SG11201407483YA - Compositions and methods for modulating smn gene family expression - Google Patents

Compositions and methods for modulating smn gene family expression

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Publication number
SG11201407483YA
SG11201407483YA SG11201407483YA SG11201407483YA SG11201407483YA SG 11201407483Y A SG11201407483Y A SG 11201407483YA SG 11201407483Y A SG11201407483Y A SG 11201407483YA SG 11201407483Y A SG11201407483Y A SG 11201407483YA SG 11201407483Y A SG11201407483Y A SG 11201407483YA
Authority
SG
Singapore
Prior art keywords
international
smnl
smn2
methods
street
Prior art date
Application number
SG11201407483YA
Other languages
English (en)
Inventor
Arthur M Krieg
Romesh Subramanian
James Mcswiggen
Jeannie T Lee
Original Assignee
Rana Therapeutics Inc
Gen Hospital Corp
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 Rana Therapeutics Inc, Gen Hospital Corp filed Critical Rana Therapeutics Inc
Publication of SG11201407483YA publication Critical patent/SG11201407483YA/en

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    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113Non-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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/70Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088Compounds having three or more nucleosides or nucleotides
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00Drugs for disorders of the muscular or neuromuscular system
    • A61P21/02Muscle relaxants, e.g. for tetanus or cramps
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    • 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
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    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113Non-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
    • C12N15/1131Non-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 against viruses
    • CCHEMISTRY; METALLURGY
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    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113Non-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
    • C12N15/1131Non-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 against viruses
    • C12N15/1132Non-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 against viruses against retroviridae, e.g. HIV
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/11Antisense
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/31Chemical structure of the backbone
    • C12N2310/315Phosphorothioates
    • CCHEMISTRY; METALLURGY
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/32Chemical structure of the sugar
    • C12N2310/323Chemical structure of the sugar modified ring structure
    • C12N2310/3231Chemical structure of the sugar modified ring structure having an additional ring, e.g. LNA, ENA
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/34Spatial arrangement of the modifications
    • C12N2310/343Spatial arrangement of the modifications having patterns, e.g. ==--==--==--
    • CCHEMISTRY; METALLURGY
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/35Nature of the modification
    • C12N2310/351Conjugate
    • C12N2310/3517Marker; Tag

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  • Health & Medical Sciences (AREA)
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  • Chemical & Material Sciences (AREA)
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  • Genetics & Genomics (AREA)
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  • Organic Chemistry (AREA)
  • Biomedical Technology (AREA)
  • Biotechnology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biochemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Microbiology (AREA)
  • Medicinal Chemistry (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Epidemiology (AREA)
  • Virology (AREA)
  • Pain & Pain Management (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Neurology (AREA)
  • AIDS & HIV (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
  • Medicinal Preparation (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
SG11201407483YA 2012-05-16 2013-05-16 Compositions and methods for modulating smn gene family expression SG11201407483YA (en)

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
US201261647858P 2012-05-16 2012-05-16
US201261719394P 2012-10-27 2012-10-27
US201361785529P 2013-03-14 2013-03-14
PCT/US2013/041440 WO2013173638A1 (en) 2012-05-16 2013-05-16 Compositions and methods for modulating smn gene family expression

Publications (1)

Publication Number Publication Date
SG11201407483YA true SG11201407483YA (en) 2014-12-30

Family

ID=49584305

Family Applications (1)

Application Number Title Priority Date Filing Date
SG11201407483YA SG11201407483YA (en) 2012-05-16 2013-05-16 Compositions and methods for modulating smn gene family expression

Country Status (13)

Country Link
US (1) US10059941B2 (ja)
EP (1) EP2850186B1 (ja)
JP (1) JP2015523854A (ja)
KR (1) KR20150030205A (ja)
CN (1) CN104540947A (ja)
AU (1) AU2013262649A1 (ja)
CA (1) CA2873794A1 (ja)
DK (1) DK2850186T3 (ja)
EA (1) EA201492123A1 (ja)
HK (1) HK1208700A1 (ja)
SG (1) SG11201407483YA (ja)
WO (1) WO2013173638A1 (ja)
ZA (1) ZA201409228B (ja)

Families Citing this family (37)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US9920317B2 (en) 2010-11-12 2018-03-20 The General Hospital Corporation Polycomb-associated non-coding RNAs
EP3702460A1 (en) 2010-11-12 2020-09-02 The General Hospital Corporation Polycomb-associated non-coding rnas
EP2850190B1 (en) 2012-05-16 2020-07-08 Translate Bio MA, Inc. Compositions and methods for modulating mecp2 expression
WO2013173598A1 (en) 2012-05-16 2013-11-21 Rana Therapeutics, Inc. Compositions and methods for modulating atp2a2 expression
DK2850186T3 (en) 2012-05-16 2019-04-08 Translate Bio Ma Inc COMPOSITIONS AND PROCEDURES FOR MODULATING SMN GENFAMILY EXPRESSION
US10174315B2 (en) 2012-05-16 2019-01-08 The General Hospital Corporation Compositions and methods for modulating hemoglobin gene family expression
WO2013173645A1 (en) 2012-05-16 2013-11-21 Rana Therapeutics, Inc. Compositions and methods for modulating utrn expression
US10837014B2 (en) 2012-05-16 2020-11-17 Translate Bio Ma, Inc. Compositions and methods for modulating SMN gene family expression
US9885040B2 (en) * 2013-04-12 2018-02-06 The Curators Of The University Of Missouri SMN2 element 1 antisense compositions and methods and uses thereof
US10174328B2 (en) 2013-10-04 2019-01-08 Translate Bio Ma, Inc. Compositions and methods for treating amyotrophic lateral sclerosis
CN103911346B (zh) * 2014-03-27 2016-09-07 江苏雄鸣医药科技有限公司 一种用于治疗脊髓性肌萎缩症药物筛选的细胞模型
WO2016040748A1 (en) 2014-09-12 2016-03-17 Ionis Pharmaceuticals, Inc. Compositions and methods for detection of smn protein in a subject and treatment of a subject
CA2966044A1 (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)
US10851371B2 (en) 2015-04-10 2020-12-01 Ionis Pharmaceuticals, Inc. Modulation of SMN expression
US11249071B2 (en) 2015-04-24 2022-02-15 California Institute Of Technology Reactivation of x chromosome genes
CA2996164A1 (en) * 2015-08-28 2017-03-09 Sarepta Therapeutics, Inc. Modified antisense oligomers for exon inclusion in spinal muscular atrophy
EP3368671A1 (en) * 2015-10-26 2018-09-05 Translate Bio Ma, Inc. Methods and compositions for increasing smn expression
CA3005434A1 (en) 2015-11-16 2017-05-26 Ohio State Innovation Foundation Methods and compositions for treating disorders and diseases using survival motor neuron (smn) protein
US11198867B2 (en) 2016-06-16 2021-12-14 Ionis Pharmaceuticals, Inc. Combinations for the modulation of SMN expression
JP2019537427A (ja) 2016-10-27 2019-12-26 カリフォルニア インスティチュート オブ テクノロジー X染色体の再活性化のためのhdac阻害剤組成物
JP7130639B2 (ja) 2016-11-01 2022-09-05 ザ・リサーチ・ファウンデーション・フォー・ザ・ステイト・ユニヴァーシティ・オブ・ニューヨーク 5-ハロウラシル修飾マイクロrna及びがんの処置におけるその使用
AU2017368050A1 (en) 2016-11-29 2019-06-20 Puretech Lyt, Inc. Exosomes for delivery of therapeutic agents
EP3599844A4 (en) * 2017-06-07 2021-01-13 University of Massachusetts ANTI-ADAM33 OLIGONUCLEOTIDES AND RELATED PROCESSES
US20200208152A1 (en) 2017-09-08 2020-07-02 Mina Therapeutics Limited Stabilized sarna compositions and methods of use
EP3679138B1 (en) 2017-09-08 2023-03-22 MiNA Therapeutics Limited Hnf4a sarna compositions and methods of use
EP3694530A4 (en) * 2017-10-12 2021-06-30 Wave Life Sciences Ltd. OLIGONUCLEOTIDE COMPOSITIONS AND METHOD FOR THEREFORE
US11185079B2 (en) 2018-09-26 2021-11-30 Greenlight Biosciences, Inc. Control of Coleopteran insects
US11142768B2 (en) 2018-11-08 2021-10-12 Greenlight Biosciences, Inc. Control of insect infestation
BR112021013612A2 (pt) * 2019-01-09 2021-09-28 Coyote Bioscience Usa Inc. Métodos e sistemas para a identificação de atrofia muscular espinhal
US20220280548A1 (en) * 2019-08-15 2022-09-08 Biogen Ma Inc. Combination therapy for spinal muscular atrophy
EP4017539A1 (en) 2019-08-19 2022-06-29 MiNA Therapeutics Limited Oligonucleotide conjugate compositions and methods of use
MX2022010602A (es) 2020-02-28 2022-09-09 Ionis Pharmaceuticals Inc Compuestos y metodos para modular smn2.
CN113444722B (zh) * 2020-03-24 2024-09-24 中国科学院脑科学与智能技术卓越创新中心 单碱基编辑介导的剪接修复在制备治疗脊髓性肌萎缩症中的应用
WO2022178273A1 (en) * 2021-02-19 2022-08-25 University Of Florida Research Foundation, Incorporated Methods and compositions to confer regulation to gene therapy cargoes by heterologous use of alternative splicing cassettes
WO2023020421A1 (en) * 2021-08-16 2023-02-23 Hangzhou Jiayin Biotech Ltd. Regulation of imprinting silenced genes by smn1 and snrpn expression and uses thereof
CA3230274A1 (en) * 2021-09-03 2023-03-09 Alfica Sehgal Modulation of gene transcription using antisense oligonucleotides targeting regulatory rnas

Family Cites Families (400)

* 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
US4469863A (en) 1980-11-12 1984-09-04 Ts O Paul O P Nonionic nucleic acid alkyl and aryl phosphonates and processes for manufacture and use thereof
DE3280400D1 (de) 1981-10-23 1992-06-04 Molecular Biosystems Inc Oligonukleotides heilmittel und dessen herstellungsverfahren.
US5023243A (en) 1981-10-23 1991-06-11 Molecular Biosystems, Inc. Oligonucleotide therapeutic agent and method of making same
US4476301A (en) 1982-04-29 1984-10-09 Centre National De La Recherche Scientifique Oligonucleotides, a process for preparing the same and their application as mediators of the action of interferon
JPS5927900A (ja) 1982-08-09 1984-02-14 Wakunaga Seiyaku Kk 固定化オリゴヌクレオチド
FR2540122B1 (fr) 1983-01-27 1985-11-29 Centre Nat Rech Scient Nouveaux composes comportant une sequence d'oligonucleotide liee a un agent d'intercalation, leur procede de synthese et leur application
US4605735A (en) 1983-02-14 1986-08-12 Wakunaga Seiyaku Kabushiki Kaisha Oligonucleotide derivatives
US4948882A (en) 1983-02-22 1990-08-14 Syngene, Inc. Single-stranded labelled oligonucleotides, reactive monomers and methods of synthesis
US4824941A (en) 1983-03-10 1989-04-25 Julian Gordon Specific antibody to the native form of 2'5'-oligonucleotides, the method of preparation and the use as reagents in immunoassays or for binding 2'5'-oligonucleotides in biological systems
DE3329892A1 (de) 1983-08-18 1985-03-07 Köster, Hubert, Prof. Dr., 2000 Hamburg Verfahren zur herstellung von oligonucleotiden
US4587044A (en) 1983-09-01 1986-05-06 The Johns Hopkins University Linkage of proteins to nucleic acids
US5118802A (en) 1983-12-20 1992-06-02 California Institute Of Technology DNA-reporter conjugates linked via the 2' or 5'-primary amino group of the 5'-terminal nucleoside
US5550111A (en) 1984-07-11 1996-08-27 Temple University-Of The Commonwealth System Of Higher Education Dual action 2',5'-oligoadenylate antiviral derivatives and uses thereof
US5430136A (en) 1984-10-16 1995-07-04 Chiron Corporation Oligonucleotides having selectably cleavable and/or abasic sites
US5258506A (en) 1984-10-16 1993-11-02 Chiron Corporation Photolabile reagents for incorporation into oligonucleotide chains
US5367066A (en) 1984-10-16 1994-11-22 Chiron Corporation Oligonucleotides with selectably cleavable and/or abasic sites
US4828979A (en) 1984-11-08 1989-05-09 Life Technologies, Inc. Nucleotide analogs for nucleic acid labeling and detection
FR2575751B1 (fr) 1985-01-08 1987-04-03 Pasteur Institut Nouveaux nucleosides de derives de l'adenosine, leur preparation et leurs applications biologiques
US5235033A (en) 1985-03-15 1993-08-10 Anti-Gene Development Group Alpha-morpholino ribonucleoside derivatives and polymers thereof
US5166315A (en) 1989-12-20 1992-11-24 Anti-Gene Development Group Sequence-specific binding polymers for duplex nucleic acids
US5185444A (en) 1985-03-15 1993-02-09 Anti-Gene Deveopment Group Uncharged morpolino-based polymers having phosphorous containing chiral intersubunit linkages
US5405938A (en) 1989-12-20 1995-04-11 Anti-Gene Development Group Sequence-specific binding polymers for duplex nucleic acids
US5034506A (en) 1985-03-15 1991-07-23 Anti-Gene Development Group Uncharged morpholino-based polymers having achiral intersubunit linkages
US4762779A (en) 1985-06-13 1988-08-09 Amgen Inc. Compositions and methods for functionalizing nucleic acids
US5317098A (en) 1986-03-17 1994-05-31 Hiroaki Shizuya Non-radioisotope tagging of fragments
JPS638396A (ja) 1986-06-30 1988-01-14 Wakunaga Pharmaceut Co Ltd ポリ標識化オリゴヌクレオチド誘導体
DE3788914T2 (de) 1986-09-08 1994-08-25 Ajinomoto Kk Verbindungen zur Spaltung von RNS an eine spezifische Position, Oligomere, verwendet bei der Herstellung dieser Verbindungen und Ausgangsprodukte für die Synthese dieser Oligomere.
US5264423A (en) 1987-03-25 1993-11-23 The United States Of America As Represented By The Department Of Health And Human Services Inhibitors for replication of retroviruses and for the expression of oncogene products
US5276019A (en) 1987-03-25 1994-01-04 The United States Of America As Represented By The Department Of Health And Human Services Inhibitors for replication of retroviruses and for the expression of oncogene products
US4904582A (en) 1987-06-11 1990-02-27 Synthetic Genetics Novel amphiphilic nucleic acid conjugates
JP2828642B2 (ja) 1987-06-24 1998-11-25 ハワード フローレイ インスティテュト オブ イクスペリメンタル フィジオロジー アンド メディシン ヌクレオシド誘導体
US5585481A (en) 1987-09-21 1996-12-17 Gen-Probe Incorporated Linking reagents for nucleotide probes
US4924624A (en) 1987-10-22 1990-05-15 Temple University-Of The Commonwealth System Of Higher Education 2,',5'-phosphorothioate oligoadenylates and plant antiviral uses thereof
US5188897A (en) 1987-10-22 1993-02-23 Temple University Of The Commonwealth System Of Higher Education Encapsulated 2',5'-phosphorothioate oligoadenylates
US5525465A (en) 1987-10-28 1996-06-11 Howard Florey Institute Of Experimental Physiology And Medicine Oligonucleotide-polyamide conjugates and methods of production and applications of the same
DE3738460A1 (de) 1987-11-12 1989-05-24 Max Planck Gesellschaft Modifizierte oligonukleotide
WO1989005358A1 (en) 1987-11-30 1989-06-15 University Of Iowa Research Foundation Dna and rna molecules stabilized by modifications of the 3'-terminal phosphodiester linkage and their use as nucleic acid probes and as therapeutic agents to block the expression of specifically targeted genes
US5403711A (en) 1987-11-30 1995-04-04 University Of Iowa Research Foundation Nucleic acid hybridization and amplification method for detection of specific sequences in which a complementary labeled nucleic acid probe is cleaved
US5082830A (en) 1988-02-26 1992-01-21 Enzo Biochem, Inc. End labeled nucleotide probe
EP0406309A4 (en) 1988-03-25 1992-08-19 The University Of Virginia Alumni Patents Foundation Oligonucleotide n-alkylphosphoramidates
US5278302A (en) 1988-05-26 1994-01-11 University Patents, Inc. Polynucleotide phosphorodithioates
US5109124A (en) 1988-06-01 1992-04-28 Biogen, Inc. Nucleic acid probe linked to a label having a terminal cysteine
US5216141A (en) 1988-06-06 1993-06-01 Benner Steven A Oligonucleotide analogs containing sulfur linkages
US5175273A (en) 1988-07-01 1992-12-29 Genentech, Inc. Nucleic acid intercalating agents
US5262536A (en) 1988-09-15 1993-11-16 E. I. Du Pont De Nemours And Company Reagents for the preparation of 5'-tagged oligonucleotides
US5512439A (en) 1988-11-21 1996-04-30 Dynal As Oligonucleotide-linked magnetic particles and uses thereof
US5457183A (en) 1989-03-06 1995-10-10 Board Of Regents, The University Of Texas System Hydroxylated texaphyrins
US5599923A (en) 1989-03-06 1997-02-04 Board Of Regents, University Of Tx Texaphyrin metal complexes having improved functionalization
US5391723A (en) 1989-05-31 1995-02-21 Neorx Corporation Oligonucleotide conjugates
US5256775A (en) 1989-06-05 1993-10-26 Gilead Sciences, Inc. Exonuclease-resistant oligonucleotides
US4958013A (en) 1989-06-06 1990-09-18 Northwestern University Cholesteryl modified oligonucleotides
US5451463A (en) 1989-08-28 1995-09-19 Clontech Laboratories, Inc. Non-nucleoside 1,3-diol reagents for labeling synthetic oligonucleotides
US5134066A (en) 1989-08-29 1992-07-28 Monsanto Company Improved probes using nucleosides containing 3-dezauracil analogs
US5254469A (en) 1989-09-12 1993-10-19 Eastman Kodak Company Oligonucleotide-enzyme conjugate that can be used as a probe in hybridization assays and polymerase chain reaction procedures
US5399676A (en) 1989-10-23 1995-03-21 Gilead Sciences Oligonucleotides with inverted polarity
US5264562A (en) 1989-10-24 1993-11-23 Gilead Sciences, Inc. Oligonucleotide analogs with novel linkages
US5264564A (en) 1989-10-24 1993-11-23 Gilead Sciences Oligonucleotide analogs with novel linkages
US5292873A (en) 1989-11-29 1994-03-08 The Research Foundation Of State University Of New York Nucleic acids labeled with naphthoquinone probe
US5177198A (en) 1989-11-30 1993-01-05 University Of N.C. At Chapel Hill Process for preparing oligoribonucleoside and oligodeoxyribonucleoside boranophosphates
US5130302A (en) 1989-12-20 1992-07-14 Boron Bilogicals, Inc. Boronated nucleoside, nucleotide and oligonucleotide compounds, compositions and methods for using same
US5486603A (en) 1990-01-08 1996-01-23 Gilead Sciences, Inc. Oligonucleotide having enhanced binding affinity
US5623065A (en) 1990-08-13 1997-04-22 Isis Pharmaceuticals, Inc. Gapped 2' modified oligonucleotides
US6005087A (en) 1995-06-06 1999-12-21 Isis Pharmaceuticals, Inc. 2'-modified oligonucleotides
US6395492B1 (en) 1990-01-11 2002-05-28 Isis Pharmaceuticals, Inc. Derivatized oligonucleotides having improved uptake and other properties
US5578718A (en) 1990-01-11 1996-11-26 Isis Pharmaceuticals, Inc. Thiol-derivatized nucleosides
US5587361A (en) 1991-10-15 1996-12-24 Isis Pharmaceuticals, Inc. Oligonucleotides having phosphorothioate linkages of high chiral purity
US5914396A (en) 1990-01-11 1999-06-22 Isis Pharmaceuticals, Inc. 2'-O-modified nucleosides and phosphoramidites
US6753423B1 (en) 1990-01-11 2004-06-22 Isis Pharmaceuticals, Inc. Compositions and methods for enhanced biostability and altered biodistribution of oligonucleotides in mammals
US5681941A (en) 1990-01-11 1997-10-28 Isis Pharmaceuticals, Inc. Substituted purines and oligonucleotide cross-linking
US5587470A (en) 1990-01-11 1996-12-24 Isis Pharmaceuticals, Inc. 3-deazapurines
US5459255A (en) 1990-01-11 1995-10-17 Isis Pharmaceuticals, Inc. N-2 substituted purines
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
AU7579991A (en) 1990-02-20 1991-09-18 Gilead Sciences, Inc. Pseudonucleosides and pseudonucleotides and their polymers
US5214136A (en) 1990-02-20 1993-05-25 Gilead Sciences, Inc. Anthraquinone-derivatives oligonucleotides
US5321131A (en) 1990-03-08 1994-06-14 Hybridon, Inc. Site-specific functionalization of oligodeoxynucleotides for non-radioactive labelling
US5470967A (en) 1990-04-10 1995-11-28 The Dupont Merck Pharmaceutical Company Oligonucleotide analogs with sulfamate linkages
EP0455905B1 (en) 1990-05-11 1998-06-17 Microprobe Corporation Dipsticks for nucleic acid hybridization assays and methods for covalently immobilizing oligonucleotides
AU8196791A (en) 1990-06-27 1992-01-23 Trustees Of Columbia University In The City Of New York, The Methods for detecting spinal muscular atrophy type i and types ii/iii and marker therefor
US5688941A (en) 1990-07-27 1997-11-18 Isis Pharmaceuticals, Inc. Methods of making conjugated 4' desmethyl nucleoside analog compounds
US5602240A (en) 1990-07-27 1997-02-11 Ciba Geigy Ag. Backbone modified oligonucleotide analogs
US5677437A (en) 1990-07-27 1997-10-14 Isis Pharmaceuticals, Inc. Heteroatomic oligonucleoside linkages
US5138045A (en) 1990-07-27 1992-08-11 Isis Pharmaceuticals Polyamine conjugated oligonucleotides
US5541307A (en) 1990-07-27 1996-07-30 Isis Pharmaceuticals, Inc. Backbone modified oligonucleotide analogs and solid phase synthesis thereof
DE69126530T2 (de) 1990-07-27 1998-02-05 Isis Pharmaceutical, Inc., Carlsbad, Calif. Nuklease resistente, pyrimidin modifizierte oligonukleotide, die die gen-expression detektieren und modulieren
US5618704A (en) 1990-07-27 1997-04-08 Isis Pharmacueticals, Inc. Backbone-modified oligonucleotide analogs and preparation thereof through radical coupling
US5489677A (en) 1990-07-27 1996-02-06 Isis Pharmaceuticals, Inc. Oligonucleoside linkages containing adjacent oxygen and nitrogen atoms
US5610289A (en) 1990-07-27 1997-03-11 Isis Pharmaceuticals, Inc. Backbone modified oligonucleotide analogues
US5218105A (en) 1990-07-27 1993-06-08 Isis Pharmaceuticals Polyamine conjugated oligonucleotides
US5623070A (en) 1990-07-27 1997-04-22 Isis Pharmaceuticals, Inc. Heteroatomic oligonucleoside linkages
US5608046A (en) 1990-07-27 1997-03-04 Isis Pharmaceuticals, Inc. Conjugated 4'-desmethyl nucleoside analog compounds
PT98562B (pt) 1990-08-03 1999-01-29 Sanofi Sa Processo para a preparacao de composicoes que compreendem sequencias de nucleo-sidos com cerca de 6 a cerca de 200 bases resistentes a nucleases
US5245022A (en) 1990-08-03 1993-09-14 Sterling Drug, Inc. Exonuclease resistant terminally substituted oligonucleotides
US5177196A (en) 1990-08-16 1993-01-05 Microprobe Corporation Oligo (α-arabinofuranosyl nucleotides) and α-arabinofuranosyl precursors thereof
US5512667A (en) 1990-08-28 1996-04-30 Reed; Michael W. Trifunctional intermediates for preparing 3'-tailed oligonucleotides
US5214134A (en) 1990-09-12 1993-05-25 Sterling Winthrop Inc. Process of linking nucleosides with a siloxane bridge
US5561225A (en) 1990-09-19 1996-10-01 Southern Research Institute Polynucleotide analogs containing sulfonate and sulfonamide internucleoside linkages
EP0549686A4 (en) 1990-09-20 1995-01-18 Gilead Sciences Inc Modified internucleoside linkages
US5432272A (en) 1990-10-09 1995-07-11 Benner; Steven A. Method for incorporating into a DNA or RNA oligonucleotide using nucleotides bearing heterocyclic bases
DE69132510T2 (de) 1990-11-08 2001-05-03 Hybridon, Inc. Verbindung von mehrfachreportergruppen auf synthetischen oligonukleotiden
JP3514779B2 (ja) 1991-04-17 2004-03-31 株式会社アズウェル アポリポプロテインe遺伝子タイプの検査方法及びその検査に好適なプライマー及びプローブ
WO1994008003A1 (en) 1991-06-14 1994-04-14 Isis Pharmaceuticals, Inc. ANTISENSE OLIGONUCLEOTIDE INHIBITION OF THE ras GENE
US5965722A (en) 1991-05-21 1999-10-12 Isis Pharmaceuticals, Inc. Antisense inhibition of ras gene with chimeric and alternating oligonucleotides
US7015315B1 (en) 1991-12-24 2006-03-21 Isis Pharmaceuticals, Inc. Gapped oligonucleotides
US5719262A (en) 1993-11-22 1998-02-17 Buchardt, Deceased; Ole Peptide nucleic acids having amino acid side chains
US5539082A (en) 1993-04-26 1996-07-23 Nielsen; Peter E. Peptide nucleic acids
US5714331A (en) 1991-05-24 1998-02-03 Buchardt, Deceased; Ole Peptide nucleic acids having enhanced binding affinity, sequence specificity and solubility
US5371241A (en) 1991-07-19 1994-12-06 Pharmacia P-L Biochemicals Inc. Fluorescein labelled phosphoramidites
US5571799A (en) 1991-08-12 1996-11-05 Basco, Ltd. (2'-5') oligoadenylate analogues useful as inhibitors of host-v5.-graft response
US5661134A (en) 1991-10-15 1997-08-26 Isis Pharmaceuticals, Inc. Oligonucleotides for modulating Ha-ras or Ki-ras having phosphorothioate linkages of high chiral purity
US6831166B2 (en) 1992-10-23 2004-12-14 Isis Pharmaceuticals, Inc. Derivatized oligonucleotides having improved uptake and other properties
US8153602B1 (en) 1991-11-19 2012-04-10 Isis Pharmaceuticals, Inc. Composition and methods for the pulmonary delivery of nucleic acids
US5484908A (en) 1991-11-26 1996-01-16 Gilead Sciences, Inc. Oligonucleotides containing 5-propynyl pyrimidines
DE69233599T2 (de) 1991-12-24 2006-12-14 Isis Pharmaceuticals, Inc., Carlsbad Unterbrochene 2'-modifizierte Oligonukleotide
US5700922A (en) 1991-12-24 1997-12-23 Isis Pharmaceuticals, Inc. PNA-DNA-PNA chimeric macromolecules
US20060270624A1 (en) 1991-12-24 2006-11-30 Isis Pharmaceuticals, Inc. Gapped 2' modified oligonucleotides
US5595726A (en) 1992-01-21 1997-01-21 Pharmacyclics, Inc. Chromophore probe for detection of nucleic acid
US5565552A (en) 1992-01-21 1996-10-15 Pharmacyclics, Inc. Method of expanded porphyrin-oligonucleotide conjugate synthesis
US5633360A (en) 1992-04-14 1997-05-27 Gilead Sciences, Inc. Oligonucleotide analogs capable of passive cell membrane permeation
US5434257A (en) 1992-06-01 1995-07-18 Gilead Sciences, Inc. Binding compentent oligomers containing unsaturated 3',5' and 2',5' linkages
US5272250A (en) 1992-07-10 1993-12-21 Spielvogel Bernard F Boronated phosphoramidate compounds
US6346614B1 (en) 1992-07-23 2002-02-12 Hybridon, Inc. Hybrid oligonucleotide phosphorothioates
US5652355A (en) 1992-07-23 1997-07-29 Worcester Foundation For Experimental Biology Hybrid oligonucleotide phosphorothioates
TW244371B (ja) 1992-07-23 1995-04-01 Tri Clover Inc
ATE162198T1 (de) 1992-07-27 1998-01-15 Hybridon Inc Oligonukleotid alkylphosphonothiate
US5574142A (en) 1992-12-15 1996-11-12 Microprobe Corporation Peptide linkers for improved oligonucleotide delivery
EP0677056B1 (en) 1993-01-25 1996-05-22 HYBRIDON, Inc. Oligonucleotide alkylphosphonates and alkylphosphonothioates
US5476925A (en) 1993-02-01 1995-12-19 Northwestern University Oligodeoxyribonucleotides including 3'-aminonucleoside-phosphoramidate linkages and terminal 3'-amino groups
GB9304618D0 (en) 1993-03-06 1993-04-21 Ciba Geigy Ag Chemical compounds
CA2159629A1 (en) 1993-03-31 1994-10-13 Sanofi Oligonucleotides with amide linkages replacing phosphodiester linkages
PT698092E (pt) 1993-05-11 2007-10-29 Univ North Carolina Oligonucleótidos complementares de uma cadeia codificadora que combatem splicing aberrante e métodos para a sua utilização
US5502177A (en) 1993-09-17 1996-03-26 Gilead Sciences, Inc. Pyrimidine derivatives for labeled binding partners
US5457187A (en) 1993-12-08 1995-10-10 Board Of Regents University Of Nebraska Oligonucleotides containing 5-fluorouracil
EP0733059B1 (en) 1993-12-09 2000-09-13 Thomas Jefferson University Compounds and methods for site-directed mutations in eukaryotic cells
US5596091A (en) 1994-03-18 1997-01-21 The Regents Of The University Of California Antisense oligonucleotides comprising 5-aminoalkyl pyrimidine nucleotides
US5625050A (en) 1994-03-31 1997-04-29 Amgen Inc. Modified oligonucleotides and intermediates useful in nucleic acid therapeutics
US5525711A (en) 1994-05-18 1996-06-11 The United States Of America As Represented By The Secretary Of The Department Of Health And Human Services Pteridine nucleotide analogs as fluorescent DNA probes
FR2720757B1 (fr) 1994-06-03 1996-08-02 Inst Nat Sante Rech Med Procédé et sondes pour la détection de marqueurs liés au locus des amyotrophies spinales infantiles.
US5597696A (en) 1994-07-18 1997-01-28 Becton Dickinson And Company Covalent cyanine dye oligonucleotide conjugates
US5580731A (en) 1994-08-25 1996-12-03 Chiron Corporation N-4 modified pyrimidine deoxynucleotides and oligonucleotide probes synthesized therewith
EP0708178A1 (en) 1994-10-19 1996-04-24 Institut National De La Sante Et De La Recherche Medicale (Inserm) Survival motor neuron (SMN) gene: a gene for spinal muscular atrophy
US6608035B1 (en) 1994-10-25 2003-08-19 Hybridon, Inc. Method of down-regulating gene expression
US5652356A (en) 1995-08-17 1997-07-29 Hybridon, Inc. Inverted chimeric and hybrid oligonucleotides
US8217015B2 (en) 2003-04-04 2012-07-10 Arrowhead Madison Inc. Endosomolytic polymers
DK0882061T3 (da) 1996-02-14 2004-09-27 Isis Pharmaceuticals Inc Sukkermodificerede gapped oligonukleotider
US6015710A (en) 1996-04-09 2000-01-18 The University Of Texas System Modulation of mammalian telomerase by peptide nucleic acids
US5898031A (en) 1996-06-06 1999-04-27 Isis Pharmaceuticals, Inc. Oligoribonucleotides for cleaving RNA
US5912332A (en) 1996-07-26 1999-06-15 Hybridon, Inc. Affinity-based purification of oligonucleotides using soluble multimeric oligonucleotides
CN1261791A (zh) 1997-07-02 2000-08-02 Sdg有限公司 诊断或治疗用途的靶向脂质体构建物
JPH1133863A (ja) 1997-07-23 1999-02-09 Howa Mach Ltd 工具交換装置及び工具交換用の押出し装置
US6794499B2 (en) * 1997-09-12 2004-09-21 Exiqon A/S Oligonucleotide analogues
US7572582B2 (en) 1997-09-12 2009-08-11 Exiqon A/S Oligonucleotide analogues
US7715989B2 (en) 1998-04-03 2010-05-11 Elitech Holding B.V. Systems and methods for predicting oligonucleotide melting temperature (TmS)
AU4595399A (en) 1998-06-19 2000-01-10 Mcgill University Antisense oligonucleotide constructs based on beta-arabinofuranose and its analogues
US6503754B1 (en) 2000-09-07 2003-01-07 Isis Pharmaceuticals, Inc. Antisense modulation of BH3 interacting domain death agonist expression
US6646113B1 (en) * 1998-09-17 2003-11-11 The Trustees Of The University Of Pennsylvania Nucleic acid molecule encoding human survival of motor neuron-interacting protein 1 (SIP1) deletion mutants
US6210892B1 (en) 1998-10-07 2001-04-03 Isis Pharmaceuticals, Inc. Alteration of cellular behavior by antisense modulation of mRNA processing
EP0999270A1 (en) * 1998-10-09 2000-05-10 Institut National De La Sante Et De La Recherche Medicale (Inserm) Method of regulating SMN gene expression
US6465628B1 (en) 1999-02-04 2002-10-15 Isis Pharmaceuticals, Inc. Process for the synthesis of oligomeric compounds
CZ296576B6 (cs) 1999-02-12 2006-04-12 Sankyo Company Limited Nukleosidový analog a oligonukleotidový analog a farmaceutický prostredek, sonda a primer s jeho obsahem
US9029523B2 (en) 2000-04-26 2015-05-12 Ceres, Inc. Promoter, promoter control elements, and combinations, and uses thereof
US20080281041A1 (en) 1999-06-07 2008-11-13 Rozema David B Reversibly Masked Polymers
US6284538B1 (en) 1999-07-21 2001-09-04 Isis Pharmaceuticals, Inc. Antisense inhibition of PTEN expression
US20040002153A1 (en) 1999-07-21 2004-01-01 Monia Brett P. Modulation of PTEN expression via oligomeric compounds
WO2001007662A1 (en) 1999-07-22 2001-02-01 Genaissance Pharmaceuticals, Inc. Drug target isogenes: polymorphisms in the prostaglandin-endoperoxide synthase 2 gene
US6677445B1 (en) 1999-08-27 2004-01-13 Chiron Corporation Chimeric antisense oligonucleotides and cell transfecting formulations thereof
IL132972A0 (en) 1999-11-16 2001-03-19 Yissum Res Dev Co Pharmaceutical compositions comprising acetylcholinesterase antisense deoxynucleotides for the treatment of muscular and neuromuscular disorders
US6187545B1 (en) * 2000-01-19 2001-02-13 Isis Pharmaceuticals Inc. Antisense modulation of pepck-cytosolic expression
US6287860B1 (en) 2000-01-20 2001-09-11 Isis Pharmaceuticals, Inc. Antisense inhibition of MEKK2 expression
EP1133993A1 (en) 2000-03-10 2001-09-19 Max-Planck-Gesellschaft zur Förderung der Wissenschaften e.V. Substances for the treatment of spinal muscular atrophy
EP1268768A2 (en) 2000-03-27 2003-01-02 University Of Delaware Targeted chromosomal genomic alterations with modified single stranded oligonucleotides
AU2000240366B2 (en) 2000-03-28 2005-08-04 Isis Pharmaceuticals, Inc. Alteration of cellular behavior by antisense modulation of mRNA processing
US20040241651A1 (en) 2000-04-07 2004-12-02 Alexander Olek Detection of single nucleotide polymorphisms (snp's) and cytosine-methylations
US6784291B2 (en) 2000-05-04 2004-08-31 Avi Biopharma, Inc. Splice-region antisense composition and method
EP1289472A4 (en) 2000-05-30 2004-09-08 Advanced Res & Tech Inst COMPOSITIONS AND METHODS FOR IDENTIFYING MODULATORS OF PTEN FUNCTION AND PI-3 KINASE MECHANISMS
US6727355B2 (en) 2000-08-25 2004-04-27 Jcr Pharmaceuticals Co., Ltd. Pharmaceutical composition for treatment of Duchenne muscular dystrophy
CA2437942C (en) 2000-11-09 2013-06-11 Cold Spring Harbor Laboratory Chimeric molecules to modulate gene expression
AU2567002A (en) 2000-11-20 2002-05-27 Univ Illinois Membrane scaffold proteins
US20040058356A1 (en) 2001-03-01 2004-03-25 Warren Mary E. Methods for global profiling gene regulatory element activity
US6825338B2 (en) * 2001-03-30 2004-11-30 Isis Pharmaceuticals, Inc. Labeled oligonucleotides, methods for making same, and compounds useful therefor
JP4353701B2 (ja) 2001-05-08 2009-10-28 ダーウィン モレキュラー コーポレイション Foxp3蛋白質を用いた霊長類における免疫機能の調節方法
US20050176025A1 (en) 2001-05-18 2005-08-11 Sirna Therapeutics, Inc. RNA interference mediated inhibition of B-cell CLL/Lymphoma-2 (BCL-2) gene expression using short interfering nucleic acid (siNA)
AU2002344853A1 (en) 2001-06-14 2003-01-02 Active Pass Pharmaceuticals, Inc. Abca10 transporter
US7407943B2 (en) 2001-08-01 2008-08-05 Isis Pharmaceuticals, Inc. Antisense modulation of apolipoprotein B expression
US7259150B2 (en) 2001-08-07 2007-08-21 Isis Pharmaceuticals, Inc. Modulation of apolipoprotein (a) expression
JP2005508634A (ja) 2001-10-29 2005-04-07 マクギル・ユニヴァーシティ 非環式リンカー含有オリゴヌクレオチド及びその用途
EP1472335A4 (en) * 2001-11-08 2005-12-28 Univ Johns Hopkins METHOD AND SYSTEMS FOR NUCLEIC ACID SEQUENCING
US20030198983A1 (en) 2002-02-01 2003-10-23 Affymetrix, Inc. Methods of genetic analysis of human genes
US20080207542A1 (en) 2002-03-26 2008-08-28 Sirna Therapeutics, Inc. RNA inteference mediated inhibition of hepatitis C virus (HVC) gene expression using short interfering nucleic acid (siNA)
EP2264172B1 (en) 2002-04-05 2017-09-27 Roche Innovation Center Copenhagen A/S Oligomeric compounds for the modulation of hif-1alpha expression
US20050130924A1 (en) 2002-06-26 2005-06-16 Monia Brett P. Antisense inhibition via RNAse H-independent reduction in mRNA
WO2004011613A2 (en) 2002-07-29 2004-02-05 Epigenesis Pharmaceuticals, Inc. Composition & methods for treatment and screening
US20050003369A1 (en) 2002-10-10 2005-01-06 Affymetrix, Inc. Method for depleting specific nucleic acids from a mixture
US20040219565A1 (en) 2002-10-21 2004-11-04 Sakari Kauppinen Oligonucleotides useful for detecting and analyzing nucleic acids of interest
WO2004044132A2 (en) * 2002-11-05 2004-05-27 Isis Pharmaceuticals, Inc. Modified oligonucleotides for use in rna interference
US7655785B1 (en) 2002-11-14 2010-02-02 Rosetta Genomics Ltd. Bioinformatically detectable group of novel regulatory oligonucleotides and uses thereof
DK2284266T3 (da) 2002-11-14 2014-01-13 Thermo Fisher Scient Biosciences Inc sIRNA-MOLEKYLE MOD TP53
US7250496B2 (en) 2002-11-14 2007-07-31 Rosetta Genomics Ltd. Bioinformatically detectable group of novel regulatory genes and uses thereof
ES2607471T3 (es) 2002-11-18 2017-03-31 Roche Innovation Center Copenhagen A/S Diseño antisentido
WO2004069992A2 (en) 2003-02-10 2004-08-19 Santaris Pharma A/S Oligomeric compounds for the modulation of ras expression
CA2519860C (en) 2003-03-21 2018-01-16 Santaris Pharma A/S Short interfering rna (sirna) analogues
FR2853663B1 (fr) 2003-04-14 2007-08-31 Aventis Pharma Sa Procede d'obtention de lignees de mastocytes a partir de tissus de porcs et procede de production de molecules de type heparine
WO2005001110A2 (en) 2003-05-29 2005-01-06 The Salk Institute For Biological Studies Transcriptional regulation of gene expression by small double-stranded modulatory rna
WO2004113867A2 (en) 2003-06-16 2004-12-29 University Of Massachusetts Exon analysis
WO2005013901A2 (en) 2003-07-31 2005-02-17 Isis Pharmaceuticals, Inc. Oligomeric compounds and compositions for use in modulation of small non-coding rnas
US7888497B2 (en) 2003-08-13 2011-02-15 Rosetta Genomics Ltd. Bioinformatically detectable group of novel regulatory oligonucleotides and uses thereof
US20050108783A1 (en) * 2003-09-23 2005-05-19 Chihiro Koike Porcine invariant chain protein, full length cDNA, genomic organization, and regulatory region
EP1682661A2 (en) 2003-10-23 2006-07-26 Sirna Therapeutics, Inc. Rna interference mediated inhibition of gene expression using short interfering nucleic acid (sina)
GB0324854D0 (en) 2003-10-24 2003-11-26 Expresson Biosystems Ltd App/ena antisense
KR101107818B1 (ko) 2003-10-30 2012-01-31 콜레이 파마시티컬 그룹, 인코포레이티드 향상된 면역자극 효능을 가진 c-부류 올리고뉴클레오티드유사체
CA2566823A1 (en) 2003-11-05 2005-05-19 Board Of Regents, The University Of Texas System Diagnostic and therapeutic methods and compositions involving pten and breast cancer
AU2004303464B2 (en) 2003-12-23 2009-10-01 Santaris Pharma A/S Oligomeric compounds for the modulation of BCL-2
US20050244851A1 (en) 2004-01-13 2005-11-03 Affymetrix, Inc. Methods of analysis of alternative splicing in human
CA2553104A1 (en) 2004-01-23 2005-08-11 Avi Biopharma, Inc. Antisense oligomers and methods for inducing immune tolerance and immunosuppression
US20050164209A1 (en) 2004-01-23 2005-07-28 Bennett C. F. Hepatocyte free uptake assays
US20080249039A1 (en) 2004-01-30 2008-10-09 Santaris Pharma A/S Modified Short Interfering Rna (Modified Sirna)
WO2005089169A2 (en) 2004-03-12 2005-09-29 Exelixis, Inc. Mptens as modifiers of the pten pathway and methods of use
US8314226B2 (en) 2004-03-29 2012-11-20 The General Hospital Corporation Oligonucleotide complex compositions and methods of use as gene alteration tools
US7374927B2 (en) 2004-05-03 2008-05-20 Affymetrix, Inc. Methods of analysis of degraded nucleic acid samples
KR20070085113A (ko) 2004-05-11 2007-08-27 가부시키가이샤 알파젠 Rna간섭을 생기게 하는 폴리뉴클레오티드, 및 이를 이용한유전자발현억제 방법
US7687616B1 (en) 2004-05-14 2010-03-30 Rosetta Genomics Ltd Small molecules modulating activity of micro RNA oligonucleotides and micro RNA targets and uses thereof
US20050265927A1 (en) 2004-05-17 2005-12-01 Yale University Intranasal delivery of nucleic acid molecules
EP2206781B1 (en) 2004-06-28 2015-12-02 The University Of Western Australia Antisense oligonucleotides for inducing exon skipping and methods of use thereof
US8029985B2 (en) 2004-09-01 2011-10-04 Vybion, Inc. Amplified bioassay
US7838657B2 (en) 2004-12-03 2010-11-23 University Of Massachusetts Spinal muscular atrophy (SMA) treatment via targeting of SMN2 splice site inhibitory sequences
WO2006063356A1 (en) 2004-12-10 2006-06-15 Isis Phamaceuticals, Inc. Regulation of epigenetic control of gene expression
US7879992B2 (en) 2005-01-31 2011-02-01 Isis Pharmaceuticals, Inc. Modification of MyD88 splicing using modified oligonucleotides
WO2006086667A2 (en) 2005-02-09 2006-08-17 Avi Bio Pharma, Inc. Antisense composition and method for treating muscle atrophy
US8999943B2 (en) 2005-03-14 2015-04-07 Board Of Regents, The University Of Texas System Antigene oligomers inhibit transcription
US7449297B2 (en) * 2005-04-14 2008-11-11 Euclid Diagnostics Llc Methods of copying the methylation pattern of DNA during isothermal amplification and microarrays
WO2006113246A2 (en) 2005-04-15 2006-10-26 The Regents Of The University Of California Small activating rna molecules and methods of use
US8586719B2 (en) 2005-04-27 2013-11-19 Pacific Arrow Limited Triterpenes for modulating gene expression and cell membrane, and as antiprotozoal agents
HUE027486T2 (en) 2005-06-23 2016-09-28 Isis Pharmaceuticals Inc Preparations and methods for modifying SMN2-Splicing
US8067571B2 (en) 2005-07-13 2011-11-29 Avi Biopharma, Inc. Antibacterial antisense oligonucleotide and method
WO2007031091A2 (en) 2005-09-15 2007-03-22 Santaris Pharma A/S Rna antagonist compounds for the modulation of p21 ras expression
EP2641970B1 (en) 2005-11-17 2014-12-24 Board of Regents, The University of Texas System Modulation of gene expression by oligomers targeted to chromosomal DNA
AU2006315102A1 (en) 2005-11-21 2007-05-24 Johnson & Johnson Research Pty Limited Multitargeting interfering RNAs having two active strands and methods for their design and use
WO2007087113A2 (en) 2005-12-28 2007-08-02 The Scripps Research Institute Natural antisense and non-coding rna transcripts as drug targets
US20090011004A1 (en) 2005-12-30 2009-01-08 Philadelphia Health & Education Corp., D/B/A/ Drexel University Of College Of Medicine Improved carriers for delivery of nucleic acid agents to cells and tissues
PL2314594T3 (pl) 2006-01-27 2014-12-31 Isis Pharmaceuticals Inc Zmodyfikowane w pozycji 6 analogi bicykliczne kwasów nukleinowych
US7569686B1 (en) 2006-01-27 2009-08-04 Isis Pharmaceuticals, Inc. Compounds and methods for synthesis of bicyclic nucleic acid analogs
EP1984499B1 (en) 2006-01-27 2015-05-27 Isis Pharmaceuticals, Inc. Oligomeric compounds and compositions for the use in modulation of micrornas
US8362229B2 (en) 2006-02-08 2013-01-29 Quark Pharmaceuticals, Inc. Tandem siRNAS
MX2008012219A (es) 2006-04-03 2008-10-02 Santaris Pharma As Composicion farmaceutica que comprende oligonucleotidos antisentido anti-miarn.
CA2647125C (en) 2006-04-07 2015-02-03 Georg-August-Universitaet Goettingen Stiftung Oeffentlichen Rechts Synthetic mecp2 sequence for protein substitution therapy
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
WO2008002996A2 (en) 2006-06-27 2008-01-03 Shanghai Institutes For Biological Sciences Rheumatoid arthritis t cell vaccine
JP5274461B2 (ja) 2006-08-18 2013-08-28 アローヘッド リサーチ コーポレイション ポリヌクレオチドのインビボ送達のためのポリ結合体
US9617581B2 (en) 2006-08-25 2017-04-11 Duke University Methods for in vivo identification of endogenous mRNA targets of MicroRNAs
WO2008025069A1 (en) 2006-08-28 2008-03-06 The Walter And Eliza Hall Institute Of Medical Research Methods of modulating cellular activity and compositions therefor
WO2008029619A1 (fr) 2006-09-07 2008-03-13 Daiichi Sankyo Company, Limited Oligonucléotide antisens ena ayant une action spécifique de la séquence
US20080176793A1 (en) 2006-09-18 2008-07-24 The Regents Of The University Of California Upregulating activity or expression of bdnf to mitigate cognitive impairment in asymptomatic huntington's subjects
DK2092065T4 (da) 2006-10-18 2019-10-21 Ionis Pharmaceuticals Inc Antisense-forbindelser
WO2008061537A2 (en) 2006-11-23 2008-05-29 Querdenker Aps Oligonucleotides for modulating target rna activity
WO2008066784A2 (en) 2006-11-27 2008-06-05 Ludwig Institute For Cancer Research Expression of foxp3 by cancer cells
CA2676039A1 (en) 2007-01-19 2008-07-24 The Regents Of The University Of Michigan Adrb2 cancer markers
ITMI20070127A1 (it) 2007-01-29 2008-07-30 Fond I R C C S Proteine e-o peptidi per la prevenzione e-o cura di malattie neurodegenerative
WO2008103761A2 (en) 2007-02-20 2008-08-28 Sequenom, Inc. Methods and compositions for cancer diagnosis and treatment based on nucleic acid methylation
US7858592B2 (en) 2007-02-26 2010-12-28 The Board Of Regents Of The University Of Texas System Interfering RNAs against the promoter region of P53
DK2149605T3 (da) 2007-03-22 2013-09-30 Santaris Pharma As Korte RNA antagonist forbindelser til modulering af det ønskede mRNA
US8158596B2 (en) 2007-05-11 2012-04-17 The Regents Of The University Of Michigan Materials and methods for FOXP3 tumor suppression
CN101821390A (zh) 2007-06-14 2010-09-01 米尔克斯治疗有限责任公司 用于调节靶rna活性的寡核苷酸
US20090082297A1 (en) 2007-06-25 2009-03-26 Lioy Daniel T Compositions and Methods for Regulating Gene Expression
EP2170363B1 (en) * 2007-06-29 2018-08-08 Sarepta Therapeutics, Inc. Tissue specific peptide conjugates and methods
WO2009027349A2 (en) 2007-08-24 2009-03-05 Oryzon Genomics Sa Treatment and prevention of neurodegenerative diseases
EP2205746A4 (en) 2007-10-04 2010-12-22 Univ Texas MODULATION OF GENE EXPRESSION WITH AGRNA AND GAPS WITH ANTISENSE TRANSCRIPTS AS A TARGET
MY156951A (en) 2007-10-04 2016-04-15 Santaris Pharma As Micromirs
ITRM20070523A1 (it) 2007-10-05 2009-04-06 Consiglio Nazionale Ricerche Acido nucleico codificante per una proteina regolatrice specifica della trascrizione dell'utrofina proteina da esso codificata e sue applicazioni
EP2227545A2 (en) 2007-11-09 2010-09-15 Isis Pharmaceuticals, Inc. Modulation of factor 7 expression
WO2009064920A2 (en) 2007-11-13 2009-05-22 Isis Pharmaceuticals, Inc. Compounds and methods for modulating protein expression
US8937217B2 (en) 2007-12-18 2015-01-20 E. I. Du Pont De Nemours And Company Down-regulation of gene expression using artificial microRNAs
WO2009090182A1 (en) 2008-01-14 2009-07-23 Santaris Pharma A/S C4'-substituted - dna nucleotide gapmer oligonucleotides
CA2717637A1 (en) 2008-03-07 2009-09-17 Senesco Technologies, Inc. Use of sirna to achieve down regulation of an endogenous gene in combination with the use of a sense construct to achieve expression of a desired polynucleotide
US8361980B2 (en) 2008-03-07 2013-01-29 Santaris Pharma A/S Pharmaceutical compositions for treatment of microRNA related diseases
WO2009124295A2 (en) 2008-04-04 2009-10-08 Isis Pharmaceuticals, Inc. Oligomeric compounds comprising bicyclic nucleosides and having reduced toxicity
US20110263687A1 (en) 2008-04-07 2011-10-27 Riken Rna molecules and uses thereof
US8916532B2 (en) 2008-04-28 2014-12-23 The Trustees Of The University Of Pennsylvania Methods for enhancing utrophin production via inhibition of microRNA
WO2009151546A2 (en) 2008-05-27 2009-12-17 Ptc Therapeutics, Inc. Methods for treating spinal muscular atrophy
US8222221B2 (en) 2008-06-04 2012-07-17 The Board Of Regents Of The University Of Texas System Modulation of gene expression through endogenous small RNA targeting of gene promoters
CA2635187A1 (en) 2008-06-05 2009-12-05 The Royal Institution For The Advancement Of Learning/Mcgill University Oligonucleotide duplexes and uses thereof
DK2310505T3 (en) 2008-06-30 2017-10-16 Roche Innovation Ct Copenhagen As ANTIDOT oligomers
WO2010014592A1 (en) 2008-07-29 2010-02-04 The Board Of Regents Of The University Of Texas Sytem Selective inhibition of polyglutamine protein expression
EP2331543A4 (en) 2008-09-26 2013-06-19 Agency Science Tech & Res 3-DÉSAZANEPLANOCINE DERIVATIVES
MY188457A (en) 2008-10-03 2021-12-10 Opko Curna Llc Treatment of apolipoprotein-a1 related diseases by inhibition of natural antisense transcript to apolipoprotein-a1
AU2009305639B2 (en) 2008-10-16 2016-06-23 Marina Biotech, Inc. Processes and compositions for liposomal and efficient delivery of gene silencing therapeutics
CA2743249A1 (en) 2008-11-17 2010-05-20 F. Hoffmann-La Roche Ag Compositions and methods for inhibiting expression of factor vii genes
GB0821457D0 (en) 2008-11-24 2008-12-31 Trillion Genomics Ltd Oligonucleotides
KR101866152B1 (ko) 2008-12-04 2018-06-08 큐알엔에이, 인크. 종양 억제 유전자에 대한 천연 안티센스 전사체의 억제에 의해 종양 억제 유전자 관련된 질환의 치료
CN102317458B (zh) 2008-12-04 2018-01-02 库尔纳公司 通过红细胞生成素(epo)天然反义转录物的抑制对epo相关疾病的治疗
EP2370581B1 (en) 2008-12-04 2016-08-03 CuRNA, Inc. Treatment of vascular endothelial growth factor (vegf) related diseases by inhibition of natural antisense transcript to vegf
CN102307997B (zh) 2008-12-04 2018-03-30 库尔纳公司 通过抑制针对沉默调节蛋白1的天然反义转录物来治疗沉默调节蛋白1(sirt1)相关的疾病
EP2405921A4 (en) 2009-01-26 2013-05-22 Protiva Biotherapeutics Inc COMPOSITIONS AND METHODS FOR INACTIVATION OF APOLIPOPROTEIN C-III EXPRESSION
HUE026280T2 (en) 2009-02-12 2016-06-28 Curna Inc Treatment of brain-derived neurotrophic factor (BDNF) -related diseases by inhibition of natural antisense transcripts associated with BDNF \ t
EP2396408B1 (en) 2009-02-12 2017-09-20 CuRNA, Inc. Treatment of glial cell derived neurotrophic factor (gdnf) related diseases by inhibition of natural antisense transcript to gdnf
WO2010093860A2 (en) 2009-02-13 2010-08-19 The General Hospital Corporation Isolation of factors that associate directly or indirectly with chromatin
US20110319317A1 (en) 2009-03-04 2011-12-29 Opko Curna, Llc Treatment of sirtuin 1 (sirt1) related diseases by inhibition of natural antisense transcript to sirt1
WO2010107733A2 (en) 2009-03-16 2010-09-23 Curna, Inc. Treatment of nuclear factor (erythroid-derived 2)-like 2 (nrf2) related diseases by inhibition of natural antisense transcript to nrf2
JP5904935B2 (ja) 2009-03-17 2016-04-20 クルナ・インコーポレーテッド デルタ様1ホモログ(dlk1)に対する天然アンチセンス転写物の抑制によるdlk1関連疾患の治療
US20120149756A1 (en) 2009-04-10 2012-06-14 Associatin Institut de Myologie Tricyclo-dna antisense oligonucleotides, compositions, and methods for the treatment of disease
EP2421972A2 (en) 2009-04-24 2012-02-29 The Board of Regents of The University of Texas System Modulation of gene expression using oligomers that target gene regions downstream of 3' untranslated regions
ES2661787T3 (es) 2009-05-01 2018-04-04 Curna, Inc. Tratamiento de enfermedades relacionadas con hemoglobina (hbf/hbg) por inhibición de transcrito antisentido natural para hbf/hbg
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
CA2761152A1 (en) 2009-05-06 2010-11-11 Opko Curna, Llc Treatment of lipid transport and metabolism gene related diseases by inhibition of natural antisense transcript to a lipid transport and metabolism gene
KR101742334B1 (ko) 2009-05-08 2017-06-01 큐알엔에이, 인크. Dmd 패밀리에 대한 천연 안티센스 전사체의 억제에 의한 디스트로핀 패밀리 관련된 질환의 치료
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
EP2435571B1 (en) 2009-05-28 2016-12-14 CuRNA, Inc. Treatment of antiviral gene related diseases by inhibition of natural antisense transcript to an antiviral gene
KR101702689B1 (ko) 2009-06-16 2017-02-06 큐알엔에이, 인크. Pon1에 대한 천연 안티센스 전사체의 억제에 의한 파라옥소나제 1(pon1) 관련된 질환의 치료
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
CA2765889A1 (en) 2009-06-24 2010-12-29 Opko Curna, Llc 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
CA2767225A1 (en) 2009-07-06 2011-01-13 Alnylam Pharmaceuticals, Inc. Compositions and methods for enhancing production of a biological product
CA2768598A1 (en) 2009-07-22 2011-01-27 Cenix Bioscience Gmbh Delivery system and conjugates for compound delivery via naturally occurring intracellular transport routes
WO2011010706A1 (ja) 2009-07-23 2011-01-27 武田薬品工業株式会社 Fgf21シスエレメント結合物質
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
EP2464731B1 (en) 2009-08-11 2016-10-05 CuRNA, Inc. Treatment of adiponectin (adipoq) related diseases by inhibition of natural antisense transcript to an adiponectin (adipoq)
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
US9023822B2 (en) 2009-08-25 2015-05-05 Curna, Inc. Treatment of 'IQ motif containing GTPase activating protein' (IQGAP) related diseases by inhibition of natural antisense transcript to IQGAP
WO2011025862A2 (en) 2009-08-28 2011-03-03 Curna, Inc. Treatment of atp-binding cassette sub-family b member 1 (abcb1) related diseases by inhibition of natural antisense transcript to abcb1
WO2011032109A1 (en) 2009-09-11 2011-03-17 Sma Foundation Biomarkers for spinal muscular atrophy
EP2480669B1 (en) 2009-09-25 2017-11-08 CuRNA, Inc. Treatment of filaggrin (flg) related diseases by modulation of flg expression and activity
WO2011038205A2 (en) 2009-09-25 2011-03-31 Curna, Inc. Treatment of growth hormone (gh) related diseases by inhibition of natural antisense transcript to gh
US9364495B2 (en) 2009-10-20 2016-06-14 Roche Innovation Center Copenhagen A/S Oral delivery of therapeutically effective LNA oligonucleotides
KR101138048B1 (ko) 2009-11-06 2012-04-23 성균관대학교산학협력단 Bdnf의 발현을 증가시키는 신규 펩타이드 및 이를 포함하는 알츠하이머병 또는 파킨슨병의 예방 및 치료용 약학 조성물
CN102712927B (zh) 2009-12-16 2017-12-01 库尔纳公司 通过抑制膜结合转录因子肽酶,位点1(mbtps1)的天然反义转录物来治疗mbtps1相关疾病
CN102781480B (zh) 2009-12-23 2018-07-27 库尔纳公司 通过抑制解偶联蛋白2(ucp2)的天然反义转录物而治疗ucp2相关疾病
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
CN102770540B (zh) 2009-12-29 2017-06-23 库尔纳公司 通过抑制肿瘤蛋白63(p63)的天然反义转录物而治疗p63相关疾病
JP6083735B2 (ja) 2009-12-31 2017-02-22 カッパーアールエヌエー,インコーポレイテッド インスリン受容体基質2(irs2)および転写因子3(tfe3)に対する天然アンチセンス転写物の阻害によるインスリン受容体基質2(irs2)関連疾患の治療
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
CN102822342B (zh) 2010-01-06 2017-05-10 库尔纳公司 通过抑制胰腺发育基因的天然反义转录物而治疗胰腺发育基因相关疾病
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
NO2529015T3 (ja) 2010-01-25 2018-04-14
WO2011097388A1 (en) 2010-02-03 2011-08-11 Alnylam Pharmaceuticals, Inc. Selective inhibition of polyglutamine protein expression
WO2011097582A2 (en) 2010-02-08 2011-08-11 Opko Curna Llc Treatment of arachidonate 12-lipoxygenase, 12r type (alox12b) related diseases by inhibition of natural antisense transcript to alox12b
WO2011097641A1 (en) 2010-02-08 2011-08-11 Isis Pharmaceuticals, Inc. Methods and compositions useful in treatment of diseases or conditions related to repeat expansion
US8962586B2 (en) 2010-02-22 2015-02-24 Curna, Inc. Treatment of pyrroline-5-carboxylate reductase 1 (PYCR1) related diseases by inhibition of natural antisense transcript to PYCR1
WO2011117353A1 (en) 2010-03-24 2011-09-29 Mirrx Therapeutics A/S Bivalent antisense oligonucleotides
EP2553098B1 (en) 2010-04-02 2017-10-11 CuRNA, Inc. Treatment of colony-stimulating factor 3 (csf3) related diseases by inhibition of natural antisense transcript to csf3
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
EP2558578B1 (en) 2010-04-13 2015-10-28 Life Technologies Corporation Compositions and methods for inhibition of nucleic acids function
CA2798218A1 (en) 2010-05-03 2011-11-10 Curna, Inc. 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
WO2011146675A2 (en) 2010-05-19 2011-11-24 Opko Curna Llc Treatment of lim homeobox 2 (lhx2) related diseases by inhibition of natural antisense transcript to lhx2
AR081209A1 (es) 2010-05-19 2012-07-04 Opko Curna Llc Tratamiento de enfermedades relacionadas con la proteina 11 del tipo linfoma 2 de celulas b, (bcl2, bcl2l11) mediante la inhibicion del transcripto antisentido natural a bcl2l11
NO2576783T3 (ja) 2010-05-26 2018-04-28
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
EP2582397A4 (en) 2010-06-15 2014-10-29 Isis Pharmaceuticals Inc COMPOUNDS AND METHODS FOR MODULATING THE INTERACTION BETWEEN PROTEINS AND TARGET NUCLEIC ACIDS
US20120004278A1 (en) 2010-06-18 2012-01-05 The Board Of Trustees Of The Leland Stanford Junior University Linc rnas in cancer diagnosis and treatment
GB201010557D0 (en) 2010-06-23 2010-08-11 Mina Therapeutics Ltd RNA molecules and uses thereof
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
EP2591797A4 (en) 2010-07-09 2014-01-15 Inst Pasteur Of Shanghai Cas FACTOR FOR REGULATING FOXP3 AND REGULATORY T CELLS AND USE THEREOF
WO2012009347A2 (en) 2010-07-12 2012-01-19 Opko Curna Llc Treatment of bcl2 binding component 3 (bbc3) related diseases by inhibition of natural antisense transcript to bbc3
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
WO2012012443A2 (en) 2010-07-19 2012-01-26 Bennett C Frank Modulation of dystrophia myotonica-protein kinase (dmpk) expression
WO2012027033A1 (en) 2010-07-19 2012-03-01 Isis Pharmaceuticals, Inc. Compounds and methods for modulating target nuclear and sub-nuclear nucleic acid molecules in cells and animals
WO2012018881A2 (en) 2010-08-03 2012-02-09 Alnylam Pharmaceuticals, Inc. Methods and compositions for the regulation of rna
WO2012024478A2 (en) 2010-08-19 2012-02-23 Opko Curna Llc Treatment of nicotinamide phosphoribosyltransferase (nampt) related diseases by inhibition of natural antisense transcript to nampt
RU2765155C2 (ru) 2010-09-10 2022-01-26 Эпизайм, Инк. Ингибиторы ezh2 человека и способы их применения
KR101886457B1 (ko) 2010-10-06 2018-08-07 큐알엔에이, 인크. 시알리다아제 4 (neu4)에 대한 자연 안티센스 전사체의 저해에 의한 neu4 관련된 질환의 치료
EP2630241B1 (en) 2010-10-22 2018-10-17 CuRNA, Inc. Treatment of alpha-l-iduronidase (idua) related diseases by inhibition of natural antisense transcript to idua
DK2633052T3 (en) 2010-10-27 2018-07-16 Curna Inc TREATMENT OF INTERFERON-RELATED DEVELOPMENT REGULATOR 1 (IFRD1) -RELATED DISEASES BY INHIBITION OF NATURAL ANTISENCE TRANSCRIPT TO IFRD1
WO2012064806A2 (en) 2010-11-11 2012-05-18 The University Of North Carolina At Chapel Hill Methods and compositions for unsilencing imprinted genes
EP3702460A1 (en) 2010-11-12 2020-09-02 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
WO2012068340A2 (en) 2010-11-18 2012-05-24 Opko Curna Llc Antagonat compositions and methods of use
KR102010598B1 (ko) 2010-11-23 2019-08-13 큐알엔에이, 인크. Nanog에 대한 자연 안티센스 전사체의 저해에 의한 nanog 관련된 질환의 치료
EP2655621B1 (en) 2010-12-20 2018-05-23 The General Hospital Corporation Polycomb-associated non-coding rnas
WO2012109476A2 (en) 2011-02-09 2012-08-16 The University Of Rochester Methods and compositions related to staufen 1 binding sites formed by duplexing alu elements
WO2012144220A1 (en) 2011-04-22 2012-10-26 Oncotherapy Science, Inc. Ezh2 as target gene for cancer therapy and diagnosis
US8557787B2 (en) 2011-05-13 2013-10-15 The Board Of Trustees Of The Leland Stanford Junior University Diagnostic, prognostic and therapeutic uses of long non-coding RNAs for cancer and regenerative medicine
US9593330B2 (en) 2011-06-09 2017-03-14 Curna, Inc. Treatment of frataxin (FXN) related diseases by inhibition of natural antisense transcript to FXN
AU2012272518A1 (en) 2011-06-24 2014-01-09 Murdoch Childrens Research Institute Treatment and diagnosis of epigenetic disorders and conditions
CA2844144A1 (en) 2011-07-05 2013-01-10 The General Hospital Corporation Rna-yy1 interactions
WO2013036403A1 (en) 2011-09-06 2013-03-14 Curna, Inc. TREATMENT OF DISEASES RELATED TO ALPHA SUBUNITS OF SODIUM CHANNELS, VOLTAGE-GATED (SCNxA) WITH SMALL MOLECULES
AU2012308320C1 (en) 2011-09-14 2018-08-23 Translate Bio Ma, Inc. Multimeric oligonucleotide compounds
WO2013080784A1 (ja) 2011-11-30 2013-06-06 シャープ株式会社 メモリ回路とその駆動方法、及び、これを用いた不揮発性記憶装置、並びに、液晶表示装置
ITMI20120275A1 (it) 2012-02-24 2013-08-25 Biogenera Societa A Responsabilita Limitata Oligonucleotidi per la modulazione dell'espressione genica e loro usi
HUE040179T2 (hu) 2012-03-15 2019-02-28 Curna Inc Agyi eredetû neutrotróf faktorral (Brain-derived neurotrophic factor, BDNF) összefüggõ betegségek kezelése a BDNF-fel kapcsolatos természetes antiszensz transzkriptumok gátlása révén
WO2013173637A1 (en) 2012-05-16 2013-11-21 Rana Therapeutics, Inc. Compositions and methods for modulating gene expression
WO2013173601A1 (en) 2012-05-16 2013-11-21 Rana Therapeutics, Inc. Compositions and methods for modulating bdnf expression
EA201492119A1 (ru) 2012-05-16 2015-05-29 Рана Терапьютикс, Инк. Композиции и способы для модулирования экспрессии apoa1 и abca1
WO2013173652A1 (en) 2012-05-16 2013-11-21 Rana Therapeutics, Inc. Compositions and methods for modulating gene expression
AU2013262706A1 (en) 2012-05-16 2015-01-22 Rana Therapeutics, Inc. Compositions and methods for modulating PTEN expression
US20150232836A1 (en) 2012-05-16 2015-08-20 Rana Therapeutics, Inc. Compositions and methods for modulating gene expression
WO2013173598A1 (en) 2012-05-16 2013-11-21 Rana Therapeutics, Inc. Compositions and methods for modulating atp2a2 expression
DK2850186T3 (en) 2012-05-16 2019-04-08 Translate Bio Ma Inc COMPOSITIONS AND PROCEDURES FOR MODULATING SMN GENFAMILY EXPRESSION
WO2013173645A1 (en) 2012-05-16 2013-11-21 Rana Therapeutics, Inc. Compositions and methods for modulating utrn expression
US10174315B2 (en) 2012-05-16 2019-01-08 The General Hospital Corporation Compositions and methods for modulating hemoglobin gene family expression
EP2850190B1 (en) 2012-05-16 2020-07-08 Translate Bio MA, Inc. Compositions and methods for modulating mecp2 expression
US9567581B2 (en) 2012-08-07 2017-02-14 The General Hospital Corporation Selective reactivation of genes on the inactive X chromosome
EP2895200B1 (en) 2012-09-14 2019-11-06 Translate Bio MA, Inc. Multimeric oligonucleotide compounds
KR20150083920A (ko) 2012-11-15 2015-07-20 로슈 이노베이션 센터 코펜하겐 에이/에스 항 apob 안티센스 접합체 화합물
US10590412B2 (en) 2013-04-19 2020-03-17 Ionis Pharmaceuticals, Inc. Compositions and methods for modulation nucleic acids through nonsense mediated decay
JP2016521556A (ja) 2013-06-07 2016-07-25 ラナ セラピューティクス インコーポレイテッド Foxp3発現を調節するための組成物及び方法
AU2014282666A1 (en) 2013-06-16 2016-01-07 National University Corporation Tokyo Medical And Dental University Double-stranded antisense nucleic acid with exon-skipping effect
WO2015035476A1 (en) 2013-09-16 2015-03-19 University Of Western Sydney Modulation of gene expression

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EP2850186A4 (en) 2016-01-27
EP2850186A1 (en) 2015-03-25
JP2015523854A (ja) 2015-08-20

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