JP2011529686A - ポリグルタミンタンパク質発現の選択的阻害 - Google Patents

ポリグルタミンタンパク質発現の選択的阻害 Download PDF

Info

Publication number
JP2011529686A
JP2011529686A JP2011521239A JP2011521239A JP2011529686A JP 2011529686 A JP2011529686 A JP 2011529686A JP 2011521239 A JP2011521239 A JP 2011521239A JP 2011521239 A JP2011521239 A JP 2011521239A JP 2011529686 A JP2011529686 A JP 2011529686A
Authority
JP
Japan
Prior art keywords
nucleic acid
pna
acid analog
mrna
repeat region
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Withdrawn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
JP2011521239A
Other languages
English (en)
Japanese (ja)
Other versions
JP2011529686A5 (https=
Inventor
デイビッド アール． コーリー
チアシン フー
雅之 松井
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.)
University of Texas System
Original Assignee
University of Texas System
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 University of Texas System filed Critical University of Texas System
Publication of JP2011529686A publication Critical patent/JP2011529686A/ja
Publication of JP2011529686A5 publication Critical patent/JP2011529686A5/ja
Withdrawn legal-status Critical Current

Links

Images

Classifications

    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/50—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
    • A61K47/51—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent
    • A61K47/62—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being a protein, peptide or polyamino acid
    • A61K47/64—Drug-peptide, drug-protein or drug-polyamino acid conjugates, i.e. the modifying agent being a peptide, protein or polyamino acid which is covalently bonded or complexed to a therapeutically active agent
    • A61K47/645—Polycationic or polyanionic oligopeptides, polypeptides or polyamino acids, e.g. polylysine, polyarginine, polyglutamic acid or peptide TAT
    • 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
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/14—Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/28—Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • 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
    • C12N2310/10—Type of nucleic acid
    • C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
    • 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
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/318—Chemical structure of the backbone where the PO2 is completely replaced, e.g. MMI or formacetal
    • C12N2310/3181—Peptide nucleic acid, PNA
    • 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
    • C12N2310/323—Chemical structure of the sugar modified ring structure
    • C12N2310/3231—Chemical structure of the sugar modified ring structure having an additional ring, e.g. LNA, ENA

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Chemical & Material Sciences (AREA)
  • Biomedical Technology (AREA)
  • General Health & Medical Sciences (AREA)
  • Organic Chemistry (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Genetics & Genomics (AREA)
  • Molecular Biology (AREA)
  • Neurosurgery (AREA)
  • General Chemical & Material Sciences (AREA)
  • Neurology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Biotechnology (AREA)
  • Epidemiology (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Biochemistry (AREA)
  • Physics & Mathematics (AREA)
  • Psychiatry (AREA)
  • Psychology (AREA)
  • Hospice & Palliative Care (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicinal Preparation (AREA)
JP2011521239A 2008-07-29 2009-07-28 ポリグルタミンタンパク質発現の選択的阻害 Withdrawn JP2011529686A (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US8435008P 2008-07-29 2008-07-29
US61/084,350 2008-07-29
PCT/US2009/051938 WO2010014592A1 (en) 2008-07-29 2009-07-28 Selective inhibition of polyglutamine protein expression

Related Child Applications (1)

Application Number Title Priority Date Filing Date
JP2015098107A Division JP6265940B2 (ja) 2008-07-29 2015-05-13 ポリグルタミンタンパク質発現の選択的阻害

Publications (2)

Publication Number Publication Date
JP2011529686A true JP2011529686A (ja) 2011-12-15
JP2011529686A5 JP2011529686A5 (https=) 2012-09-13

Family

ID=41610691

Family Applications (2)

Application Number Title Priority Date Filing Date
JP2011521239A Withdrawn JP2011529686A (ja) 2008-07-29 2009-07-28 ポリグルタミンタンパク質発現の選択的阻害
JP2015098107A Active JP6265940B2 (ja) 2008-07-29 2015-05-13 ポリグルタミンタンパク質発現の選択的阻害

Family Applications After (1)

Application Number Title Priority Date Filing Date
JP2015098107A Active JP6265940B2 (ja) 2008-07-29 2015-05-13 ポリグルタミンタンパク質発現の選択的阻害

Country Status (6)

Country Link
US (2) US8901095B2 (https=)
EP (1) EP2317847B1 (https=)
JP (2) JP2011529686A (https=)
AU (3) AU2009276763B2 (https=)
CA (1) CA2732343C (https=)
WO (1) WO2010014592A1 (https=)

Cited By (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2017510271A (ja) * 2014-03-19 2017-04-13 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Ｉｏｎｉｓ Ｐｈａｒｍａｃｅｕｔｉｃａｌｓ，Ｉｎｃ． アタキシン２の発現を調節するための組成物
JP2023520405A (ja) * 2020-03-30 2023-05-17 ニューベース セラピューティクス， インコーポレイテッド 改変ペプチド核酸組成物

Families Citing this family (34)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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
ES2639852T3 (es) 2007-10-26 2017-10-30 Academisch Ziekenhuis Leiden Medios y métodos para contrarrestar los trastornos musculares
WO2011097388A1 (en) * 2010-02-03 2011-08-11 Alnylam Pharmaceuticals, Inc. Selective inhibition of polyglutamine protein expression
JP2013518603A (ja) * 2010-02-08 2013-05-23 アイシス ファーマシューティカルズ， インコーポレーテッド 反復伸張に関連する疾患または病態の治療に有用な方法および組成物
EP2595663A4 (en) * 2010-07-19 2014-03-05 Isis Pharmaceuticals Inc MODULATION OF DYSTROPHIA MYOTONICA PROTEIN KINASE (DMPK) EXPRESSION
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
WO2013033223A1 (en) 2011-08-29 2013-03-07 Isis Pharmaceuticals, Inc. Methods and compounds useful in conditions related to repeat expansion
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
US20150315595A1 (en) * 2012-03-12 2015-11-05 Santaris Pharma A/S Compositions and Methods for Modulation of ATXN3 Expression
LT2841578T (lt) 2012-04-23 2017-09-25 Biomarin Technologies B.V. Rnr moduliuojantys oligonukleotidai su pagerintomis savybėmis, skirti neuromuskulinių susirgimų gydymui
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
EA201492116A1 (ru) 2012-05-16 2015-05-29 Рана Терапьютикс, Инк. Композиции и способы для модулирования экспрессии mecp2
EA201492122A1 (ru) 2012-05-16 2015-10-30 Рана Терапьютикс, Инк. Композиции и способы для модулирования экспрессии utrn
BR112014028631A2 (pt) 2012-05-16 2017-10-17 Rana Therapeutics Inc composições e métodos para modulação da expressão da família de genes da hemoglobina
US10435430B2 (en) 2013-07-31 2019-10-08 Ionis Pharmaceuticals, Inc. Methods and compounds useful in conditions related to repeat expansion
TW201536329A (zh) 2013-08-09 2015-10-01 Isis Pharmaceuticals Inc 用於調節失養性肌強直蛋白質激酶(dmpk)表現之化合物及方法
US10006027B2 (en) 2014-03-19 2018-06-26 Ionis Pharmaceuticals, Inc. Methods for modulating Ataxin 2 expression
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)
EP4285912A3 (en) * 2015-09-25 2024-07-10 Ionis Pharmaceuticals, Inc. Compositions and methods for modulating ataxin 3 expression
EP3368089B1 (en) 2015-10-26 2025-11-05 Translate Bio Ma, Inc. Nanoparticle formulations for delivery of nucleic acid complexes
AU2016381174A1 (en) 2015-12-31 2018-05-31 Ionis Pharmaceuticals, Inc. Methods for reducing Ataxin-2 expression
JP6430999B2 (ja) * 2016-06-24 2018-11-28 トヨタ自動車株式会社 自動車用エンジン冷却液組成物、自動車用エンジン濃縮冷却液組成物及び内燃機関の運転方法
JOP20190104A1 (ar) 2016-11-10 2019-05-07 Ionis Pharmaceuticals Inc مركبات وطرق لتقليل التعبير عن atxn3
EP4663758A3 (en) 2018-05-09 2026-04-29 Ionis Pharmaceuticals, Inc. Compounds and methods for reducing atxn3 expression
JP7379387B2 (ja) 2018-06-05 2023-11-14 エフ． ホフマン－ラ ロシュ アーゲー Ａｔｘｎ２発現を制御するためのオリゴヌクレオチド
AR115847A1 (es) 2018-07-25 2021-03-03 Ionis Pharmaceuticals Inc Compuestos y métodos para reducir la expresión de la atxn2
US11286485B2 (en) 2019-04-04 2022-03-29 Hoffmann-La Roche Inc. Oligonucleotides for modulating ATXN2 expression
EP4314296A2 (en) 2021-03-29 2024-02-07 Alnylam Pharmaceuticals, Inc. Huntingtin (htt) irna agent compositions and methods of use thereof
WO2023034870A2 (en) 2021-09-01 2023-03-09 Ionis Pharmaceuticals, Inc. Compounds and methods for reducing dmpk expression

Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2007022470A2 (en) * 2005-08-18 2007-02-22 Alnylam Pharmaceuticals, Inc. Methods and compositions for treating neurological disease
WO2008018795A1 (en) * 2006-08-11 2008-02-14 Prosensa Technologies B.V. Methods and means for treating dna repeat instability associated genetic disorders

Family Cites Families (81)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US4529561A (en) 1978-03-24 1985-07-16 The Regents Of The University Of California Method for producing liposomes in selected size range
US5118800A (en) 1983-12-20 1992-06-02 California Institute Of Technology Oligonucleotides possessing a primary amino group in the terminal nucleotide
FR2567892B1 (fr) 1984-07-19 1989-02-17 Centre Nat Rech Scient Nouveaux oligonucleotides, leur procede de preparation et leurs applications comme mediateurs dans le developpement des effets des interferons
US4737323A (en) 1986-02-13 1988-04-12 Liposome Technology, Inc. Liposome extrusion method
US5591722A (en) 1989-09-15 1997-01-07 Southern Research Institute 2'-deoxy-4'-thioribonucleosides and their antiviral activity
DE69034150T2 (de) 1989-10-24 2005-08-25 Isis Pharmaceuticals, Inc., Carlsbad 2'-Modifizierte Oligonukleotide
US6753423B1 (en) 1990-01-11 2004-06-22 Isis Pharmaceuticals, Inc. Compositions and methods for enhanced biostability and altered biodistribution of oligonucleotides in mammals
US6005087A (en) 1995-06-06 1999-12-21 Isis Pharmaceuticals, Inc. 2'-modified oligonucleotides
US5646265A (en) 1990-01-11 1997-07-08 Isis Pharmceuticals, Inc. Process for the preparation of 2'-O-alkyl purine phosphoramidites
US5670633A (en) 1990-01-11 1997-09-23 Isis Pharmaceuticals, Inc. Sugar modified oligonucleotides that detect and modulate gene expression
GB9009980D0 (en) 1990-05-03 1990-06-27 Amersham Int Plc Phosphoramidite derivatives,their preparation and the use thereof in the incorporation of reporter groups on synthetic oligonucleotides
ES2116977T3 (es) 1990-05-11 1998-08-01 Microprobe Corp Soportes solidos para ensayos de hibridacion de acidos nucleicos y metodos para inmovilizar oligonucleotidos de modo covalente.
US6582908B2 (en) * 1990-12-06 2003-06-24 Affymetrix, Inc. Oligonucleotides
EP0538194B1 (de) 1991-10-17 1997-06-04 Novartis AG Bicyclische Nukleoside, Oligonukleotide, Verfahren zu deren Herstellung und Zwischenprodukte
US5359044A (en) 1991-12-13 1994-10-25 Isis Pharmaceuticals Cyclobutyl oligonucleotide surrogates
US5700922A (en) * 1991-12-24 1997-12-23 Isis Pharmaceuticals, Inc. PNA-DNA-PNA chimeric macromolecules
FR2687679B1 (fr) 1992-02-05 1994-10-28 Centre Nat Rech Scient Oligothionucleotides.
EP0577558A2 (de) 1992-07-01 1994-01-05 Ciba-Geigy Ag Carbocyclische Nukleoside mit bicyclischen Ringen, Oligonukleotide daraus, Verfahren zu deren Herstellung, deren Verwendung und Zwischenproduckte
JPH08504559A (ja) 1992-12-14 1996-05-14 ハネウエル・インコーポレーテッド 個別に制御される冗長巻線を有するモータシステム
CA2159631A1 (en) 1993-03-30 1994-10-13 Sanofi Acyclic nucleoside analogs and oligonucleotide sequences containing them
DE4311944A1 (de) 1993-04-10 1994-10-13 Degussa Umhüllte Natriumpercarbonatpartikel, Verfahren zu deren Herstellung und sie enthaltende Wasch-, Reinigungs- und Bleichmittelzusammensetzungen
FR2705099B1 (fr) 1993-05-12 1995-08-04 Centre Nat Rech Scient Oligonucléotides phosphorothioates triesters et procédé de préparation.
US5801154A (en) * 1993-10-18 1998-09-01 Isis Pharmaceuticals, Inc. Antisense oligonucleotide modulation of multidrug resistance-associated protein
US5446137B1 (en) 1993-12-09 1998-10-06 Behringwerke Ag Oligonucleotides containing 4'-substituted nucleotides
US5595756A (en) * 1993-12-22 1997-01-21 Inex Pharmaceuticals Corporation Liposomal compositions for enhanced retention of bioactive agents
US5519134A (en) 1994-01-11 1996-05-21 Isis Pharmaceuticals, Inc. Pyrrolidine-containing monomers and oligomers
US5627053A (en) 1994-03-29 1997-05-06 Ribozyme Pharmaceuticals, Inc. 2'deoxy-2'-alkylnucleotide containing nucleic acid
US5597909A (en) 1994-08-25 1997-01-28 Chiron Corporation Polynucleotide reagents containing modified deoxyribose moieties, and associated methods of synthesis and use
US5792747A (en) 1995-01-24 1998-08-11 The Administrators Of The Tulane Educational Fund Highly potent agonists of growth hormone releasing hormone
US20080119427A1 (en) 1996-06-06 2008-05-22 Isis Pharmaceuticals, Inc. Double Strand Compositions Comprising Differentially Modified Strands for Use in Gene Modulation
ES2192672T3 (es) * 1996-11-18 2003-10-16 Takeshi Imanishi Nuevos analogos de nucleotidos.
JP3756313B2 (ja) 1997-03-07 2006-03-15 武 今西 新規ビシクロヌクレオシド及びオリゴヌクレオチド類縁体
US6770748B2 (en) 1997-03-07 2004-08-03 Takeshi Imanishi Bicyclonucleoside and oligonucleotide analogue
CA2294988C (en) 1997-07-01 2015-11-24 Isis Pharmaceuticals Inc. Compositions and methods for the delivery of oligonucleotides via the alimentary canal
US6794499B2 (en) 1997-09-12 2004-09-21 Exiqon A/S Oligonucleotide analogues
JP4236812B2 (ja) 1997-09-12 2009-03-11 エクシコン エ／エス オリゴヌクレオチド類似体
US20030228597A1 (en) * 1998-04-13 2003-12-11 Cowsert Lex M. Identification of genetic targets for modulation by oligonucleotides and generation of oligonucleotides for gene modulation
US6043352A (en) 1998-08-07 2000-03-28 Isis Pharmaceuticals, Inc. 2'-O-Dimethylaminoethyloxyethyl-modified oligonucleotides
ES2234563T5 (es) 1999-02-12 2018-01-17 Daiichi Sankyo Company, Limited Nuevos análogos de nucleósidos y oligonucleótidos
US7084125B2 (en) 1999-03-18 2006-08-01 Exiqon A/S Xylo-LNA analogues
US5998148A (en) * 1999-04-08 1999-12-07 Isis Pharmaceuticals Inc. Antisense modulation of microtubule-associated protein 4 expression
US7053207B2 (en) 1999-05-04 2006-05-30 Exiqon A/S L-ribo-LNA analogues
US6525191B1 (en) 1999-05-11 2003-02-25 Kanda S. Ramasamy Conformationally constrained L-nucleosides
JP4151751B2 (ja) 1999-07-22 2008-09-17 第一三共株式会社 新規ビシクロヌクレオシド類縁体
US6147200A (en) * 1999-08-19 2000-11-14 Isis Pharmaceuticals, Inc. 2'-O-acetamido modified monomers and oligomers
US6632616B2 (en) * 2000-03-16 2003-10-14 Duke University Compounds that selectively bind to expanded polyglutamine repeat domains and methods of use thereof
AU4819101A (en) 2000-04-13 2001-10-30 University Of British Columbia, The Modulating cell survival by modulating huntingtin function
WO2002027033A1 (en) * 2000-09-29 2002-04-04 Isis Pharmaceuticals, Inc. Antisense modulation of mekk4 expression
US20050191638A1 (en) * 2002-02-20 2005-09-01 Sirna Therapeutics, Inc. RNA interference mediated treatment of polyglutamine (polyQ) repeat expansion diseases using short interfering nucleic acid (siNA)
US20040096880A1 (en) * 2001-08-07 2004-05-20 Kmiec Eric B. Compositions and methods for the treatment of diseases exhibiting protein misassembly and aggregation
WO2003013437A2 (en) 2001-08-07 2003-02-20 University Of Delaware Compositions and methods for the prevention and treatment of huntington's disease
ATE556714T1 (de) 2002-02-01 2012-05-15 Life Technologies Corp Doppelsträngige oligonukleotide
US20050096284A1 (en) * 2002-02-20 2005-05-05 Sirna Therapeutics, Inc. RNA interference mediated treatment of polyglutamine (polyQ) repeat expansion diseases using short interfering nucleic acid (siNA)
US7569575B2 (en) 2002-05-08 2009-08-04 Santaris Pharma A/S Synthesis of locked nucleic acid derivatives
US20040092465A1 (en) * 2002-11-11 2004-05-13 Isis Pharmaceuticals Inc. Modulation of huntingtin interacting protein 1 expression
US20040102398A1 (en) 2002-11-23 2004-05-27 Isis Pharmaceuticals Inc. Modulation of B7H expression
US20050255086A1 (en) * 2002-08-05 2005-11-17 Davidson Beverly L Nucleic acid silencing of Huntington's Disease gene
US20080274989A1 (en) * 2002-08-05 2008-11-06 University Of Iowa Research Foundation Rna Interference Suppression of Neurodegenerative Diseases and Methods of Use Thereof
US20050106731A1 (en) 2002-08-05 2005-05-19 Davidson Beverly L. siRNA-mediated gene silencing with viral vectors
US20050042646A1 (en) * 2002-08-05 2005-02-24 Davidson Beverly L. RNA interference suppresion of neurodegenerative diseases and methods of use thereof
EP1546344A4 (en) * 2002-09-18 2007-10-03 Isis Pharmaceuticals Inc EFFICIENT PRODUCTION OF TARGET RNAS USING SINGLE AND DOUBLE-STRONG OLIGOMER COMPOUNDS
US20040219565A1 (en) 2002-10-21 2004-11-04 Sakari Kauppinen Oligonucleotides useful for detecting and analyzing nucleic acids of interest
WO2004041889A2 (en) 2002-11-05 2004-05-21 Isis Pharmaceuticals, Inc. Polycyclic sugar surrogate-containing oligomeric compounds and compositions for use in gene modulation
US7250496B2 (en) * 2002-11-14 2007-07-31 Rosetta Genomics Ltd. Bioinformatically detectable group of novel regulatory genes and uses thereof
AU2003295600A1 (en) * 2002-11-14 2004-06-15 Dharmacon, Inc. Functional and hyperfunctional sirna
US7250289B2 (en) * 2002-11-20 2007-07-31 Affymetrix, Inc. Methods of genetic analysis of mouse
US7589189B2 (en) * 2003-05-14 2009-09-15 Japan Science And Technology Agency Inhibition of the expression of huntingtin gene
EP1661905B9 (en) 2003-08-28 2012-12-19 IMANISHI, Takeshi Novel artificial nucleic acids of n-o bond crosslinkage type
DK2821085T3 (da) 2003-09-12 2020-08-03 Univ Massachusetts Rna-interferens til behandling af "gain-of-function"-forstyrrelser
WO2005045032A2 (en) 2003-10-20 2005-05-19 Sima Therapeutics, Inc. RNA INTERFERENCE MEDIATED INHIBITION OF EARLY GROWTH RESPONSE GENE EXPRESSION USING SHORT INTERFERING NUCLEIC ACID (siNA)
WO2005105995A2 (en) 2004-04-14 2005-11-10 Sirna Therapeutics, Inc. RNA INTERFERENCE MEDIATED TREATMENT OF POLYGLUTAMINE (POLYQ) REPEAT EXPANSION DISEASES USING SHORT INTERFERING NUCLEIC ACID (siNA)
WO2005116204A1 (ja) 2004-05-11 2005-12-08 Rnai Co., Ltd. Ｒｎａ干渉を生じさせるポリヌクレオチド、および、これを用いた遺伝子発現抑制方法
AU2005247509C1 (en) 2004-05-27 2012-09-20 Alnylam Pharmaceuticals, Inc. Nuclease resistant double-stranded ribonucleic acid
US7323308B2 (en) * 2004-09-03 2008-01-29 Affymetrix, Inc. Methods of genetic analysis of E. coli
JP5111385B2 (ja) * 2005-10-28 2013-01-09 アルナイラム ファーマシューティカルズ， インコーポレイテッド ハンチンチン遺伝子の発現を抑制するための組成物および方法
WO2007086990A2 (en) * 2005-11-17 2007-08-02 Board Of Regents, The University Of Texas System Modulation of gene expression by oligomers targeted to chromosomal dna
EP1991677A2 (en) * 2006-01-26 2008-11-19 Isis Pharmaceuticals, Inc. Compositions and their uses directed to huntingtin
JP2009536222A (ja) 2006-05-05 2009-10-08 アイシス ファーマシューティカルズ， インコーポレーテッド Ｐｃｓｋ９の発現を調節するための化合物および方法
KR101654007B1 (ko) * 2007-08-15 2016-09-05 아이오니스 파마수티컬즈, 인코포레이티드 테트라하이드로피란 핵산 유사체
NZ714887A (en) * 2009-09-11 2019-07-26 Ionis Pharmaceuticals Inc Modulation of huntingtin expression
WO2011097388A1 (en) 2010-02-03 2011-08-11 Alnylam Pharmaceuticals, Inc. Selective inhibition of polyglutamine protein expression

Patent Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2007022470A2 (en) * 2005-08-18 2007-02-22 Alnylam Pharmaceuticals, Inc. Methods and compositions for treating neurological disease
WO2008018795A1 (en) * 2006-08-11 2008-02-14 Prosensa Technologies B.V. Methods and means for treating dna repeat instability associated genetic disorders

Non-Patent Citations (5)

* Cited by examiner, † Cited by third party
Title
JPN6014005919; ＰＮＡＳ Ｖｏｌ．１０４, 2007, ｐ．１７２０４-１７２０９ *
JPN6014005921; Ｇｅｎｅ　Ｔｈｅｒａｐｙ Ｖｏｌ．１３, 2006, ｐ．５２５-５３１ *
JPN6014005923; ＰＬｏＳ Ｖｏｌ．２, 2006, ｐ．ｅ１４０ *
JPN6015000921; ＮＡＴＵＲＥ　ＢＩＯＴＥＣＨＮＯＬＯＧＹ ２７　（５）, 200905, ｐ．４７８-４８４ *
JPN6015000924; Ｊ．　Ａｍ．　Ｃｈｅｍ．　Ｓｏｃ． １３０, 20080830, ｐ．１２５７４-１２５７５ *

Cited By (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2017510271A (ja) * 2014-03-19 2017-04-13 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Ｉｏｎｉｓ Ｐｈａｒｍａｃｅｕｔｉｃａｌｓ，Ｉｎｃ． アタキシン２の発現を調節するための組成物
JP2021104035A (ja) * 2014-03-19 2021-07-26 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Ｉｏｎｉｓ Ｐｈａｒｍａｃｅｕｔｉｃａｌｓ，Ｉｎｃ． アタキシン２の発現を調節するための組成物
JP7194222B2 (ja) 2014-03-19 2022-12-21 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 アタキシン２の発現を調節するための組成物
JP2023027236A (ja) * 2014-03-19 2023-03-01 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 アタキシン２の発現を調節するための組成物
JP2023520405A (ja) * 2020-03-30 2023-05-17 ニューベース セラピューティクス， インコーポレイテッド 改変ペプチド核酸組成物
JP7796038B2 (ja) 2020-03-30 2026-01-08 ニューベース セラピューティクス， インコーポレイテッド 改変ペプチド核酸組成物

Also Published As

Publication number Publication date
JP6265940B2 (ja) 2018-01-24
AU2009276763A1 (en) 2010-02-04
AU2015238876A1 (en) 2015-10-29
AU2017216467A1 (en) 2017-08-31
US9340785B2 (en) 2016-05-17
US20150211007A1 (en) 2015-07-30
EP2317847B1 (en) 2019-04-17
EP2317847A4 (en) 2014-10-15
CA2732343C (en) 2017-05-09
CA2732343A1 (en) 2010-02-04
US8901095B2 (en) 2014-12-02
AU2009276763B2 (en) 2015-07-16
EP2317847A1 (en) 2011-05-11
US20110190222A1 (en) 2011-08-04
JP2015171368A (ja) 2015-10-01
WO2010014592A1 (en) 2010-02-04

Similar Documents

Publication Publication Date Title
JP6265940B2 (ja) ポリグルタミンタンパク質発現の選択的阻害
US9574191B2 (en) Selective inhibition of polyglutamine protein expression
JP6513135B2 (ja) トランスサイレチン（ＴＴＲ）関連疾患を治療するためのＲＮＡｉ剤、組成物、およびこれらの使用方法
JP5934408B2 (ja) Ｄｎａリピートの不安定性に関連した遺伝的障害を治療するための方法及び手段
CN103747805B (zh) 用于治疗、延缓和/或预防人类遗传性疾病如强直性肌营养不良症1型（dm1）的化合物
TWI736514B (zh) 酸性α葡萄糖苷酶之反義股誘導之外顯子２包含
CN114375194B (zh) 血管生成素样7（angptl7）相关疾病的治疗
US20240141348A1 (en) Allele selective inhibition of mutant c9orf72 foci expression by duplex rnas targeting the expanded hexanucleotide repeat
US20140128449A1 (en) Oligonucleotide modulation of splicing
KR20240036132A (ko) 대상에게서 ｓｍｎ２ 스플라이싱을 조정하기 위한 조성물 및 방법
JP2011510678A (ja) Ｄｎａ反復不安定性関連遺伝性障害を治療するための方法及び手段
US20240229029A9 (en) Compositions and methods for treatment of friedreich's ataxia
JP2023543494A (ja) 視神経萎縮症の治療
US12612630B2 (en) Compositions and methods for splicing modulation of UNC13A
US20250297249A1 (en) Antisense oligonucleotide complex
WO2024005158A1 (ja) オリゴヌクレオチド
JP2025537104A (ja) アンチセンスオリゴマーコンジュゲートの製剤化

Legal Events

Date Code Title Description
A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20120726

A621 Written request for application examination

Free format text: JAPANESE INTERMEDIATE CODE: A621

Effective date: 20120726

A131 Notification of reasons for refusal

Free format text: JAPANESE INTERMEDIATE CODE: A131

Effective date: 20140212

A601 Written request for extension of time

Free format text: JAPANESE INTERMEDIATE CODE: A601

Effective date: 20140509

A602 Written permission of extension of time

Free format text: JAPANESE INTERMEDIATE CODE: A602

Effective date: 20140516

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20140606

A02 Decision of refusal

Free format text: JAPANESE INTERMEDIATE CODE: A02

Effective date: 20150114

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20150513

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20150525

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20150616

A911 Transfer to examiner for re-examination before appeal (zenchi)

Free format text: JAPANESE INTERMEDIATE CODE: A911

Effective date: 20150622

A912 Re-examination (zenchi) completed and case transferred to appeal board

Free format text: JAPANESE INTERMEDIATE CODE: A912

Effective date: 20150807

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20160812

A761 Written withdrawal of application

Free format text: JAPANESE INTERMEDIATE CODE: A761

Effective date: 20161019