DE69729179T2 - Lückenhaft Zucker-modifizierte Oligonukleotide - Google Patents

Lückenhaft Zucker-modifizierte Oligonukleotide Download PDF

Info

Publication number
DE69729179T2
DE69729179T2 DE69729179T DE69729179T DE69729179T2 DE 69729179 T2 DE69729179 T2 DE 69729179T2 DE 69729179 T DE69729179 T DE 69729179T DE 69729179 T DE69729179 T DE 69729179T DE 69729179 T2 DE69729179 T2 DE 69729179T2
Authority
DE
Germany
Prior art keywords
oligonucleotides
oligonucleotide
sequence
sub
ras
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.)
Expired - Lifetime
Application number
DE69729179T
Other languages
German (de)
English (en)
Other versions
DE69729179D1 (de
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 DE69729179D1 publication Critical patent/DE69729179D1/de
Application granted granted Critical
Publication of DE69729179T2 publication Critical patent/DE69729179T2/de
Anticipated expiration legal-status Critical
Expired - Lifetime 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)
DE69729179T 1996-02-14 1997-02-07 Lückenhaft Zucker-modifizierte Oligonukleotide Expired - Lifetime DE69729179T2 (de)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US1162096P 1996-02-14 1996-02-14
US11620P 1996-02-14
PCT/US1997/002043 WO1997030067A1 (en) 1996-02-14 1997-02-07 Sugar-modified gapped oligonucleotides

Publications (2)

Publication Number Publication Date
DE69729179D1 DE69729179D1 (de) 2004-06-24
DE69729179T2 true DE69729179T2 (de) 2004-12-30

Family

ID=21751248

Family Applications (1)

Application Number Title Priority Date Filing Date
DE69729179T Expired - Lifetime DE69729179T2 (de) 1996-02-14 1997-02-07 Lückenhaft Zucker-modifizierte Oligonukleotide

Country Status (22)

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

Families Citing this family (123)

* 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
JP4785252B2 (ja) 1999-02-26 2011-10-05 ザ・ユニバーシティ・オブ・ブリティッシュ・コロンビア Trpm−2アンチセンス療法
US7569551B2 (en) 2000-02-25 2009-08-04 The University Of British Columbia Chemo- and radiation-sensitization of cancer by antisense TRPM-2 oligodeoxynucleotides
ES2336887T5 (es) 2000-03-30 2019-03-06 Whitehead Inst Biomedical Res Mediadores de interferencia por ARN específicos de secuencias de ARN
EP1191097A1 (en) 2000-09-21 2002-03-27 Leids Universitair Medisch Centrum Induction of exon skipping in eukaryotic cells
CA2429814C (en) 2000-12-01 2014-02-18 Thomas Tuschl Rna interference mediating small rna molecules
WO2003062421A1 (en) * 2002-01-17 2003-07-31 The University Of British Columbia Bispecific antisense olignucleotides that inhibit igfbp-2 and igfbp-5 and methods of using same
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
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
EP2933332A1 (en) 2004-06-28 2015-10-21 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
US7935811B2 (en) 2004-11-22 2011-05-03 Dharmacon, Inc. Apparatus and system having dry gene silencing compositions
US7923207B2 (en) 2004-11-22 2011-04-12 Dharmacon, Inc. Apparatus and system having dry gene silencing pools
CA2584646C (en) * 2004-11-23 2015-11-03 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
JP5713377B2 (ja) 2005-12-28 2015-05-07 ザ スクリプス リサーチ インスティテュート 薬物標的としての天然アンチセンスおよび非コードrna転写物
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
SI2049664T1 (sl) 2006-08-11 2012-04-30 Prosensa Technologies Bv Enojna veriga oligonukleotidov, komplementarna repetitivnim elementom, za zdravljenje genetskih bolezni, povezanih z nestabilnostjo dnk ponovitev
AU2007299705B2 (en) 2006-09-22 2012-09-06 Dharmacon, Inc. Duplex oligonucleotide complexes and methods for gene silencing by RNA interference
WO2009008727A2 (en) 2007-07-12 2009-01-15 Prosensa Technologies B.V. Molecules for targeting compounds to various selected organs or tissues
ES2639852T3 (es) 2007-10-26 2017-10-30 Academisch Ziekenhuis Leiden Medios y métodos para contrarrestar los trastornos musculares
USRE48468E1 (en) 2007-10-26 2021-03-16 Biomarin Technologies B.V. Means and methods for counteracting muscle disorders
WO2009099326A1 (en) 2008-02-08 2009-08-13 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
CN102239260B (zh) 2008-10-03 2017-04-12 库尔纳公司 通过抑制针对载脂蛋白‑a1的天然反义转录物治疗载脂蛋白‑a1相关疾病
BR122020021379B1 (pt) 2008-10-24 2021-05-11 Sarepta Therapeutics, Inc. oligômero morfolino fosforodiamidato, composição que compreende o mesmo e uso do dito oligômero para tratar distrofia muscular
US20120059045A1 (en) * 2008-10-24 2012-03-08 Isis Pharmaceuticals, Inc. Methods of using oligomeric compounds comprising 2'-substituted nucleosides
ES2629630T3 (es) 2008-12-04 2017-08-11 Curna, Inc. Tratamiento de enfermedades relacionadas con eritropoyetina (EPO) mediante inhibición del transcrito antisentido natural a EPO
US20110294870A1 (en) 2008-12-04 2011-12-01 Opko Curna, Llc Treatment of tumor suppressor gene related diseases by inhibition of natural antisense transcript to the gene
ES2600781T3 (es) 2008-12-04 2017-02-10 Curna, Inc. Tratamiento para enfermedades relacionadas con el factor de crecimiento del endotelio vascular (vegf) mediante la inhibición de transcritos antisentido naturales de vegf
ES2762610T3 (es) 2009-02-12 2020-05-25 Curna Inc Tratamiento de enfermedades relacionadas con el factor neurotrófico derivado de cerebro (BDNF) por inhibición de transcrito antisentido natural para BDNF
CN102482677B (zh) 2009-03-16 2017-10-17 库尔纳公司 通过抑制nrf2的天然反义转录物治疗核因子(红细胞衍生2)‑样2(nrf2)相关疾病
WO2010107740A2 (en) 2009-03-17 2010-09-23 Curna, Inc. Treatment of delta-like 1 homolog (dlk1) related diseases by inhibition of natural antisense transcript to dlk1
EP2421971B1 (en) 2009-04-24 2016-07-06 BioMarin Technologies B.V. Oligonucleotide comprising an inosine for treating dmd
CN103223177B (zh) 2009-05-06 2016-08-10 库尔纳公司 通过针对脂质转运和代谢基因的天然反义转录物的抑制治疗脂质转运和代谢基因相关疾病
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
JP5931720B2 (ja) 2009-05-08 2016-06-08 クルナ・インコーポレーテッド Dmdファミリーに対する天然アンチセンス転写物の抑制によるジストロフィンファミリー関連疾患の治療
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
CA2762987A1 (en) 2009-05-22 2010-11-25 Joseph Collard Treatment of transcription factor e3 (tfe3) and insulin receptor substrate 2 (irs2) related diseases by inhibition of natural antisense transcript to tfe3
KR101704988B1 (ko) 2009-05-28 2017-02-08 큐알엔에이, 인크. 항바이러스 유전자에 대한 천연 안티센스 전사체의 억제에 의한 항바이러스 유전자 관련된 질환의 치료
WO2010148050A2 (en) 2009-06-16 2010-12-23 Curna, Inc. Treatment of collagen gene related diseases by inhibition of natural antisense transcript to a collagen gene
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
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
EP2446037B1 (en) 2009-06-26 2016-04-20 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
CN102712925B (zh) 2009-07-24 2017-10-27 库尔纳公司 通过抑制sirtuin(sirt)的天然反义转录物来治疗sirtuin(sirt)相关性疾病
CN102762731B (zh) 2009-08-05 2018-06-22 库尔纳公司 通过抑制针对胰岛素基因(ins)的天然反义转录物来治疗胰岛素基因(ins)相关的疾病
CN104313027B (zh) 2009-08-11 2018-11-20 库尔纳公司 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
KR101805213B1 (ko) 2009-08-21 2017-12-06 큐알엔에이, 인크. Chip에 대한 천연 안티센스 전사체의 억제에 의한 “hsp70-상호작용 단백질(chip)의 c-말단” 관련된 질환의 치료
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
US20120295952A1 (en) 2009-09-25 2012-11-22 Curna, Inc. Treatment of filaggrin (flg) related diseases by modulation of flg expression and activity
TR201816523T4 (tr) 2009-11-12 2018-11-21 Univ Western Australia Patolojilerin tedavisine yönelik antisens moleküller ve yöntemler.
JP6025567B2 (ja) 2009-12-16 2016-11-16 カッパーアールエヌエー,インコーポレイテッド 膜結合転写因子ペプチダーゼ、部位1(mbtps1)に対する天然アンチセンス転写物の阻害によるmbtps1関連性疾患の治療
RU2619185C2 (ru) 2009-12-23 2017-05-12 Курна, Инк. Лечение заболеваний, связанных с разобщающим белком 2 (ucp2), путем ингибирования природного антисмыслового транскрипта к ucp2
CN102869776B (zh) 2009-12-23 2017-06-23 库尔纳公司 通过抑制肝细胞生长因子(hgf)的天然反义转录物而治疗hgf相关疾病
EP2519633B1 (en) 2009-12-29 2017-10-25 CuRNA, Inc. Treatment of nuclear respiratory factor 1 (nrf1) related diseases by inhibition of natural antisense transcript to nrf1
ES2585829T3 (es) 2009-12-29 2016-10-10 Curna, Inc. Tratamiento de las enfermedades relacionadas con la proteína tumoral 63 (p63) por inhibición de transcripción antisentido natural a p63
CA2785727C (en) 2009-12-31 2020-01-07 Curna, Inc. Treatment of insulin receptor substrate 2 (irs2) related diseases by inhibition of natural antisense transcript to irs2 and transcription factor e3 (tfe3)
CA2785832A1 (en) 2010-01-04 2011-07-07 Curna, Inc. Treatment of interferon regulatory factor 8 (irf8) related diseases by inhibition of natural antisense transcript to 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
EP2529015B1 (en) 2010-01-25 2017-11-15 CuRNA, Inc. Treatment of rnase h1 related diseases by inhibition of natural antisense transcript to rnase h1
CN102844435B (zh) 2010-02-22 2017-05-10 库尔纳公司 通过抑制吡咯啉‑5‑羧酸还原酶1(pycr1)的天然反义转录物而治疗pycr1相关疾病
EP2542155B1 (en) 2010-03-01 2015-11-04 TAU Therapeutics LLC Method for imaging a disease
WO2011112516A1 (en) * 2010-03-08 2011-09-15 Ico Therapeutics Inc. Treating and preventing hepatitis c virus infection using c-raf kinase antisense oligonucleotides
RU2612884C2 (ru) 2010-04-02 2017-03-13 Курна, Инк. Лечение заболеваний, связанных с колониестимулирующим фактором 3 (csf3), путем ингибирования природного антисмыслового транскрипта k csf3
WO2011127337A2 (en) 2010-04-09 2011-10-13 Opko Curna Llc Treatment of fibroblast growth factor 21 (fgf21) related diseases by inhibition of natural antisense transcript to fgf21
WO2011139387A1 (en) 2010-05-03 2011-11-10 Opko Curna, Llc Treatment of sirtuin (sirt) related diseases by inhibition of natural antisense transcript to a sirtuin (sirt)
TWI531370B (zh) 2010-05-14 2016-05-01 可娜公司 藉由抑制par4天然反股轉錄本治療par4相關疾病
US8895528B2 (en) 2010-05-26 2014-11-25 Curna, Inc. Treatment of atonal homolog 1 (ATOH1) related diseases by inhibition of natural antisense transcript to 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
CN109112126B (zh) 2010-06-23 2024-06-14 库尔纳公司 通过抑制电压门控钠通道α亚基(SCNA)的天然反义转录物而治疗SCNA相关疾病
JP5998131B2 (ja) 2010-07-14 2016-09-28 カッパーアールエヌエー,インコーポレイテッド Discslargehomolog(dlg)dlg1への天然アンチセンス転写物の阻害によるdlg関連疾患の治療
CN103210086B (zh) 2010-10-06 2017-06-09 库尔纳公司 通过抑制唾液酸酶4(neu4)的天然反义转录物而治疗neu4相关疾病
KR101865433B1 (ko) 2010-10-22 2018-07-13 큐알엔에이, 인크. 알파-l 이두로니다아제 (idua)에 대한 자연 안티센스 전사체의 저해에 의한 idua 관련된 질환의 치료
US9920317B2 (en) 2010-11-12 2018-03-20 The General Hospital Corporation Polycomb-associated non-coding RNAs
CA2817256A1 (en) 2010-11-12 2012-05-18 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
WO2012071238A2 (en) 2010-11-23 2012-05-31 Opko Curna Llc Treatment of nanog related diseases by inhibition of natural antisense transcript to nanog
KR102043422B1 (ko) 2011-06-09 2019-11-11 큐알엔에이, 인크. 프라탁신 (fxn)에 대한 자연 안티센스 전사체의 저해에 의한 프라탁신 (fxn) 관련된 질환의 치료
MX365525B (es) 2011-09-06 2019-06-06 Curna Inc Compuestos que regulan la expresión de subunidades alfa de canales de sodio regulados por voltaje en enfermedades relacionadas con epilepsia mioclónica severa de la infancia.
CN103957923B (zh) 2011-09-12 2018-05-25 卡维昂公司 用于抑制包括癌干细胞在内的干细胞的增殖、发育或分化的hs.459642单基因簇产物的拮抗剂
AU2012308320C1 (en) 2011-09-14 2018-08-23 Translate Bio Ma, Inc. Multimeric oligonucleotide compounds
NZ627896A (en) 2012-01-27 2016-11-25 Biomarin Technologies B V Rna modulating oligonucleotides with improved characteristics for the treatment of duchenne and becker muscular dystrophy
HK1210211A1 (en) 2012-03-15 2016-04-15 科纳公司 Treatment of brain derived neurotrophic factor (bdnf) related diseases by inhibition of natural antisense transcript to bdnf
AU2013262699A1 (en) 2012-05-16 2015-01-22 Rana Therapeutics, Inc. Compositions and methods for modulating ATP2A2 expression
SG11201407483YA (en) 2012-05-16 2014-12-30 Rana Therapeutics Inc Compositions and methods for modulating smn gene family expression
US10837014B2 (en) 2012-05-16 2020-11-17 Translate Bio Ma, Inc. Compositions and methods for modulating SMN gene family expression
EA201492122A1 (ru) 2012-05-16 2015-10-30 Рана Терапьютикс, Инк. Композиции и способы для модулирования экспрессии utrn
EA201492116A1 (ru) 2012-05-16 2015-05-29 Рана Терапьютикс, Инк. Композиции и способы для модулирования экспрессии mecp2
BR112014028631A2 (pt) 2012-05-16 2017-10-17 Rana Therapeutics Inc composições e métodos para modulação da expressão da família de genes da hemoglobina
US20150247141A1 (en) 2012-09-14 2015-09-03 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
CN110218727A (zh) 2013-03-14 2019-09-10 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
US20140329762A1 (en) 2013-03-15 2014-11-06 Sarepta Therapeutics, Inc. Compositions for treating muscular dystrophy
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
RS57970B1 (sr) 2014-03-20 2019-01-31 Cymabay Therapeutics Inc Tretman intrahepatičkih holestatskih oboljenja
US10272058B2 (en) 2014-03-20 2019-04-30 Cymabay Therapeutics, Inc. Treatment of intrahepatic cholestatic diseases
KR102374499B1 (ko) 2014-04-11 2022-03-14 사이머베이 쎄라퓨틱스, 인코퍼레이티드 Nafld 및 nash 의 치료
GB201410693D0 (en) 2014-06-16 2014-07-30 Univ Southampton Splicing modulation
AU2015327836B2 (en) 2014-10-03 2021-07-01 Cold Spring Harbor Laboratory Targeted augmentation of nuclear gene output
WO2016070060A1 (en) 2014-10-30 2016-05-06 The General Hospital Corporation Methods for modulating atrx-dependent gene repression
US10758558B2 (en) 2015-02-13 2020-09-01 Translate Bio Ma, 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
DK3364993T3 (da) 2015-10-22 2023-01-09 Cavion Inc Fremgangsmåder til behandling af angelman syndrom
US11096956B2 (en) 2015-12-14 2021-08-24 Stoke Therapeutics, Inc. Antisense oligomers and uses thereof
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
PH12018501207B1 (en) 2015-12-21 2024-02-23 Novartis Ag Compositions and methods for decreasing tau expression
CN110545806A (zh) 2017-02-15 2019-12-06 卡维昂公司 钙通道抑制剂
CN110770221B (zh) 2017-04-26 2023-09-08 卡维昂公司 用于改善记忆和认知以及用于治疗记忆和认知障碍的方法
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
KR102318434B1 (ko) 2017-08-25 2021-11-01 스톡 테라퓨틱스, 인크. 병태 및 질환 치료용 안티센스 올리고머
CA3099280A1 (en) 2018-05-04 2019-11-07 Stoke Therapeutics, Inc. Methods and compositions for treatment of cholesteryl ester storage disease
TWI879741B (zh) 2018-10-03 2025-04-11 美商卡凡恩公司 以(r)-2-(4-異丙基苯基)-n-(1-(5-(2,2,2-三氟乙氧基)吡啶-2-基)乙基)乙醯胺治療自發性震顫
AU2020227825B2 (en) 2019-02-27 2026-03-26 Stoke Therapeutics, Inc. Antisense oligomers for treatment of conditions and diseases
CN115867657A (zh) 2020-05-11 2023-03-28 斯托克制药公司 用于治疗疾患和疾病的opa1反义寡聚物

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
DE68927417T2 (de) 1988-04-27 1997-03-20 Isis Pharmaceutical Inc Oligoribonukleotid-Derivate und ihre Anwendung als antivirale Mittel
US5256775A (en) 1989-06-05 1993-10-26 Gilead Sciences, Inc. Exonuclease-resistant oligonucleotides
WO1992003568A1 (en) 1990-08-13 1992-03-05 Isis Pharmaceuticals, Inc. Sugar modified oligonucleotides that detect and modulate gene expression
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
ATE186072T1 (de) 1991-06-14 1999-11-15 Isis Pharmaceuticals Inc Antisense-oligonukleotid-inhibition des ras-gen
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 (enExample) 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
ATE299509T1 (de) 1992-07-23 2005-07-15 Isis Pharmaceuticals Inc Neue 2'-o-alkyl-nukleoside und -phosphoramidite, verfahren zu ihrer herstellung und ihre verwendungen
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
SK109198A3 (en) 1999-06-11
PL328563A1 (en) 1999-02-01
EP0882061B1 (en) 2004-05-19
CA2246229C (en) 2011-08-23
TR199801581T2 (xx) 1998-10-21
CN1214688A (zh) 1999-04-21
ATE267207T1 (de) 2004-06-15
US6451991B1 (en) 2002-09-17
DK0882061T3 (da) 2004-09-27
WO1997030067A1 (en) 1997-08-21
ZA971208B (en) 1997-10-23
NO983718L (no) 1998-10-13
JP2000504725A (ja) 2000-04-18
AU1955297A (en) 1997-09-02
KR19990082476A (ko) 1999-11-25
AU725262B2 (en) 2000-10-12
ES2221039T3 (es) 2004-12-16
CZ243498A3 (cs) 1999-09-15
DE69729179D1 (de) 2004-06-24
HUP9901118A2 (hu) 1999-07-28
HUP9901118A3 (en) 2000-06-28
JP4293636B2 (ja) 2009-07-08
NZ331217A (en) 2000-02-28
EP0882061A1 (en) 1998-12-09
EP0882061A4 (en) 2000-12-13
CA2749312A1 (en) 1997-08-21
IL125759A0 (en) 1999-04-11
CA2246229A1 (en) 1997-08-21
NO983718D0 (no) 1998-08-13
BR9707529A (pt) 2000-01-04

Similar Documents

Publication Publication Date Title
DE69729179T2 (de) Lückenhaft Zucker-modifizierte Oligonukleotide
DE69232032T2 (de) Antisense oligonukleotide
DE69133405T2 (de) Oligonukleotidderivate zur detektieren und modulation von rna aktivität und genexpression
DE69232699T2 (de) Über chirale phosphoratome gebundene oligonukleotide
DE69126530T2 (de) Nuklease resistente, pyrimidin modifizierte oligonukleotide, die die gen-expression detektieren und modulieren
DE69430818T2 (de) Pns-dns-pns-chimäre makromoleküle
DE60038495T2 (de) Oligonukleotid n3'-p5' thiophosphoramidate: ihre synthese und verwendung
DE69533697T2 (de) ANTISENSE OLIGONUKLEOTIDMODULATION DER raf GENEXPRESSION
DE69838449T2 (de) Aminooxy-modifizierte oligonukleotide
DE69133511T2 (de) Zuckermodifizierte oligonukleotide zum erkennen und steuern der genexpression
DE69230223T2 (de) Antisense-oligonukleotid-inhibition des ras-gen
DE69732911T2 (de) Oligoribonukleotide und ribonukleasen zur spaltung von rns
US5955589A (en) Gapped 2' modified oligonucleotides
DE69620906T2 (de) Antisense-oligonukleotidmodulation der raf-genexpression
US7015315B1 (en) Gapped oligonucleotides
DE69427705T2 (de) Bor-cluster enthaltende nukleoside und oligonukleoside
DE69331524T2 (de) Verfahren zum spezifischen schneiden von rna-strängen
DE69433036T2 (de) Aminoderivatisierte nukleoside und oligonukleoside
DE69033495T2 (de) 2'-modifizierte nukleotide
US5856455A (en) Gapped 2'-modified oligonucleotides
DE69432934T2 (de) Oligonukleotidmodulation der expression von multiwirkstoff-resistenz -assoziiertem protein
DE69432315T2 (de) ANTISENSE NUKLEINSÄUREN ZUR VORBEUGUNG UND BEHANDLUNG VON BESCHWERDEN IN WELCHEN DIE EXPRIMIERUNG VON C-erbB-2 EINE ROLLE SPIELT
DE69934227T2 (de) Auf Beta-Arabinose und dessen Analoga basierte Antisense-Oligonukleotide
US20060270624A1 (en) Gapped 2' modified oligonucleotides
DE69130569T2 (de) Mit psoralen konjugierte methylphosphonat-oligonukleotide als therapeutische wirkstoffe für chronische myologene leukämie

Legal Events

Date Code Title Description
8364 No opposition during term of opposition