JPH08510130A - 異常スプライシングを阻害するアンチセンスオリゴヌクレオチドと同物質の利用法 - Google Patents

異常スプライシングを阻害するアンチセンスオリゴヌクレオチドと同物質の利用法

Info

Publication number
JPH08510130A
JPH08510130A JP6525643A JP52564394A JPH08510130A JP H08510130 A JPH08510130 A JP H08510130A JP 6525643 A JP6525643 A JP 6525643A JP 52564394 A JP52564394 A JP 52564394A JP H08510130 A JPH08510130 A JP H08510130A
Authority
JP
Japan
Prior art keywords
mrna
splicing
normal
antisense oligonucleotide
mutation
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.)
Withdrawn
Application number
JP6525643A
Other languages
English (en)
Japanese (ja)
Inventor
コール，リスザード
ドミンスキー，ズビグニエフ
Original Assignee
ザ・ユニヴァーシティ・オヴ・ノース・キャロライナ・アト・チャペル・ヒル
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 ザ・ユニヴァーシティ・オヴ・ノース・キャロライナ・アト・チャペル・ヒル filed Critical ザ・ユニヴァーシティ・オヴ・ノース・キャロライナ・アト・チャペル・ヒル
Publication of JPH08510130A publication Critical patent/JPH08510130A/ja
Withdrawn legal-status Critical Current

Links

Classifications

    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/321—2'-O-R Modification
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/352—Nature of the modification linked to the nucleic acid via a carbon atom
    • C12N2310/3521—Methyl

Landscapes

  • Genetics & Genomics (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Health & Medical Sciences (AREA)
  • Biomedical Technology (AREA)
  • Molecular Biology (AREA)
  • Wood Science & Technology (AREA)
  • Organic Chemistry (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Zoology (AREA)
  • Physics & Mathematics (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Biochemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Biophysics (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Saccharide Compounds (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Preparation Of Compounds By Using Micro-Organisms (AREA)
JP6525643A 1993-05-11 1994-05-10 異常スプライシングを阻害するアンチセンスオリゴヌクレオチドと同物質の利用法 Withdrawn JPH08510130A (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US6247193A 1993-05-11 1993-05-11
US062,471 1993-05-11
PCT/US1994/005181 WO1994026887A1 (en) 1993-05-11 1994-05-10 Antisense oligonucleotides which combat aberrant splicing and methods of using the same

Related Child Applications (1)

Application Number Title Priority Date Filing Date
JP2008000648A Division JP2008092960A (ja) 1993-05-11 2008-01-07 異常スプライシングを阻害するアンチセンスオリゴヌクレオチドと同物質の利用法

Publications (1)

Publication Number Publication Date
JPH08510130A true JPH08510130A (ja) 1996-10-29

Family

ID=22042714

Family Applications (3)

Application Number Title Priority Date Filing Date
JP6525643A Withdrawn JPH08510130A (ja) 1993-05-11 1994-05-10 異常スプライシングを阻害するアンチセンスオリゴヌクレオチドと同物質の利用法
JP2008000648A Withdrawn JP2008092960A (ja) 1993-05-11 2008-01-07 異常スプライシングを阻害するアンチセンスオリゴヌクレオチドと同物質の利用法
JP2009042209A Pending JP2009159971A (ja) 1993-05-11 2009-02-25 異常スプライシングを阻害するアンチセンスオリゴヌクレオチドと同物質の利用法

Family Applications After (2)

Application Number Title Priority Date Filing Date
JP2008000648A Withdrawn JP2008092960A (ja) 1993-05-11 2008-01-07 異常スプライシングを阻害するアンチセンスオリゴヌクレオチドと同物質の利用法
JP2009042209A Pending JP2009159971A (ja) 1993-05-11 2009-02-25 異常スプライシングを阻害するアンチセンスオリゴヌクレオチドと同物質の利用法

Country Status (15)

Country Link
US (4) US5665593A (enExample)
EP (2) EP0698092B1 (enExample)
JP (3) JPH08510130A (enExample)
CN (1) CN1123038A (enExample)
AT (1) ATE368107T1 (enExample)
AU (1) AU6786594A (enExample)
CA (1) CA2162361C (enExample)
DE (1) DE69435005T2 (enExample)
DK (1) DK0698092T3 (enExample)
ES (1) ES2290951T3 (enExample)
FI (1) FI955428A7 (enExample)
NO (1) NO954550L (enExample)
NZ (1) NZ266386A (enExample)
PT (1) PT698092E (enExample)
WO (1) WO1994026887A1 (enExample)

Cited By (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US6653467B1 (en) 2000-04-26 2003-11-25 Jcr Pharmaceutical Co., Ltd. Medicament for treatment of Duchenne muscular dystrophy
US6727355B2 (en) 2000-08-25 2004-04-27 Jcr Pharmaceuticals Co., Ltd. Pharmaceutical composition for treatment of Duchenne muscular dystrophy
WO2018164275A1 (ja) * 2017-03-10 2018-09-13 国立研究開発法人国立成育医療研究センター アンチセンスオリゴヌクレオチドおよび糖原病Ｉａ型予防または治療用組成物
WO2019172286A1 (ja) * 2018-03-09 2019-09-12 第一三共株式会社 糖原病Ｉａ型治療薬
WO2021112106A1 (ja) * 2019-12-02 2021-06-10 第一三共株式会社 色素性乾皮症ｆ群治療薬

Families Citing this family (158)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US8153602B1 (en) * 1991-11-19 2012-04-10 Isis Pharmaceuticals, Inc. Composition and methods for the pulmonary delivery of nucleic acids
GB9514435D0 (en) * 1995-07-14 1995-09-13 Danisco Inhibition of gene expression
US5652356A (en) * 1995-08-17 1997-07-29 Hybridon, Inc. Inverted chimeric and hybrid oligonucleotides
US7074768B2 (en) 1995-08-17 2006-07-11 Idera Pharmaceuticals, Inc. Modified protein kinase A-specific oligonucleotides and methods of their use
US6624293B1 (en) 1995-08-17 2003-09-23 Hybridon, Inc. Modified protein kinase A-specific oligonucleotides and methods of their use
GB9519299D0 (en) 1995-09-21 1995-11-22 Farrar Gwyneth J Genetic strategy
GB9604449D0 (en) * 1996-03-01 1996-05-01 Farrar Gwyneth J Genetic strategy ii
GB9606961D0 (en) 1996-04-02 1996-06-05 Farrar Gwyneth J Genetic strategy III
US8551970B2 (en) 1996-04-02 2013-10-08 Optigen Patents Limited Genetic suppression and replacement
US20030036519A1 (en) * 1998-04-21 2003-02-20 Ryszard Kole Stable alteration of pre-mrna splicing patterns by modified rnas
US6210892B1 (en) 1998-10-07 2001-04-03 Isis Pharmaceuticals, Inc. Alteration of cellular behavior by antisense modulation of mRNA processing
US6214986B1 (en) 1998-10-07 2001-04-10 Isis Pharmaceuticals, Inc. Antisense modulation of bcl-x expression
JP2000325085A (ja) 1999-05-21 2000-11-28 Masafumi Matsuo デュシェンヌ型筋ジストロフィー治療剤
US6677445B1 (en) 1999-08-27 2004-01-13 Chiron Corporation Chimeric antisense oligonucleotides and cell transfecting formulations thereof
CA2400573A1 (en) * 2000-03-28 2001-10-04 Isis Pharmaceuticals Inc. Alteration of cellular behavior by antisense modulation of mrna processing
AU5752601A (en) * 2000-05-04 2001-11-12 Avi Biopharma Inc Splice-region antisense composition and method
EP1191097A1 (en) * 2000-09-21 2002-03-27 Leids Universitair Medisch Centrum Induction of exon skipping in eukaryotic cells
GB2382577A (en) * 2001-11-28 2003-06-04 Talat Nasim A reporter-based assay for assessing relative RNA processing activity in mammaliian cells
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
EP1651338B1 (en) 2003-07-17 2011-05-18 Unilever N.V. Process for the preparation of an edible dispersion comprising oil and structuring agent
AU2005208710A1 (en) 2004-01-23 2005-08-11 Avi Biopharma, Inc. Antisense oligomers and methods for inducing immune tolerance and immunosuppression
WO2005094370A2 (en) * 2004-03-29 2005-10-13 The General Hospital Corporation Oligonucleotide complex compositions and methods of use as gene alteration tools
KR101147147B1 (ko) 2004-04-01 2012-05-25 머크 샤프 앤드 돔 코포레이션 Ｒｎａ 간섭의 오프 타겟 효과 감소를 위한 변형된폴리뉴클레오타이드
USRE48960E1 (en) 2004-06-28 2022-03-08 The University Of Western Australia Antisense oligonucleotides for inducing exon skipping and methods of use thereof
PL1766010T3 (pl) 2004-06-28 2011-07-29 Univ Western Australia Antysensowne oligonukleotydy do indukcji pominięcia egzonu i sposoby ich zastosowania
US7879992B2 (en) 2005-01-31 2011-02-01 Isis Pharmaceuticals, Inc. Modification of MyD88 splicing using modified oligonucleotides
EP1855694B1 (en) 2005-02-09 2020-12-02 Sarepta Therapeutics, Inc. Antisense composition for treating muscle atrophy
EP1865786B2 (en) 2005-02-17 2015-10-14 Unilever N.V. Process for the preparation of a spreadable dispersion
US20100196335A1 (en) * 2005-04-29 2010-08-05 Samulski Richard J Methods and Compositions for Regulated Expressions of Nucleic Acid at Post-Transcriptional Level
EP3470072A1 (en) 2005-06-23 2019-04-17 Biogen MA Inc. Compositions and methods for modulation of smn2 splicing
US8501703B2 (en) 2005-08-30 2013-08-06 Isis Pharmaceuticals, Inc. Chimeric oligomeric compounds for modulation of splicing
WO2007047913A2 (en) * 2005-10-20 2007-04-26 Isis Pharmaceuticals, Inc Compositions and methods for modulation of lmna expression
US7785834B2 (en) * 2005-11-10 2010-08-31 Ercole Biotech, Inc. Soluble TNF receptors and their use in treatment of disease
EP2535413A3 (en) * 2005-11-10 2016-04-13 Roche Innovation Center Copenhagen A/S Splice switching oligomers for the TNF superfamily receptors and their use in treatment of disease
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
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
US20090264353A1 (en) * 2007-10-19 2009-10-22 Santaris Pharma A/S Splice Switching Oligomers for TNF Superfamily Receptors and their Use in Treatment of Disease
ES2798758T3 (es) * 2007-06-06 2020-12-14 Sarepta Therapeutics Inc Proteínas variantes de empalme her2 y her3 solubles, oligonucleótidos de cambio de empalme y su uso en el tratamiento de enfermedades
AU2008273094B2 (en) * 2007-07-12 2013-05-09 Prosensa Technologies B.V. Molecules for targeting compounds to various selected organs, tissues or tumor cells
USRE48468E1 (en) 2007-10-26 2021-03-16 Biomarin Technologies B.V. Means and methods for counteracting muscle disorders
CN101896186A (zh) 2007-10-26 2010-11-24 莱顿教学医院 对抗肌肉病症的方式和方法
US8637478B2 (en) 2007-11-13 2014-01-28 Isis Pharmaceuticals, Inc. Compounds and methods for modulating protein expression
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
SMT201900159T1 (it) * 2008-10-24 2019-05-10 Sarepta Therapeutics Inc Composizioni di salto di esone per dmd
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
KR101840618B1 (ko) 2008-12-04 2018-03-20 큐알엔에이, 인크. 종양 억제 유전자에 대한 천연 안티센스 전사체의 억제에 의해 종양 억제 유전자 관련된 질환의 치료
CA2746003C (en) 2008-12-04 2020-03-31 Opko Curna, Llc Treatment of vascular endothelial growth factor (vegf) related diseases by inhibition of natural antisense transcript to vegf
WO2010091308A2 (en) 2009-02-06 2010-08-12 Isis Pharmaceuticals, Inc. Oligomeric compounds and methods
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
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
CA2755404C (en) 2009-03-17 2020-03-24 Joseph Collard Treatment of delta-like 1 homolog (dlk1) related diseases by inhibition of natural antisense transcript to dlk1
CA2759899A1 (en) 2009-04-24 2010-10-28 Prosensa Technologies B.V. Oligonucleotide comprising an inosine for treating dmd
EP2427553A4 (en) * 2009-05-06 2012-11-07 Opko Curna Llc TREATMENT OF LIPID TRANSPORT AND METABOLISM-RELATED DISEASES BY INHIBITING THE NATURAL ANTISENSE TRANSCRIPT AGAINST A LIPID TRANSPORT AND METABOLIC TREATMENT
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
WO2010129861A2 (en) 2009-05-08 2010-11-11 Curna, Inc. Treatment of dystrophin family related diseases by inhibition of natural antisense transcript to dmd family
US8957037B2 (en) 2009-05-18 2015-02-17 Curna, Inc. 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
CA2764683A1 (en) 2009-05-28 2010-12-02 Joseph Collard Treatment of antiviral gene related diseases by inhibition of natural antisense transcript to an antiviral gene
JP6128846B2 (ja) 2009-06-16 2017-05-17 クルナ・インコーポレーテッド パラオキソナーゼ（ｐｏｎ１）に対する天然アンチセンス転写物の抑制によるｐｏｎ１遺伝子関連疾患の治療
CN102695797B (zh) 2009-06-16 2018-05-25 库尔纳公司 通过抑制针对胶原基因的天然反义转录物来治疗胶原基因相关的疾病
KR20120093138A (ko) 2009-06-17 2012-08-22 콜드스프링하버러보러토리 대상에게서 ｓｍｎ２ 스플라이싱을 조정하기 위한 조성물 및 방법
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
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
JP2013500017A (ja) 2009-07-24 2013-01-07 カッパーアールエヌエー，インコーポレイテッド サ−チュイン（ｓｉｒｔ）への天然アンチセンス転写物の阻止によるサ−チュイン（ｓｉｒｔ）関連疾患の治療
EP2462229B1 (en) 2009-08-05 2016-05-11 CuRNA, Inc. Treatment of insulin gene (ins) related diseases by inhibition of natural antisense transcript to an insulin gene (ins)
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
CA2771228C (en) 2009-08-21 2020-12-29 Opko Curna, Llc Treatment of 'c terminus of hsp70-interacting protein' (chip) related diseases by inhibition of natural antisense transcript to chip
KR101892760B1 (ko) 2009-08-25 2018-08-28 큐알엔에이, 인크. ＩＱＧＡＰ에 대한 천연 안티센스 전사체의 억제에 의한 ＧＴＰａｓｅ 활성화 단백질을 함유하는 ＩＱ 모티프(ＩＱＧＡＰ)와 관련된 질환의 치료
JP6175236B2 (ja) 2009-09-25 2017-08-09 カッパーアールエヌエー，インコーポレイテッド フィラグリン（ｆｌｇ）の発現および活性の調整によるｆｌｇ関連疾患の処置
EE05711B1 (et) * 2009-10-07 2014-04-15 Tartu Ülikool Meetod ja kompositsioon konditsionaalselt letaalsete viirusmutantide loomiseks ja eukarootse raku elulisuse k?rvaldamiseks
KR20230137491A (ko) 2009-11-12 2023-10-04 더 유니버시티 오브 웨스턴 오스트레일리아 안티센스 분자 및 이를 이용한 질환 치료방법
NO2513310T3 (enExample) 2009-12-16 2018-03-31
NO2516648T3 (enExample) 2009-12-23 2018-04-07
CA2782375C (en) 2009-12-23 2023-10-31 Opko Curna, Llc Treatment of uncoupling protein 2 (ucp2) related diseases by inhibition of natural antisense transcript to ucp2
ES2616561T3 (es) 2009-12-24 2017-06-13 Biomarin Technologies B.V. Molécula para tratar un trastorno inflamatorio
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
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
CN102791862B (zh) 2009-12-31 2017-04-05 库尔纳公司 通过抑制胰岛素受体底物2(irs2)和转录因子e3(tfe3)的天然反义转录物而治疗irs2相关疾病
CN102906264B (zh) 2010-01-04 2017-08-04 库尔纳公司 通过抑制干扰素调节因子8(irf8)的天然反义转录物而治疗irf8相关疾病
JP5963680B2 (ja) 2010-01-06 2016-08-03 カッパーアールエヌエー，インコーポレイテッド 膵臓発生遺伝子に対する天然アンチセンス転写物の阻害による膵臓発生遺伝子疾患の治療
CA2786535C (en) 2010-01-11 2019-03-26 Curna, Inc. Treatment of sex hormone binding globulin (shbg) related diseases by inhibition of natural antisense transcript to shbg
JP5981850B2 (ja) 2010-01-25 2016-08-31 カッパーアールエヌエー，インコーポレイテッド ＲＮａｓｅＨ１に対する天然アンチセンス転写物の阻害によるＲＮａｓｅＨ１関連疾患の治療
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
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
CN102858979B (zh) 2010-04-09 2018-01-26 库尔纳公司 通过抑制成纤维细胞生长因子21(fgf21)的天然反义转录物而治疗fgf21相关疾病
US8802642B2 (en) 2010-04-28 2014-08-12 Iowa State University Research Foundation, Inc. Spinal muscular atrophy treatment via targeting SMN2 catalytic core
KR101936011B1 (ko) 2010-05-03 2019-01-07 큐알엔에이, 인크. 시르투인 (sirt)에 대한 자연 안티센스 전사체의 저해에 의한 시르투인 (sirt) 관련된 질환의 치료
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
US8980858B2 (en) 2010-05-26 2015-03-17 Curna, Inc. Treatment of methionine sulfoxide reductase a (MSRA) related diseases by inhibition of natural antisense transcript to MSRA
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
EA024216B1 (ru) 2010-06-22 2016-08-31 Юнилевер Н.В. Порошкообразные пищевые жиры
KR101914309B1 (ko) 2010-06-23 2018-11-02 큐알엔에이, 인크. 전압 작동 나트륨 통로, 알파 소단위(scna)에 대한 자연 안티센스 전사체의 저해에 의한 전압 작동 나트륨 통로, 알파 소단위(scna) 관련된 질환의 치료
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
KR101886457B1 (ko) 2010-10-06 2018-08-07 큐알엔에이, 인크. 시알리다아제 4 (neu4)에 대한 자연 안티센스 전사체의 저해에 의한 neu4 관련된 질환의 치료
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
EP2638163B1 (en) 2010-11-12 2017-05-17 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
HUE026515T2 (en) 2010-12-17 2016-06-28 Unilever Bcs Europe Bv Edible water-in-oil emulsion
WO2012079955A1 (en) 2010-12-17 2012-06-21 Unilever Nv Process of compacting a microporous fat powder and compacted fat powder so obtained
CN103687621A (zh) 2011-06-08 2014-03-26 印第安纳大学研究与技术公司 用于诊断和治疗肺疾病和损伤的单克隆抗体和抗原
TWI678375B (zh) 2011-06-09 2019-12-01 可娜公司 藉由抑制共濟蛋白（ｆｒａｔａｘｉｎ，ｆｘｎ）之天然反股轉錄本治療ｆｘｎ相關疾病
CN103874486A (zh) 2011-09-06 2014-06-18 库尔纳公司 用小分子治疗与电压门控钠通道的α亚基(SCNxA)相关的疾病
US20130085139A1 (en) 2011-10-04 2013-04-04 Royal Holloway And Bedford New College Oligomers
IL321422A (en) 2012-01-11 2025-08-01 Ionis Pharmaceuticals Inc Compositions and methods for modulation of ikbkap splicing
BR112014018427B1 (pt) 2012-01-27 2021-11-03 Biomarin Technologies B.V. Oligonucleotídeos moduladores de rna com características melhoradas para o tratamento da distrofia muscular de duchenne e becker
EP2825648B1 (en) 2012-03-15 2018-09-05 CuRNA, Inc. Treatment of brain derived neurotrophic factor (bdnf) related diseases by inhibition of natural antisense transcript to bdnf
WO2013173638A1 (en) 2012-05-16 2013-11-21 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
AU2013262709A1 (en) 2012-05-16 2015-01-22 Rana Therapeutics, Inc. Compositions and methods for modulating MECP2 expression
SI2852668T1 (sl) 2012-07-12 2016-09-30 Proqr Therapeutics Ii B.V. Oligonukleotidi za izvedbo spremembe v sekvenci tarčne molekule rna, prisotne v živeči celici
EP2935584A1 (en) 2012-12-20 2015-10-28 Sarepta Therapeutics, Inc. Improved exon skipping compositions for treating muscular dystrophy
WO2014110291A1 (en) 2013-01-09 2014-07-17 Isis Pharmaceuticals, Inc. Compositions and methods for modulation of smn2 splicing in a subject
KR20240094032A (ko) 2013-03-14 2024-06-24 사렙타 쎄러퓨틱스 인코퍼레이티드 근육 이영양증의 치료를 위한 엑손 스키핑 조성물
NZ631245A (en) 2013-03-14 2017-09-29 Sarepta Therapeutics Inc Exon skipping compositions for treating muscular dystrophy
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
WO2014172698A1 (en) 2013-04-19 2014-10-23 Isis Pharmaceuticals, Inc. Compositions and methods for modulation nucleic acids through nonsense mediated decay
WO2015051283A1 (en) 2013-10-04 2015-04-09 Rana Therapeutics, Inc. Compositions and methods for treating amyotrophic lateral sclerosis
WO2015161170A2 (en) 2014-04-17 2015-10-22 Isis Pharmaceuticals, Inc. Compositions and methods for modulation of smn2 splicing in a subject
GB201410693D0 (en) * 2014-06-16 2014-07-30 Univ Southampton Splicing modulation
US10436802B2 (en) 2014-09-12 2019-10-08 Biogen Ma Inc. Methods for treating spinal muscular atrophy
CN107109411B (zh) 2014-10-03 2022-07-01 冷泉港实验室 核基因输出的定向增加
WO2016070060A1 (en) 2014-10-30 2016-05-06 The General Hospital Corporation Methods for modulating atrx-dependent gene repression
WO2016149455A2 (en) 2015-03-17 2016-09-22 The General Hospital Corporation The rna interactome of polycomb repressive complex 1 (prc1)
MA41795A (fr) 2015-03-18 2018-01-23 Sarepta Therapeutics Inc Exclusion d'un exon induite par des composés antisens dans la myostatine
EP3359685A1 (en) 2015-10-09 2018-08-15 University Of Southampton Modulation of gene expression and screening for deregulated protein expression
US11136584B2 (en) 2015-11-04 2021-10-05 Duke University Splice-switching oligonucleotides and methods of use
EA201800367A1 (ru) 2015-12-10 2019-02-28 ПиТиСи ТЕРАПЬЮТИКС, ИНК. Способы лечения болезни хантингтона
US11083745B2 (en) 2015-12-14 2021-08-10 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
TWI669394B (zh) * 2016-05-03 2019-08-21 臺中榮民總醫院 一種對突變型多巴脫羧酶基因做剪接調整的反義寡核苷酸及其使用方法
EP3471779A4 (en) 2016-06-16 2020-07-08 Ionis Pharmaceuticals, Inc. COMBINATIONS FOR MODULATING SMN EXPRESSION
KR20190024977A (ko) 2016-06-30 2019-03-08 사렙타 쎄러퓨틱스 인코퍼레이티드 근육 이상증에 대한 엑손 스킵핑 올리고머
SG10202012839TA (en) 2016-12-19 2021-01-28 Sarepta Therapeutics Inc Exon skipping oligomer conjugates for muscular dystrophy
MD3554554T2 (ro) 2016-12-19 2022-12-31 Sarepta Therapeutics Inc Conjugați de oligomeri de omitere a exonului, pentru distrofie musculară
BR112019012664A2 (pt) 2016-12-19 2020-01-21 Sarepta Therapeutics Inc conjugados de oligômero de salto de éxon para distrofia muscular
KR20200033249A (ko) 2017-06-05 2020-03-27 피티씨 테라퓨틱스, 인크. 헌팅턴병 치료 화합물
CA3067591A1 (en) 2017-06-28 2019-01-03 Ptc Therapeutics, Inc. Methods for treating huntington's disease
US11395822B2 (en) 2017-06-28 2022-07-26 Ptc Therapeutics, Inc. Methods for treating Huntington's disease
SMT202400071T1 (it) 2017-08-25 2024-03-13 Stoke Therapeutics Inc Oligomeri antisenso per il trattamento di condizioni e malattie
EA201991450A1 (ru) 2017-09-22 2019-12-30 Сарепта Терапьютикс, Инк. Конъюгаты олигомеров для пропуска экзона при мышечной дистрофии
US11866689B2 (en) 2018-03-26 2024-01-09 Duke University Splice-switching oligonucleotides and methods of use
US12103926B2 (en) 2018-03-27 2024-10-01 Ptc Therapeutics, Inc. Compounds for treating huntington's disease
JP2021523227A (ja) 2018-05-04 2021-09-02 ストーク セラピューティクス，インク． コレステリルエステル蓄積症の処置のための方法及び組成物
US10758629B2 (en) 2018-05-29 2020-09-01 Sarepta Therapeutics, Inc. Exon skipping oligomer conjugates for muscular dystrophy
BR112020026545A2 (pt) 2018-06-27 2021-03-23 Ptc Therapeutics, Inc. Compostos heterocíclicos e de heteroarila para o tratamento da doença de huntington
EA202092899A1 (ru) 2018-06-27 2021-05-14 ПиТиСи ТЕРАПЬЮТИКС, ИНК. Гетероарильные соединения для лечения болезни гентингтона
WO2020191212A1 (en) 2019-03-20 2020-09-24 President And Fellows Of Harvard College Antisense oligonucleotide-based progranulin augmentation therapy in neurodegenerative diseases
EP3955966A1 (en) 2019-04-18 2022-02-23 Sarepta Therapeutics, Inc. Compositions for treating muscular dystrophy
CN112111528B (zh) * 2019-06-21 2024-04-02 华东师范大学 一种内含子异常剪接的修复方法
AU2020377204A1 (en) 2019-11-01 2022-06-02 Novartis Ag The use of a splicing modulator for a treatment slowing progression of huntington's disease
JP7446443B2 (ja) 2020-02-28 2024-03-08 アイオーニス ファーマシューティカルズ， インコーポレーテッド Ｓｍｎ２を調節するための化合物及び方法
CA3173647A1 (en) 2020-05-11 2021-11-18 Isabel AZNAREZ Opa1 antisense oligomers for treatment of conditions and diseases
TW202304446A (zh) 2021-03-29 2023-02-01 瑞士商諾華公司 剪接調節子用於減慢杭丁頓氏舞蹈症進展的治療之用途
CA3218805A1 (en) 2021-05-10 2022-11-17 Ziqing QIAN Compositions and methods for intracellular therapeutics
WO2025165891A1 (en) 2024-01-29 2025-08-07 Arnatar Therapeutics, Inc Translation enhancing nucleic acid compounds: aso coupled translation - upregulation 1 (act-up1) and uses thereof

Family Cites Families (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5023243A (en) * 1981-10-23 1991-06-11 Molecular Biosystems, Inc. Oligonucleotide therapeutic agent and method of making same
US5220007A (en) * 1990-02-15 1993-06-15 The Worcester Foundation For Experimental Biology Method of site-specific alteration of RNA and production of encoded polypeptides
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

Cited By (14)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US6653467B1 (en) 2000-04-26 2003-11-25 Jcr Pharmaceutical Co., Ltd. Medicament for treatment of Duchenne muscular dystrophy
EP1544297A2 (en) 2000-04-26 2005-06-22 JCR Pharmaceuticals Co., Ltd. Medicament for treatment of Duchenne muscular dystrophy
US6727355B2 (en) 2000-08-25 2004-04-27 Jcr Pharmaceuticals Co., Ltd. Pharmaceutical composition for treatment of Duchenne muscular dystrophy
JP2022093334A (ja) * 2017-03-10 2022-06-23 国立研究開発法人国立成育医療研究センター アンチセンスオリゴヌクレオチドおよび糖原病Ｉａ型予防または治療用組成物
JPWO2018164275A1 (ja) * 2017-03-10 2020-01-16 国立研究開発法人国立成育医療研究センター アンチセンスオリゴヌクレオチドおよび糖原病Ｉａ型予防または治療用組成物
WO2018164275A1 (ja) * 2017-03-10 2018-09-13 国立研究開発法人国立成育医療研究センター アンチセンスオリゴヌクレオチドおよび糖原病Ｉａ型予防または治療用組成物
JP2024009169A (ja) * 2017-03-10 2024-01-19 国立研究開発法人国立成育医療研究センター アンチセンスオリゴヌクレオチドおよび糖原病Ｉａ型予防または治療用組成物
US11963974B2 (en) 2017-03-10 2024-04-23 National Center For Child Health And Development Antisense oligonucleotide and composition for prevention or treatment of glycogen storage disease type Ia
WO2019172286A1 (ja) * 2018-03-09 2019-09-12 第一三共株式会社 糖原病Ｉａ型治療薬
JPWO2019172286A1 (ja) * 2018-03-09 2021-03-18 第一三共株式会社 糖原病Ｉａ型治療薬
JP2021072800A (ja) * 2018-03-09 2021-05-13 第一三共株式会社 糖原病Ｉａ型治療薬
JP2023159397A (ja) * 2018-03-09 2023-10-31 第一三共株式会社 糖原病Ｉａ型治療薬
US11958878B2 (en) 2018-03-09 2024-04-16 Daiichi Sankyo Company, Limited Therapeutic agent for glycogen storage disease type IA
WO2021112106A1 (ja) * 2019-12-02 2021-06-10 第一三共株式会社 色素性乾皮症ｆ群治療薬

Also Published As

Publication number Publication date
CN1123038A (zh) 1996-05-22
NO954550D0 (no) 1995-11-10
ES2290951T3 (es) 2008-02-16
CA2162361A1 (en) 1994-11-24
FI955428L (fi) 1996-01-04
NO954550L (no) 1996-01-10
AU6786594A (en) 1994-12-12
CA2162361C (en) 2008-10-21
DE69435005D1 (de) 2007-09-06
EP0698092B1 (en) 2007-07-25
US5976879A (en) 1999-11-02
FI955428A7 (fi) 1996-01-04
WO1994026887A1 (en) 1994-11-24
EP1897942A1 (en) 2008-03-12
US5627274A (en) 1997-05-06
ATE368107T1 (de) 2007-08-15
US5665593A (en) 1997-09-09
DK0698092T3 (da) 2007-11-26
JP2008092960A (ja) 2008-04-24
FI955428A0 (fi) 1995-11-10
PT698092E (pt) 2007-10-29
NZ266386A (en) 1997-11-24
JP2009159971A (ja) 2009-07-23
US5916808A (en) 1999-06-29
EP0698092A1 (en) 1996-02-28
DE69435005T2 (de) 2008-04-17

Similar Documents

Publication Publication Date Title
JPH08510130A (ja) 異常スプライシングを阻害するアンチセンスオリゴヌクレオチドと同物質の利用法
JP2009159971A6 (ja) 異常スプライシングを阻害するアンチセンスオリゴヌクレオチドと同物質の利用法
US8546346B2 (en) Methods to reprogram splice site selection in pre-messenger RNAs
US8263760B2 (en) Methods and means for treating DNA repeat instability associated genetic disorders
JP7113532B2 (ja) スタルガルト病の処置のためのアンチセンスオリゴヌクレオチド
JP5917497B2 (ja) メチオニンスルホキシドレダクターゼａ（ｍｓｒａ）に対する天然アンチセンス転写物の阻害によるｍｓｒａ関連疾患の治療
JPH10506536A (ja) 安定化外部ガイド配列
CA2771228A1 (en) Treatment of 'c terminus of hsp70-interacting protein' (chip) related diseases by inhibition of natural antisense transcript to chip
WO1995011687A1 (en) Therapeutic use of cis-element decoys in vivo
JP2013523770A (ja) 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
TW201143782A (en) Treatment of LIM homeobox 2 (LHX2) related diseases by inhibition of natural antisense transcript to LHX2
EP3253874B1 (en) Treatment of facioscapulohumeral dystrophy
KR20200014320A (ko) Apcs의 발현을 억제하는 핵산
JP2022518808A (ja) レーバー先天黒内障の処置のためのアンチセンスオリゴヌクレオチド
US5872007A (en) CAPL-specific oligonucleotides and methods of inhibiting metastatic cancer
WO2024077262A2 (en) Methods and compositions for silencing elavl2 expression for the treatment of disease

Legal Events

Date Code Title Description
A02 Decision of refusal

Free format text: JAPANESE INTERMEDIATE CODE: A02

Effective date: 20040309

A601 Written request for extension of time

Free format text: JAPANESE INTERMEDIATE CODE: A601

Effective date: 20071004

A602 Written permission of extension of time

Free format text: JAPANESE INTERMEDIATE CODE: A602

Effective date: 20071011

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20080107

A761 Written withdrawal of application

Free format text: JAPANESE INTERMEDIATE CODE: A761

Effective date: 20080121