) 2009-12-16 2017-11-01 CuRNA, Inc. Behandlung von erkrankungen im zusammenhang mit dem membranengebundenen transkriptionsfaktor-peptidase-ort 1 (mbtps1) mittels hemmung des natürlichen antisense-transkripts gegen mbtps1
JP6031356B2 (ja) 2009-12-23 2016-11-24 カッパーアールエヌエー，インコーポレイテッド Ｕｃｐ２に対する天然アンチセンス転写産物の阻害による脱共役タンパク質２（ｕｃｐ２）関連疾患の治療
DK2516648T3 (en) 2009-12-23 2018-02-12 Curna Inc TREATMENT OF HEPATOCYTE GROWTH FACTOR (HGF) RELATED DISEASES BY INHIBITION OF NATURAL ANTISENSE TRANSCRIPT AGAINST HGF
KR101838305B1 (ko) 2009-12-29 2018-03-13 큐알엔에이, 인크. ＮＲＦ1(Ｎｕｃｌｅａｒ Ｒｅｓｐｉｒａｔｏｒｙ Ｆａｃｔｏｒ 1)에 대한 천연 안티센스 전사체의 억제에 의한 핵 호흡 인자 1 관련된 질환의 치료
KR101853508B1 (ko) 2009-12-29 2018-06-20 큐알엔에이, 인크. 종양 단백질 63 (ｐ63)에 대한 천연 안티센스 전사체의 억제에 의한 ｐ63에 관련된 질환의 치료
KR101853511B1 (ko) 2009-12-31 2018-06-20 큐알엔에이, 인크. 인슐린 수용체 기질 2 및 전사 인자 ｅ3(ｔｆｅ3)에 대한 천연 안티센스 전사체의 억제에 의한 ｉｒｓ2 관련된 질환의 치료
EP2521784B1 (de) 2010-01-04 2017-12-06 CuRNA, Inc. Behandlung von erkrankungen im zusammenhang mit dem interferonregulierungsfaktor 8 (irf8) mittels hemmung des natürlichen antisense-transkripts gegen irf8
KR101853509B1 (ko) 2010-01-06 2018-04-30 큐알엔에이, 인크. 췌장 발달 유전자에 대한 천연 안티센스 전사체의 억제에 의한 췌장 발달 유전자와 관련된 질환의 치료
JP6027893B2 (ja) 2010-01-11 2016-11-16 カッパーアールエヌエー，インコーポレイテッド 性ホルモン結合グロブリン（ｓｈｂｇ）に対する天然アンチセンス転写物の阻害による性ホルモン結合グロブリン（ｓｈｂｇ）関連疾患の治療
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
RU2608496C2 (ru) 2010-02-22 2017-01-18 Курна, Инк. Лечение заболеваний, связанных с пирролин-5 карбоксилатредуктазой 1(pycr1), путем ингибирования природного антисмыслового транскрипта к pycr1
KR101821343B1 (ko) 2010-03-01 2018-01-23 타우 쎄라퓨틱스 엘엘씨 암 진단 및 영상화
WO2011112516A1 (en) * 2010-03-08 2011-09-15 Ico Therapeutics Inc. Treating and preventing hepatitis c virus infection using c-raf kinase antisense oligonucleotides
JP5973419B2 (ja) 2010-04-02 2016-08-23 カッパーアールエヌエー，インコーポレイテッド コロニー刺激因子３（ｃｓｆ３）に対する天然アンチセンス転写物の阻害によるｃｓｆ３関連疾患の治療
KR101900962B1 (ko) 2010-04-09 2018-09-20 큐알엔에이, 인크. 섬유아세포 성장 인자 21 (ｆｇｆ21)에 대한 자연 안티센스 전사체의 저해에 의한 섬유아세포 성장 인자 21 (ｆｇｆ21) 관련된 질환의 치료
JP2013525483A (ja) 2010-05-03 2013-06-20 カッパーアールエヌエー，インコーポレイテッド サーチュイン（ｓｉｒｔ）に対する天然アンチセンス転写物の阻害によるサーチュイン（ｓｉｒｔ）関連疾患の治療
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
WO2011150007A2 (en) 2010-05-26 2011-12-01 Opko Curna Llc Treatment of methionine sulfoxide reductase a (msra) related diseases by inhibition of natural antisense transcript to msra
KR101857090B1 (ko) 2010-05-26 2018-06-26 큐알엔에이, 인크. 무조(無調)의 동소체 1 (atoh1)에 대한 자연 안티센스 전사체의 저해에 의한 atoh1 관련된 질환의 치료
WO2011163499A2 (en) 2010-06-23 2011-12-29 Opko Curna, Llc Treatment of sodium channel, voltage-gated, alpha subunit (scna) related diseases by inhibition of natural antisense transcript to scna
ES2663598T3 (es) 2010-07-14 2018-04-16 Curna, Inc. Tratamiento de enfermedades relacionadas con el homólogo de discos grandes (dlg) mediante la inhibición del transcrito antisentido natural a dlg
CA2813901C (en) 2010-10-06 2019-11-12 Curna, Inc. Treatment of sialidase 4 (neu4) related diseases by inhibition of natural antisense transcript to neu4
EP2630241B1 (de) 2010-10-22 2018-10-17 CuRNA, Inc. Behandlung von alpha-l-iduronidase (idua)-bedingten erkrankungen mittels hemmung des natürlichen antisense-transkripts gegen idua
EP2638163B1 (de) 2010-11-12 2017-05-17 The General Hospital Corporation Polycomb-assoziierte nicht-kodierende rnas
US9920317B2 (en) 2010-11-12 2018-03-20 The General Hospital Corporation Polycomb-associated non-coding RNAs
WO2012068340A2 (en) 2010-11-18 2012-05-24 Opko Curna Llc Antagonat compositions and methods of use
US8987225B2 (en) 2010-11-23 2015-03-24 Curna, Inc. Treatment of NANOG related diseases by inhibition of natural antisense transcript to NANOG
JP6188686B2 (ja) 2011-06-09 2017-08-30 カッパーアールエヌエー，インコーポレイテッド フラタキシン（ｆｘｎ）への天然アンチセンス転写物の阻害によるｆｘｎ関連疾患の治療
BR112014005234A2 (pt) 2011-09-06 2017-04-11 Curna Inc tratamento de doenças relacionadas com subunididades alfa dos canais de sódio dependentes de voltagem (scnxa) com pequenas moléculas
JP2014526475A (ja) 2011-09-12 2014-10-06 タウ セラピューティクス エルエルシー がん幹細胞を含む幹細胞の増殖、進展、または分化の阻害のためのＨｓ．４５９６４２ユニジーンクラスター産物のアンタゴニスト
DK2756080T3 (da) 2011-09-14 2019-05-20 Translate Bio Ma Inc Multimeriske oligonukleotidforbindelser
CN104203289B (zh) 2012-01-27 2020-11-03 比奥马林技术公司 用于治疗杜兴型肌营养不良症和贝克型肌营养不良症的具有改善特性的rna调节性寡核苷酸
ES2694592T3 (es) 2012-03-15 2018-12-21 Curna, Inc. Tratamiento de enfermedades relacionadas con el factor neurotrófico derivado del cerebro (BDNF) por inhibición del transcrito antisentido natural de BDNF
WO2013173608A1 (en) 2012-05-16 2013-11-21 Rana Therapeutics, Inc. Compositions and methods for modulating mecp2 expression
EA201492123A1 (ru) 2012-05-16 2015-10-30 Рана Терапьютикс, Инк. Композиции и способы для модулирования экспрессии семейства генов smn
EA201492120A1 (ru) 2012-05-16 2015-10-30 Рана Терапьютикс, Инк. Композиции и способы для модулирования экспрессии atp2a2
US10837014B2 (en) 2012-05-16 2020-11-17 Translate Bio Ma, Inc. Compositions and methods for modulating SMN gene family expression
KR20160074368A (ko) 2012-05-16 2016-06-28 라나 테라퓨틱스, 인크. Utrn 발현을 조절하기 위한 조성물 및 방법
CN104583399A (zh) 2012-05-16 2015-04-29 Rana医疗有限公司 用于调节血红蛋白基因家族表达的组合物和方法
DK2895200T3 (da) 2012-09-14 2020-02-10 Translate Bio Ma Inc Multimeriske oligonukleotidforbindelser
US10024844B2 (en) 2012-12-20 2018-07-17 Hospital For Special Surgery Identification of an inhibitor of iRhom1 or an inhibitor of iRhom2
ES2762881T3 (es) 2013-03-14 2020-05-26 Sarepta Therapeutics Inc Composiciones de salto de exón para el tratamiento de la distrofia muscular
BR112015022998A2 (pt) 2013-03-15 2017-11-14 Sarepta Therapeutics Inc composições melhoradas para o tratamento de distrofia muscular
CA2921459A1 (en) 2013-08-16 2015-02-19 Rana Therapeutics, Inc. Oligonucleotides targeting euchromatin regions of genes
PT3049085T (pt) 2013-09-26 2021-10-01 Beth Israel Deaconess Medical Ct Inc Inibidores de sgk1 no tratamento da síndrome do qt longo
KR102000332B1 (ko) 2014-03-20 2019-07-15 사이머베이 쎄라퓨틱스, 인코퍼레이티드 간내 담즙정체성 질환의 치료
US10272058B2 (en) 2014-03-20 2019-04-30 Cymabay Therapeutics, Inc. Treatment of intrahepatic cholestatic diseases
JP6865038B2 (ja) 2014-04-11 2021-04-28 サイマベイ・セラピューティクス・インコーポレイテッドＣｙｍａＢａｙ Ｔｈｅｒａｐｅｕｔｉｃｓ，Ｉｎｃ． Ｎａｆｌｄおよびｎａｓｈの治療
GB201410693D0 (en) 2014-06-16 2014-07-30 Univ Southampton Splicing modulation
CA2963288A1 (en) 2014-10-03 2016-04-07 Cold Spring Harbor Laboratory Targeted augmentation of nuclear gene output
US10858650B2 (en) 2014-10-30 2020-12-08 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)
JP6923517B2 (ja) 2015-10-09 2021-08-18 ユニバーシティ・オブ・サザンプトン 遺伝子発現の調節及び脱制御されたタンパク質発現のスクリーニング
FI3364993T3 (fi) 2015-10-22 2023-01-13 Menetelmiä angelmanin oireyhtymän hoitamiseksi
CN116059236A (zh) 2015-12-14 2023-05-05 冷泉港实验室 用于治疗常染色体显性精神发育迟滞5型和Dravet综合征的反义寡聚体
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
US11130750B2 (en) 2017-02-15 2021-09-28 Cavion, Inc. Calcium channel inhibitors
CA3061720A1 (en) 2017-04-26 2018-11-01 Cavion, Inc. Methods for improving memory and cognition and for treating memory and cognitive disorders
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
EP3673080B1 (de) 2017-08-25 2023-10-18 Stoke Therapeutics, Inc. Antisense-oligomere zur behandlung von leiden und krankheiten
IL300798A (en) 2018-10-03 2023-04-01 Cavion Inc Treatment of bone tremor using (R)-2-(4-isopropylphenyl)-N-(1-(5-(2,2,2-trifluoroethoxy)pyridin-2-yl)ethyl)acetamide
CA3173647A1 (en) 2020-05-11 2021-11-18 Isabel AZNAREZ Opa1 antisense oligomers for treatment of conditions and diseases

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 (de) 1986-09-08 1994-01-26 Ajinomoto Co., Inc. Verbindungen zur Spaltung von RNS an eine spezifische Position, Oligomere, verwendet bei der Herstellung dieser Verbindungen und Ausgangsprodukte für die Synthese dieser Oligomere
DE68929306T2 (de) 1988-04-27 2002-05-02 Isis Pharmaceutical, Inc. H-Phosphonat Ribonucleotid-Derivate
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
JPH0813274B2 (ja) 1990-08-13 1996-02-14 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 遺伝子発現を検出及び変調する糖修飾されたオリゴヌクレオチド
DK0590082T3 (da) 1991-06-14 1999-11-08 Isis Pharmaceuticals Inc Antisense-oligonukleotid-inhibering af ras-genet
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
TW244371B (de) 1992-07-23 1995-04-01 Tri Clover Inc
US5652355A (en) 1992-07-23 1997-07-29 Worcester Foundation For Experimental Biology Hybrid oligonucleotide phosphorothioates
EP0651759B1 (de) 1992-07-23 2003-12-10 Isis Pharmaceuticals, Inc. 2'-0-alkyl-nukleoside und-phosphoramidite, verfahren zu ihrer herstellung und ihre verwendungen
ES2128535T3 (es) * 1993-05-12 1999-05-16 Novartis Ag Nucleosidos y oligonucleotidos con grupos 2'-eter.
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
CA2246229A1 (en) 1997-08-21
HUP9901118A2 (hu) 1999-07-28
HUP9901118A3 (en) 2000-06-28
CZ243498A3 (cs) 1999-09-15
IL125759A0 (en) 1999-04-11
DE69729179D1 (de) 2004-06-24
KR19990082476A (ko) 1999-11-25
BR9707529A (pt) 2000-01-04
US6451991B1 (en) 2002-09-17
ZA971208B (en) 1997-10-23
JP2000504725A (ja) 2000-04-18
DE69729179T2 (de) 2004-12-30
EP0882061A4 (de) 2000-12-13
NO983718L (no) 1998-10-13
DK0882061T3 (da) 2004-09-27
AU1955297A (en) 1997-09-02
NO983718D0 (no) 1998-08-13
CN1214688A (zh) 1999-04-21
TR199801581T2 (xx) 1998-10-21
EP0882061A1 (de) 1998-12-09
SK109198A3 (en) 1999-06-11
WO1997030067A1 (en) 1997-08-21
PL328563A1 (en) 1999-02-01
CA2246229C (en) 2011-08-23
ES2221039T3 (es) 2004-12-16
AU725262B2 (en) 2000-10-12
JP4293636B2 (ja) 2009-07-08
EP0882061B1 (de) 2004-05-19
CA2749312A1 (en) 1997-08-21
NZ331217A (en) 2000-02-28

Similar Documents

Publication Publication Date Title
ATE267207T1 (de) Kohlenhydratmodifizierte luckenhafte oligonukleotide
DE69233599D1 (de) Unterbrochene 2'-modifizierte Oligonukleotide
ATE219095T1 (de) Pns-dns-pns-chimäre makromoleküle
ATE419262T1 (de) 2'-o-acetamido-modifizierte monomere und oligomere
FR2703693B1 (fr) Procédé rapide de détermination d'une séquence d'ADN et application au séquençage et au diagnostic.
ATE292141T1 (de) Oligoribonukleotide und ribonucleasen zur spaltung von rns
DK1300466T3 (da) En kaskade nukleinsyre amplifikationsreaktion
DE69732535D1 (de) Oligonucleotidzusammenstellung und verfahren zur regulierung der protein b7 expression
ES2101867T3 (es) Aplicaciones diagnosticas de la formacion de lazos d dobles.
DE69403642D1 (de) 7-deazapurin modifizierte oligonukleotide
WO1998019167A3 (en) Cell stress regulated human mhc class i gene
GB8929293D0 (en) C.difficile dna probes
GB9312315D0 (en) Leukocyte adhesion assay
TR199902434T2 (xx) Bakteriyel plazmidler.
ES2056458T3 (es) Procedimiento de estabilizacion de la hibridacion de secuencias polinucleotidicas complementarias.
FR2638090B1 (fr) Sequences de nucleotides exprimant l'adenyl cyclase de b. anthracis, proteines ayant l'activite de cette adenyl cyclase et applications biologiques
ATE108834T1 (de) Spezifische nukleinsäurefragmente des menschlichen villingens, ihre anwendung für diagnostische zwecke.

Legal Events

Date Code Title Description
UEP Publication of translation of european patent specification

Ref document number: 0882061

Country of ref document: EP