DK3031920T3 - Modulation af dystrofi myotonica-protein kinase (dmpk) ekspression - Google Patents

Modulation af dystrofi myotonica-protein kinase (dmpk) ekspression

Info

Publication number
DK3031920T3
DK3031920T3 DK16153949.9T DK16153949T DK3031920T3 DK 3031920 T3 DK3031920 T3 DK 3031920T3 DK 16153949 T DK16153949 T DK 16153949T DK 3031920 T3 DK3031920 T3 DK 3031920T3
Authority
DK
Denmark
Prior art keywords
myotonica
dmpk
dystrophy
modulation
expression
Prior art date
Application number
DK16153949.9T
Other languages
English (en)
Inventor
C Frank Bennett
Susan M Freier
Robert A Macleod
Sanjey K Pandey
Charles A Thornton
Thurman Wheeler
Seng H Cheng
Andrew Leger
Bruce M Wentworth
Original Assignee
Ionis Pharmaceuticals Inc
Univ Rochester
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
Family has litigation
First worldwide family litigation filed litigation Critical https://patents.darts-ip.com/?family=45497419&utm_source=google_patent&utm_medium=platform_link&utm_campaign=public_patent_search&patent=DK3031920(T3) "Global patent litigation dataset” by Darts-ip is licensed under a Creative Commons Attribution 4.0 International License.
Application filed by Ionis Pharmaceuticals Inc, Univ Rochester filed Critical Ionis Pharmaceuticals Inc
Application granted granted Critical
Publication of DK3031920T3 publication Critical patent/DK3031920T3/da

Links

Classifications

    • 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
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • 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
    • C12N15/1137Non-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 enzymes
    • 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
    • A61P15/00Drugs for genital or sexual disorders; Contraceptives
    • 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
    • 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
    • A61P21/04Drugs for disorders of the muscular or neuromuscular system for myasthenia gravis
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • A61P25/14Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • A61P25/18Antipsychotics, i.e. neuroleptics; Drugs for mania or schizophrenia
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00Antineoplastic agents
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • 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
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12YENZYMES
    • C12Y207/00Transferases transferring phosphorus-containing groups (2.7)
    • C12Y207/11Protein-serine/threonine kinases (2.7.11)
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/11Antisense
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/31Chemical structure of the backbone
    • C12N2310/315Phosphorothioates
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/31Chemical structure of the backbone
    • C12N2310/318Chemical structure of the backbone where the PO2 is completely replaced, e.g. MMI or formacetal
    • C12N2310/3181Peptide nucleic acid, PNA
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/32Chemical structure of the sugar
    • C12N2310/3212'-O-R Modification
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/32Chemical structure of the sugar
    • C12N2310/3222'-R Modification
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/33Chemical structure of the base
    • C12N2310/334Modified C
    • C12N2310/33415-Methylcytosine
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/34Spatial arrangement of the modifications
    • C12N2310/341Gapmers, i.e. of the type ===---===
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/34Spatial arrangement of the modifications
    • C12N2310/346Spatial arrangement of the modifications having a combination of backbone and sugar modifications
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/35Nature of the modification
    • C12N2310/352Nature of the modification linked to the nucleic acid via a carbon atom
    • C12N2310/3525MOE, methoxyethoxy

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Health & Medical Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Biomedical Technology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Medicinal Chemistry (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • General Engineering & Computer Science (AREA)
  • Molecular Biology (AREA)
  • Biotechnology (AREA)
  • Biochemistry (AREA)
  • Neurology (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Microbiology (AREA)
  • Virology (AREA)
  • Neurosurgery (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Epidemiology (AREA)
  • Psychiatry (AREA)
  • Endocrinology (AREA)
  • Reproductive Health (AREA)
  • Psychology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
DK16153949.9T 2010-07-19 2011-07-19 Modulation af dystrofi myotonica-protein kinase (dmpk) ekspression DK3031920T3 (da)

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
US36577510P 2010-07-19 2010-07-19
US36576210P 2010-07-19 2010-07-19
US201161478021P 2011-04-21 2011-04-21
EP20110810291 EP2595663A4 (en) 2010-07-19 2011-07-19 MODULATION OF DYSTROPHIA MYOTONICA PROTEIN KINASE (DMPK) EXPRESSION

Publications (1)

Publication Number Publication Date
DK3031920T3 true DK3031920T3 (da) 2019-10-14

Family

ID=45497419

Family Applications (1)

Application Number Title Priority Date Filing Date
DK16153949.9T DK3031920T3 (da) 2010-07-19 2011-07-19 Modulation af dystrofi myotonica-protein kinase (dmpk) ekspression

Country Status (13)

Country Link
US (7) US20130237585A1 (da)
EP (5) EP3489360A3 (da)
JP (5) JP6059141B2 (da)
KR (2) KR20180105730A (da)
CN (2) CN106434648A (da)
AU (6) AU2011282243B2 (da)
CA (2) CA2805765A1 (da)
DK (1) DK3031920T3 (da)
ES (1) ES2756326T3 (da)
HR (1) HRP20191753T1 (da)
IL (2) IL223962B (da)
PL (1) PL3031920T3 (da)
WO (2) WO2012012467A2 (da)

Families Citing this family (125)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AU2007282224B2 (en) 2006-08-11 2013-08-29 Vico Therapeutics B.V. Methods and means for treating DNA repeat instability associated genetic disorders
AU2008317566B2 (en) 2007-10-26 2014-05-01 Academisch Ziekenhuis Leiden Means and methods for counteracting muscle disorders
WO2012012467A2 (en) 2010-07-19 2012-01-26 Isis Pharmaceuticals, Inc. Modulation of nuclear-retained rna
US9920317B2 (en) 2010-11-12 2018-03-20 The General Hospital Corporation Polycomb-associated non-coding RNAs
EP3260540A1 (en) 2010-11-12 2017-12-27 The General Hospital Corporation Polycomb-associated non-coding rnas
US9976138B2 (en) 2011-08-29 2018-05-22 Ionis Pharmaceuticals, Inc. Methods and compounds useful in conditions related to repeat expansion
HK1200484A1 (zh) 2011-09-14 2015-08-07 Translate Bio Ma, Inc. 多聚寡核苷酸化合物
ES2673960T3 (es) * 2011-12-22 2018-06-26 Ionis Pharmaceuticals, Inc. Métodos para modular la expresión de un transcrito-1 de adenocarcinoma en pulmón asociado a metástasis (MALAT-1)
CN112251436A (zh) 2012-01-27 2021-01-22 比奥马林技术公司 治疗杜兴型肌营养不良症和贝克型肌营养不良症的具有改善特性的rna调节性寡核苷酸
SMT201700339T1 (it) 2012-04-23 2017-09-07 Biomarin Tech Bv Oligonucleotidi di modulazione dell'rna con caratteristiche migliorate per il trattamento dei disturbi neuromuscolari
CA2873769A1 (en) 2012-05-16 2013-11-21 Rana Therapeutics Inc. Compositions and methods for modulating hemoglobin gene family expression
WO2013173652A1 (en) 2012-05-16 2013-11-21 Rana Therapeutics, Inc. Compositions and methods for modulating gene expression
AU2013262709A1 (en) 2012-05-16 2015-01-22 Rana Therapeutics, Inc. Compositions and methods for modulating MECP2 expression
JP2015523854A (ja) 2012-05-16 2015-08-20 ラナ セラピューティクス インコーポレイテッド Smn遺伝子ファミリー発現を調節するための組成物及び方法
US10837014B2 (en) 2012-05-16 2020-11-17 Translate Bio Ma, Inc. Compositions and methods for modulating SMN gene family expression
BR112014028634A2 (pt) 2012-05-16 2017-06-27 Rana Therapeutics Inc composições e métodos para modulação da expressão de utrn
CA2873766A1 (en) 2012-05-16 2013-11-21 Rana Therapeutics Inc. Compositions and methods for modulating atp2a2 expression
ES2809199T3 (es) * 2012-06-25 2021-03-03 Ionis Pharmaceuticals Inc Modulación de la expresión de UBE3A-ATS
WO2014031881A2 (en) * 2012-08-22 2014-02-27 Lipovich Leonard Activity-dependent gene pairs as therapeutic targets and methods and devices to identify the same
EP2895200B1 (en) 2012-09-14 2019-11-06 Translate Bio MA, Inc. Multimeric oligonucleotide compounds
US9175291B2 (en) * 2012-10-11 2015-11-03 Isis Pharmaceuticals Inc. Modulation of androgen receptor expression
HK1212597A1 (zh) 2012-10-15 2016-06-17 Isis Pharmaceuticals, Inc. 用於調節c90rf72表達的組合物
WO2014062736A1 (en) 2012-10-15 2014-04-24 Isis Pharmaceuticals, Inc. Methods for monitoring c9orf72 expression
ES2762326T5 (es) 2012-10-15 2023-04-27 Ionis Pharmaceuticals Inc Métodos para modular la expresión de C9ORF72
KR101507505B1 (ko) * 2013-04-18 2015-04-07 사회복지법인 삼성생명공익재단 제 1 형 근긴장성 이영양증의 진단 방법
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
JP2016531570A (ja) 2013-08-16 2016-10-13 ラナ セラピューティクス インコーポレイテッド ユークロマチン領域を標的とするオリゴヌクレオチド
CA2921457A1 (en) * 2013-08-16 2015-02-19 Rana Therapeutics, Inc. Heterochromatin forming non-coding rnas
DK3043827T3 (da) * 2013-09-13 2019-08-26 Ionis Pharmaceuticals Inc Modulatorer af komplement faktor b
WO2015047512A1 (en) * 2013-09-25 2015-04-02 The University Of Chicago Inhibitors of cacna1a/alpha1a subunit internal ribosomal entry site (ires) and methods of treating spinocerebellar ataxia type 6
US10174328B2 (en) 2013-10-04 2019-01-08 Translate Bio Ma, Inc. Compositions and methods for treating amyotrophic lateral sclerosis
EP4166667A3 (en) 2013-10-11 2023-08-02 Ionis Pharmaceuticals, Inc. Compositions for modulating c9orf72 expression
US11162096B2 (en) 2013-10-14 2021-11-02 Ionis Pharmaceuticals, Inc Methods for modulating expression of C9ORF72 antisense transcript
US9783803B2 (en) 2014-02-07 2017-10-10 Vib Vzw Inhibition of NEAT1 for treatment of solid tumors
CN106103717A (zh) 2014-03-19 2016-11-09 Ionis制药公司 用于调节共济失调蛋白2表达的组合物
WO2015143245A1 (en) * 2014-03-19 2015-09-24 Isis Pharmaceuticals, Inc. Methods for modulating ataxin 2 expression
WO2015161189A1 (en) * 2014-04-17 2015-10-22 Cornell University Neat1 as a prognostic marker and therapeutic target for prostate cancer
BR112017004056A2 (pt) 2014-09-12 2017-12-05 Biogen Ma Inc composições e métodos para detecção da proteína smn em um indivíduo e tratamento de um indivíduo
CA2966044A1 (en) 2014-10-30 2016-05-06 The General Hospital Corporation Methods for modulating atrx-dependent gene repression
WO2016086104A1 (en) 2014-11-25 2016-06-02 Ionis Pharmaceuticals, Inc. Modulation of ube3a-ats expression
WO2016130943A1 (en) 2015-02-13 2016-08-18 Rana Therapeutics, Inc. Hybrid oligonucleotides and uses thereof
WO2016149455A2 (en) 2015-03-17 2016-09-22 The General Hospital Corporation The rna interactome of polycomb repressive complex 1 (prc1)
RS60230B1 (sr) 2015-04-16 2020-06-30 Ionis Pharmaceuticals Inc Kompozicije za moduliranje ekspresije c9orf72
CN105586399A (zh) * 2015-09-07 2016-05-18 张国新 一种与胃癌相关的血清/血浆lncRNA标志物试剂盒
EP3353306A4 (en) 2015-09-25 2019-06-05 Ionis Pharmaceuticals, Inc. Conjugated antisense compounds and their use
US10533175B2 (en) 2015-09-25 2020-01-14 Ionis Pharmaceuticals, Inc. Compositions and methods for modulating Ataxin 3 expression
WO2017070151A1 (en) * 2015-10-19 2017-04-27 Rxi Pharmaceuticals Corporation Reduced size self-delivering nucleic acid compounds targeting long non-coding rna
US11260073B2 (en) 2015-11-02 2022-03-01 Ionis Pharmaceuticals, Inc. Compounds and methods for modulating C90RF72
HUE067905T2 (hu) 2015-11-12 2024-11-28 Hoffmann La Roche Oligonukleotidok paternális UBE3A expresszió indukálására
CN105331720A (zh) * 2015-11-27 2016-02-17 首都医科大学宣武医院 检测dm致病基因突变的方法,及其引物、试剂盒
EA201800367A1 (ru) 2015-12-10 2019-02-28 ПиТиСи ТЕРАПЬЮТИКС, ИНК. Способы лечения болезни хантингтона
US20190002887A1 (en) 2015-12-31 2019-01-03 Ionis Pharmaceuticals, Inc. Methods for reducing ataxin-2 expression
JP2019513371A (ja) 2016-04-01 2019-05-30 アビディティー バイオサイエンシーズ エルエルシー 核酸ポリペプチド組成物とその使用
WO2017218884A1 (en) 2016-06-16 2017-12-21 Ionis Pharmaceuticals, Inc. Combinations for the modulation of smn expression
US11427838B2 (en) * 2016-06-29 2022-08-30 Vertex Pharmaceuticals Incorporated Materials and methods for treatment of myotonic dystrophy type 1 (DM1) and other related disorders
JP7211933B2 (ja) * 2016-07-15 2023-01-24 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 転写プロセシングの調節のための化合物及び方法
US20190330626A1 (en) * 2016-07-15 2019-10-31 Ionis Pharmaceuticals, Inc. Compounds and methods for use in dystrophin transcript
ES2935658T3 (es) * 2016-07-15 2023-03-09 Ionis Pharmaceuticals Inc Compuestos y métodos para la modulación de SMN2
ES2659845B1 (es) * 2016-09-19 2019-01-04 Univ Valencia Modulación de microRNAs contra la distrofia miotónica tipo 1 y antagonistas de microRNAs para ello
JOP20190104A1 (ar) * 2016-11-10 2019-05-07 Ionis Pharmaceuticals Inc مركبات وطرق لتقليل التعبير عن atxn3
EP4035659A1 (en) 2016-11-29 2022-08-03 PureTech LYT, Inc. Exosomes for delivery of therapeutic agents
MX2019008199A (es) 2017-01-06 2019-11-25 Avidity Biosciences Llc Composiciones de acido nucleico polipeptido y metodos de induccion de la omision de exon.
US20200131231A1 (en) 2017-02-17 2020-04-30 Oxford University Innovation Limited Cell penetrating peptides
WO2018165541A1 (en) * 2017-03-10 2018-09-13 The Board Of Regents Of The University Of Texas System Treatment of fuchs' endothelial corneal dystrophy
US11359200B2 (en) * 2017-04-09 2022-06-14 The Cleveland Clinic Foundation Cancer treatment by MALAT1 inhibition
EP3634953B1 (en) 2017-06-05 2024-01-03 PTC Therapeutics, Inc. Compounds for treating huntington's disease
WO2018226788A1 (en) 2017-06-07 2018-12-13 University Of Massachusetts Anti-adam33 oligonucleotides and related methods
MX2019015580A (es) 2017-06-28 2020-07-28 Ptc Therapeutics Inc Metodos para tratar la enfermedad de huntington.
CN111182898B (zh) 2017-06-28 2024-04-16 Ptc医疗公司 用于治疗亨廷顿氏病的方法
CN107446012B (zh) * 2017-07-10 2020-06-05 浙江大学 一类肿瘤荧光显像剂及制备和应用
GB201711809D0 (en) 2017-07-21 2017-09-06 Governors Of The Univ Of Alberta Antisense oligonucleotide
JP7221275B2 (ja) 2017-08-03 2023-02-13 ボイジャー セラピューティクス インコーポレイテッド Aavを送達するための組成物および方法
JP7506601B2 (ja) 2017-12-06 2024-06-26 アビディティー バイオサイエンシーズ,インク. 筋萎縮症と筋緊張性ジストロフィーを処置するための組成物および方法
EA202092001A1 (ru) 2018-03-27 2021-01-29 ПиТиСи ТЕРАПЬЮТИКС, ИНК. Соединения для лечения болезни гентингтона
SG11202010215TA (en) 2018-05-09 2020-11-27 Ionis Pharmaceuticals Inc Compounds and methods for reducing atxn3 expression
SG11202011151VA (en) * 2018-05-15 2020-12-30 Univ Washington Compositions and methods for reducing spliceopathy and treating rna dominance disorders
WO2019233922A1 (en) 2018-06-05 2019-12-12 F. Hoffmann-La Roche Ag Oligonucleotides for modulating atxn2 expression
WO2020005877A1 (en) 2018-06-27 2020-01-02 Ptc Therapeutics, Inc. Heteroaryl compounds for treating huntington's disease
HRP20240935T1 (hr) 2018-06-27 2024-11-22 Ptc Therapeutics, Inc. Heterociklični i heteroaril spojevi za liječenje huntingtonove bolesti
BR112021000308A2 (pt) 2018-07-25 2021-04-13 Ionis Pharmaceuticals, Inc. Compostos e métodos para redução da expressão de atxn2
US11911484B2 (en) 2018-08-02 2024-02-27 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating myotonic dystrophy
US12018087B2 (en) 2018-08-02 2024-06-25 Dyne Therapeutics, Inc. Muscle-targeting complexes comprising an anti-transferrin receptor antibody linked to an oligonucleotide and methods of delivering oligonucleotide to a subject
US20220193250A1 (en) 2018-08-02 2022-06-23 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating facioscapulohumeral muscular dystrophy
EP3829595A4 (en) 2018-08-02 2022-08-24 Dyne Therapeutics, Inc. MUSCLE-DIRECTED COMPLEXES AND USES THEREOF IN THE TREATMENT OF DYSTROPHINOPATHIES
US12370264B1 (en) 2018-08-02 2025-07-29 Dyne Therapeutics, Inc. Complexes comprising an anti-transferrin receptor antibody linked to an oligonucleotide and method of delivering oligonucleotide to a subject
IL321905A (en) * 2018-08-02 2025-09-01 Dyne Therapeutics Inc Muscle-targeted conjugates and their uses in the treatment of myotonia dystrophica
US12097263B2 (en) 2018-08-02 2024-09-24 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating myotonic dystrophy
GB201812980D0 (en) 2018-08-09 2018-09-26 Univ Oxford Innovation Ltd Cell-penetrating peptides
GB201812972D0 (en) 2018-08-09 2018-09-26 Univ Oxford Innovation Ltd Cell-penetrating peptides
EP3841209A4 (en) * 2018-08-22 2023-01-04 Research Institute at Nationwide Children's Hospital RECOMBINANT VIRUS PRODUCTS AND METHODS FOR INHIBITING DYSTROPHIA MYOTONICA PROTEIN KINASE EXPRESSION AND/OR INTERFERING WITH A TRINUCLEOTIDE REPEAT EXPANSION IN THE 3'-UNTRANSLATED REGION OF THE DMPK GENE
WO2020104295A1 (en) * 2018-11-19 2020-05-28 Uniqure Ip B.V. Rnai induced reduction of ataxin-3 for the treatment of spinocerebellar ataxia type 3
CN113453723A (zh) 2018-12-07 2021-09-28 牛津大学科技创新有限公司 接头
IL312067B2 (en) 2018-12-21 2025-08-01 Avidity Biosciences Inc Anti-transferrin receptor antibodies and uses thereof
TW202540426A (zh) 2019-02-27 2025-10-16 美商Ionis製藥公司 Malat1表現之調節劑
FI3947684T3 (fi) 2019-03-29 2025-05-27 Ionis Pharmaceuticals Inc Yhdisteitä ja menetelmiä ube3a-ats:n moduloimiseksi
US11286485B2 (en) 2019-04-04 2022-03-29 Hoffmann-La Roche Inc. Oligonucleotides for modulating ATXN2 expression
CN111826432A (zh) * 2019-04-18 2020-10-27 南京大学 细胞去服务化状态的分子标记物检测及其调控方法
US12497615B2 (en) 2019-04-25 2025-12-16 Avidity Biosciences, Inc. Nucleic acid compositions and methods of multi-exon skipping
JP7649257B2 (ja) 2019-05-13 2025-03-19 ピーティーシー セラピューティクス, インコーポレイテッド ハンチントン病を処置するための化合物
CA3135271A1 (en) * 2019-05-28 2020-12-03 Astellas Pharma Inc. Method for treating muscular dystrophy by targeting dmpk gene
EP3956450B1 (en) * 2019-07-26 2025-08-13 Ionis Pharmaceuticals, Inc. Compounds and methods for modulating gfap
US20230141661A1 (en) * 2019-08-21 2023-05-11 H. Lee Moffitt Cancer Center And Research Institute, Inc. Compositions and methods for treating chronic myelomonocytic leukemia
TW202131920A (zh) 2019-11-01 2021-09-01 瑞士商諾華公司 剪接調節子用於減慢杭丁頓氏舞蹈症進展的治療之用途
CN110876752B (zh) * 2019-11-18 2021-04-30 暨南大学 长链非编码rna nron功能基序在制备抑制骨吸收及防治骨质疏松药物中的应用
CR20220485A (es) 2020-02-28 2022-11-10 Ionis Pharmaceuticals Inc Compuestos y métodos para modular smn2
US11555190B2 (en) 2020-03-19 2023-01-17 Avidity Biosciences, Inc. Compositions and methods of treating Facioscapulohumeral muscular dystrophy
AU2021241682B2 (en) * 2020-03-27 2026-02-12 Avidity Biosciences, Inc. Compositions and methods of treating muscle dystrophy
MX2022013707A (es) 2020-05-01 2022-12-07 Ionis Pharmaceuticals Inc Compuestos y metodos para modular atxn1.
CN114075598B (zh) * 2020-08-12 2023-09-19 复旦大学附属华山医院 一种萎缩性肌强直蛋白激酶基因ctg区域的pcr检测试剂盒及应用
US20240117356A1 (en) 2020-12-31 2024-04-11 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating myotonic dystrophy
CN114790478B (zh) * 2021-01-26 2025-09-30 上海昂朴生物科技有限公司 一种萎缩性肌强直蛋白激酶基因ctg区域的pcr检测试剂盒及应用
US20240189434A1 (en) * 2021-03-12 2024-06-13 Pepgen Inc. Methods of treating myotonic dystrophy type 1 using peptide-oligonucleotide conjugates
TW202304446A (zh) 2021-03-29 2023-02-01 瑞士商諾華公司 剪接調節子用於減慢杭丁頓氏舞蹈症進展的治療之用途
US11969475B2 (en) 2021-07-09 2024-04-30 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating facioscapulohumeral muscular dystrophy
US11638761B2 (en) 2021-07-09 2023-05-02 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating Facioscapulohumeral muscular dystrophy
US11633498B2 (en) 2021-07-09 2023-04-25 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating myotonic dystrophy
EP4396352A4 (en) 2021-09-01 2025-10-01 Ionis Pharmaceuticals Inc COMPOUNDS AND METHODS FOR REDUCING DMPK EXPRESSION
MX2024003266A (es) 2021-09-16 2024-04-03 Avidity Biosciences Inc Composiciones y metodos de tratamiento de la distrofia muscular facioescapulohumeral.
MX2024007908A (es) * 2021-12-23 2024-09-10 Univ Massachusetts Tratamiento terapeutico para trastorno asociado con x fragil.
US12071621B2 (en) 2022-04-05 2024-08-27 Avidity Biosciences, Inc. Anti-transferrin receptor antibody-PMO conjugates for inducing DMD exon 44 skipping
US20250339553A1 (en) 2022-04-15 2025-11-06 Dyne Therapeutics, Inc. Muscle targeting complexes and formulations for treating myotonic dystrophy
WO2024063570A1 (ko) * 2022-09-23 2024-03-28 주식회사 셀리버리 근긴장성 이영양증 단백질 키나아제를 표적화하는 세포투과성 펩티드 융합 안티센스 올리고뉴클레오티드 및 이의 용도
CN116059370B (zh) * 2022-09-28 2025-03-28 南京医科大学 敲低或抑制长链非编码rna jpx的物质的医药用途
EP4692348A1 (en) * 2024-08-07 2026-02-11 Nocturna Therapeutics SL Artificial rnas for treating repeat expansion disorders

Family Cites Families (139)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US2699808A (en) 1944-10-06 1955-01-18 Mark W Lowe Apparatus for peeling tomatoes
US2699805A (en) 1951-10-23 1955-01-18 Richter Albert Guiding attachment for hand planes
US2699508A (en) 1951-12-21 1955-01-11 Selectronics Inc Method of mounting and construction of mounting for low frequency piezoelectric crystals
US2699504A (en) 1952-06-28 1955-01-11 Itt Automatic tuning device
US3687808A (en) 1969-08-14 1972-08-29 Univ Leland Stanford Junior Synthetic polynucleotides
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
US5367066A (en) 1984-10-16 1994-11-22 Chiron Corporation Oligonucleotides with selectably cleavable and/or abasic sites
FR2575751B1 (fr) 1985-01-08 1987-04-03 Pasteur Institut Nouveaux nucleosides de derives de l'adenosine, leur preparation et leurs applications biologiques
AU598946B2 (en) 1987-06-24 1990-07-05 Howard Florey Institute Of Experimental Physiology And Medicine Nucleoside derivatives
US5175273A (en) 1988-07-01 1992-12-29 Genentech, Inc. Nucleic acid intercalating agents
US5134066A (en) 1989-08-29 1992-07-28 Monsanto Company Improved probes using nucleosides containing 3-dezauracil analogs
US5591722A (en) 1989-09-15 1997-01-07 Southern Research Institute 2'-deoxy-4'-thioribonucleosides and their antiviral activity
WO1991004753A1 (en) 1989-10-02 1991-04-18 Cetus Corporation Conjugates of antisense oligonucleotides and therapeutic uses thereof
ATE269870T1 (de) 1989-10-24 2004-07-15 Isis Pharmaceuticals Inc 2'-modifizierte oligonukleotide
US5130302A (en) 1989-12-20 1992-07-14 Boron Bilogicals, Inc. Boronated nucleoside, nucleotide and oligonucleotide compounds, compositions and methods for using same
US5681941A (en) 1990-01-11 1997-10-28 Isis Pharmaceuticals, Inc. Substituted purines and oligonucleotide cross-linking
US5646265A (en) 1990-01-11 1997-07-08 Isis Pharmceuticals, Inc. Process for the preparation of 2'-O-alkyl purine phosphoramidites
US5459255A (en) 1990-01-11 1995-10-17 Isis Pharmaceuticals, Inc. N-2 substituted purines
US5670633A (en) 1990-01-11 1997-09-23 Isis Pharmaceuticals, Inc. Sugar modified oligonucleotides that detect and modulate gene expression
US5587470A (en) 1990-01-11 1996-12-24 Isis Pharmaceuticals, Inc. 3-deazapurines
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.
BR9106702A (pt) 1990-07-27 1993-06-08 Isis Pharmaceuticals Inc Analogo de oligonucleotideos e processos para modular a producao de uma proteina por um organismo e para tratar um organismo
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
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
US5594121A (en) 1991-11-07 1997-01-14 Gilead Sciences, Inc. Enhanced triple-helix and double-helix formation with oligomers containing modified purines
AU3222793A (en) 1991-11-26 1993-06-28 Gilead Sciences, Inc. Enhanced triple-helix and double-helix formation with oligomers containing modified pyrimidines
TW393513B (en) 1991-11-26 2000-06-11 Isis Pharmaceuticals Inc Enhanced triple-helix and double-helix formation with oligomers containing modified pyrimidines
US5484908A (en) 1991-11-26 1996-01-16 Gilead Sciences, Inc. Oligonucleotides containing 5-propynyl pyrimidines
US5359044A (en) 1991-12-13 1994-10-25 Isis Pharmaceuticals Cyclobutyl oligonucleotide surrogates
FR2687679B1 (fr) 1992-02-05 1994-10-28 Centre Nat Rech Scient Oligothionucleotides.
US5955265A (en) * 1992-02-06 1999-09-21 Massachusetts Institute Of Technology DNA sequence encoding the myotonic dystrophy gene and uses thereof
US5434257A (en) 1992-06-01 1995-07-18 Gilead Sciences, Inc. Binding compentent oligomers containing unsaturated 3',5' and 2',5' linkages
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
EP0673559A1 (en) 1992-12-14 1995-09-27 Honeywell Inc. Motor system with individually controlled redundant windings
US5552282A (en) 1993-02-19 1996-09-03 Baylor College Of Medicine Diagnosis of myotonic muscular dystrophy
AU6449394A (en) 1993-03-30 1994-10-24 Sterling Winthrop Inc. Acyclic nucleoside analogs and oligonucleotide sequences containing them
US5502177A (en) 1993-09-17 1996-03-26 Gilead Sciences, Inc. Pyrimidine derivatives for labeled binding partners
US5801154A (en) 1993-10-18 1998-09-01 Isis Pharmaceuticals, Inc. Antisense oligonucleotide modulation of multidrug resistance-associated protein
US5457187A (en) 1993-12-08 1995-10-10 Board Of Regents University Of Nebraska Oligonucleotides containing 5-fluorouracil
US5446137B1 (en) 1993-12-09 1998-10-06 Behringwerke Ag Oligonucleotides containing 4'-substituted nucleotides
JP3345700B2 (ja) 1994-01-11 2002-11-18 株式会社リコー 電子写真用感光体
US5519134A (en) 1994-01-11 1996-05-21 Isis Pharmaceuticals, Inc. Pyrrolidine-containing monomers and oligomers
US5596091A (en) 1994-03-18 1997-01-21 The Regents Of The University Of California Antisense oligonucleotides comprising 5-aminoalkyl pyrimidine nucleotides
US5627053A (en) 1994-03-29 1997-05-06 Ribozyme Pharmaceuticals, Inc. 2'deoxy-2'-alkylnucleotide containing nucleic acid
US5646269A (en) 1994-04-28 1997-07-08 Gilead Sciences, Inc. Method for oligonucleotide analog synthesis
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
US5597909A (en) 1994-08-25 1997-01-28 Chiron Corporation Polynucleotide reagents containing modified deoxyribose moieties, and associated methods of synthesis and use
US5656408A (en) 1996-04-29 1997-08-12 Xerox Corporation Coated carrier particles
US9096636B2 (en) 1996-06-06 2015-08-04 Isis Pharmaceuticals, Inc. Chimeric oligomeric compounds and their use in gene modulation
US6770748B2 (en) 1997-03-07 2004-08-03 Takeshi Imanishi Bicyclonucleoside and oligonucleotide analogue
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
USRE44779E1 (en) 1997-03-07 2014-02-25 Santaris Pharma A/S Bicyclonucleoside and oligonucleotide analogues
US6329501B1 (en) * 1997-05-29 2001-12-11 Auburn University Methods and compositions for targeting compounds to muscle
US6794499B2 (en) 1997-09-12 2004-09-21 Exiqon A/S Oligonucleotide analogues
AU9063398A (en) 1997-09-12 1999-04-05 Exiqon A/S Oligonucleotide analogues
US6028183A (en) 1997-11-07 2000-02-22 Gilead Sciences, Inc. Pyrimidine derivatives and oligonucleotides containing same
US6007992A (en) 1997-11-10 1999-12-28 Gilead Sciences, Inc. Pyrimidine derivatives for labeled binding partners
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
US6107092A (en) * 1999-03-29 2000-08-22 Isis Pharmaceuticals Inc. Antisense modulation of SRA expression
EP1178999B1 (en) 1999-05-04 2007-03-14 Santaris Pharma A/S L-ribo-lna analogues
US6525191B1 (en) 1999-05-11 2003-02-25 Kanda S. Ramasamy Conformationally constrained L-nucleosides
JP2003509030A (ja) 1999-09-17 2003-03-11 アイシス・ファーマシューティカルス・インコーポレーテッド トランスフォーミング成長因子−β発現のアンチセンスオリゴヌクレオチド調節
ES2261270T3 (es) 1999-12-30 2006-11-16 K.U. LEUVEN RESEARCH & DEVELOPMENT Acidos nucleicos que contienen ciclohexeno.
AU2001250572A1 (en) 2000-04-07 2001-10-23 Epigenomics Ag Detection of single nucleotide polymorphisms (snp's) and cytosine-methylations
WO2002001953A1 (en) * 2000-06-30 2002-01-10 Brown University Research Foundation Methods to screen for antibiotic agents and their use in treatment of opportunistic infections
EP1409639A4 (en) * 2001-05-07 2006-06-07 Idera Pharmaceuticals Inc ANTISENSE OLIGONUCLEOTIDES OF THE EPIDERMAL GROWTH FACTOR RECEPTOR
US20030207804A1 (en) 2001-05-25 2003-11-06 Muthiah Manoharan Modified peptide nucleic acids
JP2005504020A (ja) 2001-07-03 2005-02-10 アイシス・ファーマシューティカルス・インコーポレーテッド ヌクレアーゼ耐性キメラオリゴヌクレオチド
US20030158403A1 (en) 2001-07-03 2003-08-21 Isis Pharmaceuticals, Inc. Nuclease resistant chimeric oligonucleotides
US7407943B2 (en) * 2001-08-01 2008-08-05 Isis Pharmaceuticals, Inc. Antisense modulation of apolipoprotein B expression
AU2002326589B2 (en) 2001-08-07 2008-06-05 University Of Delaware Compositions and methods for the prevention and treatment of Huntington's disease
US20060009409A1 (en) 2002-02-01 2006-01-12 Woolf Tod M Double-stranded oligonucleotides
AU2003270900A1 (en) * 2002-09-25 2004-04-19 Pharmacia Corporation Antisense modulation of microsomal prostaglandin e2 synthase expression
CA2504694C (en) 2002-11-05 2013-10-01 Isis Pharmaceuticals, Inc. Polycyclic sugar surrogate-containing oligomeric compounds and compositions for use in gene modulation
US8604183B2 (en) 2002-11-05 2013-12-10 Isis Pharmaceuticals, Inc. Compositions comprising alternating 2′-modified nucleosides for use in gene modulation
WO2006006948A2 (en) 2002-11-14 2006-01-19 Dharmacon, Inc. METHODS AND COMPOSITIONS FOR SELECTING siRNA OF IMPROVED FUNCTIONALITY
EP2305812A3 (en) 2002-11-14 2012-06-06 Dharmacon, Inc. Fuctional and hyperfunctional sirna
US20050019746A1 (en) 2003-01-23 2005-01-27 Eirx Therapeutics Limited Apoptosis-related kinase/GPCRs
US7598227B2 (en) * 2003-04-16 2009-10-06 Isis Pharmaceuticals Inc. Modulation of apolipoprotein C-III expression
US8158354B2 (en) 2003-05-13 2012-04-17 Ibis Biosciences, Inc. Methods for rapid purification of nucleic acids for subsequent analysis by mass spectrometry by solution capture
WO2004101787A1 (ja) 2003-05-14 2004-11-25 Japan Science And Technology Agency ハンチンチン遺伝子の発現抑制
WO2004106356A1 (en) 2003-05-27 2004-12-09 Syddansk Universitet Functionalized nucleotide derivatives
EP1661905B9 (en) 2003-08-28 2012-12-19 IMANISHI, Takeshi Novel artificial nucleic acids of n-o bond crosslinkage type
WO2005027962A1 (en) 2003-09-18 2005-03-31 Isis Pharmaceuticals, Inc. 4’-thionucleosides and oligomeric compounds
US7208174B2 (en) 2003-12-18 2007-04-24 Hoffmann-La Roche Inc. Liposome compositions
WO2005063983A1 (en) 2003-12-29 2005-07-14 Galapagos Genomics N.V. Modulators of bone homeostasis identified in a high-throughput screen
US7374927B2 (en) * 2004-05-03 2008-05-20 Affymetrix, Inc. Methods of analysis of degraded nucleic acid samples
EP1752536A4 (en) 2004-05-11 2008-04-16 Alphagen Co Ltd POLYNUCLEOTIDE CAUSING RNA INTERFERENCE AND METHOD OF REGULATING GENE EXPRESSION WITH THE USE OF THE SAME
EP1766052A4 (en) 2004-06-03 2009-12-16 Isis Pharmaceuticals Inc OLIGOMERIC CHIMERIC COMPOSITIONS WITH BRECH
AU2005252663B2 (en) 2004-06-03 2011-07-07 Isis Pharmaceuticals, Inc. Double strand compositions comprising differentially modified strands for use in gene modulation
EP2397563A3 (en) * 2004-09-17 2012-07-18 Isis Pharmaceuticals, Inc. Enhanced antisense oligonucleotides
US20080261905A1 (en) 2004-11-08 2008-10-23 K.U. Leuven Research And Development Modified Nucleosides for Rna Interference
PT2161038E (pt) 2006-01-26 2014-03-10 Isis Pharmaceuticals Inc Composições e suas utilizações dirigidas à huntingtina
JP5342881B2 (ja) 2006-01-27 2013-11-13 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 6−修飾された二環式核酸類似体
US8178503B2 (en) * 2006-03-03 2012-05-15 International Business Machines Corporation Ribonucleic acid interference molecules and binding sites derived by analyzing intergenic and intronic regions of genomes
US7901882B2 (en) * 2006-03-31 2011-03-08 Affymetrix, Inc. Analysis of methylation using nucleic acid arrays
US8158364B2 (en) * 2006-04-11 2012-04-17 The Board Of Regents Of The University Of Texas System Methods and compositions involving nucleotide repeat disorders
AU2007249349B2 (en) 2006-05-11 2012-03-08 Isis Pharmaceuticals, Inc. 5'-Modified bicyclic nucleic acid analogs
CN101501193B (zh) * 2006-08-11 2013-07-03 普罗森那技术公司 用于治疗与dna重复不稳定性相关的遗传病的方法和手段
AU2007282224B2 (en) * 2006-08-11 2013-08-29 Vico Therapeutics B.V. Methods and means for treating DNA repeat instability associated genetic disorders
EP3034083B1 (en) 2006-09-21 2020-12-09 University of Rochester Antisense oligonucleotides for use in treating myotonic dystrophy
WO2008049085A1 (en) * 2006-10-18 2008-04-24 Isis Pharmaceuticals, Inc. Antisense compounds
AU2007347689B2 (en) * 2006-12-20 2013-08-15 Novarx Universal tumor cell vaccine for anti cancer therapeutic and prophylactic utilization
US20100190837A1 (en) 2007-02-15 2010-07-29 Isis Pharmaceuticals, Inc. 5'-Substituted-2-F' Modified Nucleosides and Oligomeric Compounds Prepared Therefrom
EP2170917B1 (en) 2007-05-30 2012-06-27 Isis Pharmaceuticals, Inc. N-substituted-aminomethylene bridged bicyclic nucleic acid analogs
EP2173760B2 (en) 2007-06-08 2015-11-04 Isis Pharmaceuticals, Inc. Carbocyclic bicyclic nucleic acid analogs
EP3443976A1 (en) * 2007-06-29 2019-02-20 Sarepta Therapeutics, Inc. Tissue specific peptide conjugates and methods
US20100016215A1 (en) * 2007-06-29 2010-01-21 Avi Biopharma, Inc. Compound and method for treating myotonic dystrophy
CA2692579C (en) 2007-07-05 2016-05-03 Isis Pharmaceuticals, Inc. 6-disubstituted bicyclic nucleic acid analogs
US9434997B2 (en) * 2007-08-24 2016-09-06 Lawrence Livermore National Security, Llc Methods, compounds and systems for detecting a microorganism in a sample
US7973019B1 (en) 2007-10-03 2011-07-05 Alcon Research, Ltd. Transferrin/transferrin receptor-mediated siRNA delivery
US8546556B2 (en) 2007-11-21 2013-10-01 Isis Pharmaceuticals, Inc Carbocyclic alpha-L-bicyclic nucleic acid analogs
EP2240768A1 (en) * 2008-02-04 2010-10-20 Galapagos N.V. Target sequences and methods to identify the same, useful in treatment of neurodegenerative diseases
NZ587178A (en) * 2008-02-08 2011-11-25 Prosensa Holding Bv Methods and means for treating dna repeat instability associated genetic disorders
WO2010014592A1 (en) * 2008-07-29 2010-02-04 The Board Of Regents Of The University Of Texas Sytem Selective inhibition of polyglutamine protein expression
GB0816778D0 (en) * 2008-09-12 2008-10-22 Isis Innovation Gene silencing
WO2010036698A1 (en) 2008-09-24 2010-04-01 Isis Pharmaceuticals, Inc. Substituted alpha-l-bicyclic nucleosides
US8604192B2 (en) 2008-09-24 2013-12-10 Isis Pharmaceuticals, Inc. Cyclohexenyl nucleic acids analogs
AT507215B1 (de) 2009-01-14 2010-03-15 Boehler Edelstahl Gmbh & Co Kg Verschleissbeständiger werkstoff
US8623107B2 (en) 2009-02-17 2014-01-07 Mcalister Technologies, Llc Gas hydrate conversion system for harvesting hydrocarbon hydrate deposits
US20120149756A1 (en) * 2009-04-10 2012-06-14 Associatin Institut de Myologie Tricyclo-dna antisense oligonucleotides, compositions, and methods for the treatment of disease
EP2462153B1 (en) 2009-08-06 2015-07-29 Isis Pharmaceuticals, Inc. Bicyclic cyclohexose nucleic acid analogs
WO2011097388A1 (en) 2010-02-03 2011-08-11 Alnylam Pharmaceuticals, Inc. Selective inhibition of polyglutamine protein expression
EP2536738A4 (en) 2010-02-08 2014-09-17 Isis Pharmaceuticals Inc METHODS AND COMPOSITIONS FOR THE TREATMENT OF DISEASES OR SUFFERING IN CONNECTION WITH REPEAT EXTENSIONS
CA2792696C (en) 2010-03-17 2020-01-07 Association Institut De Myologie Modified u7 snrnas for treatment of neuromuscular diseases
WO2012012467A2 (en) 2010-07-19 2012-01-26 Isis Pharmaceuticals, Inc. Modulation of nuclear-retained rna
US20150141320A1 (en) 2012-05-16 2015-05-21 Rana Therapeutics, Inc. Compositions and methods for modulating gene expression
BR112014028634A2 (pt) 2012-05-16 2017-06-27 Rana Therapeutics Inc composições e métodos para modulação da expressão de utrn
US9984408B1 (en) 2012-05-30 2018-05-29 Amazon Technologies, Inc. Method, medium, and system for live video cooperative shopping
WO2014120861A2 (en) 2013-01-31 2014-08-07 Isis Pharmaceuticals, Inc. Method of preparing oligomeric compounds using modified coupling protocols
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
US20210052631A1 (en) 2015-09-25 2021-02-25 Ionis Pharmaceuticals, Inc. Conjugated antisense compounds and their use
US9778708B1 (en) 2016-07-18 2017-10-03 Lenovo Enterprise Solutions (Singapore) Pte. Ltd. Dual sided latching retainer for computer modules
EP4396352A4 (en) 2021-09-01 2025-10-01 Ionis Pharmaceuticals Inc COMPOUNDS AND METHODS FOR REDUCING DMPK EXPRESSION
EP4396351A4 (en) 2021-09-01 2025-12-24 Ionis Pharmaceuticals Inc COMPOUNDS AND METHODS FOR REDUCING DMPK EXPRESSION

Also Published As

Publication number Publication date
CN106434648A (zh) 2017-02-22
JP6698740B2 (ja) 2020-05-27
IL223962B (en) 2018-03-29
IL257936A (en) 2018-05-31
AU2011282243A1 (en) 2013-02-21
US12480126B2 (en) 2025-11-25
WO2012012443A3 (en) 2013-05-23
CA2805791A1 (en) 2012-01-26
CN103167883A (zh) 2013-06-19
JP2013538561A (ja) 2013-10-17
US10526604B2 (en) 2020-01-07
HRP20191753T1 (hr) 2019-12-27
JP2018150344A (ja) 2018-09-27
US20160068845A1 (en) 2016-03-10
EP2595664A4 (en) 2014-09-17
EP3031920B1 (en) 2019-08-21
EP2595664B1 (en) 2018-10-17
EP3633038A3 (en) 2020-07-29
JP2017014235A (ja) 2017-01-19
WO2012012443A2 (en) 2012-01-26
JP6343308B2 (ja) 2018-06-13
KR101900770B1 (ko) 2018-09-20
PL3031920T3 (pl) 2020-01-31
AU2011282243A2 (en) 2013-10-10
AU2018200825A1 (en) 2018-02-22
JP6059141B2 (ja) 2017-01-11
US20130237585A1 (en) 2013-09-12
EP2595663A4 (en) 2014-03-05
EP3633038A2 (en) 2020-04-08
WO2012012467A3 (en) 2012-04-26
EP3489360A2 (en) 2019-05-29
AU2016269447A1 (en) 2017-01-05
KR20130106811A (ko) 2013-09-30
US20180127758A1 (en) 2018-05-10
AU2016269447B2 (en) 2018-12-13
AU2020202315A1 (en) 2020-04-23
AU2016269447C1 (en) 2019-06-20
US9765338B2 (en) 2017-09-19
US20230174987A1 (en) 2023-06-08
EP3031920A1 (en) 2016-06-15
EP2595663A2 (en) 2013-05-29
US20200362353A1 (en) 2020-11-19
AU2011282243B2 (en) 2016-09-22
US20180163209A1 (en) 2018-06-14
EP2595664A2 (en) 2013-05-29
CA2805765A1 (en) 2012-01-26
US20130225659A1 (en) 2013-08-29
JP5981428B2 (ja) 2016-08-31
JP2020183379A (ja) 2020-11-12
EP3489360A3 (en) 2019-08-28
KR20180105730A (ko) 2018-09-28
AU2011282217A9 (en) 2015-12-03
JP2013538560A (ja) 2013-10-17
ES2756326T3 (es) 2020-04-27
WO2012012467A2 (en) 2012-01-26
AU2016201288A1 (en) 2016-03-31
CN103167883B (zh) 2016-08-03
AU2011282217A1 (en) 2013-02-21
AU2011282217B2 (en) 2015-12-03

Similar Documents

Publication Publication Date Title
DK3031920T3 (da) Modulation af dystrofi myotonica-protein kinase (dmpk) ekspression
CYD2019001I1 (el) Διαμορφωση της εκφρασης τρανσθυρετινης
IL225867B (en) Coelenterazine substrates
DK3461895T3 (da) Modulation af ube3a-ats-ekspression
DK3450434T3 (da) Deutererede derivater af ruxolitinib
DK2521750T3 (da) Hærdning af væsker
DK2603232T3 (da) Stabile formuleringer af linaclotid
DK3725778T4 (da) Formuleringer af enzalutamid
DK2560683T4 (da) Fremstilling af heteromultimeriske proteiner
EP2877458A4 (en) CYSTATHIONINE-Y-GAMMA-LYASE (CSE) -HEMMER
DK3329775T3 (da) Fremgangsmåder til syntese af diarylthiohydantoin- og diarylhydantoinforbindelser
DK2558105T3 (da) Bardoxolonmethyl til behandling af fedme
DK3517541T3 (da) Polymorf form af fluticasonpropionat
DK2874506T3 (da) Fremgangsmåde til fremstilling af enzymgranulater
DK2528602T4 (da) Formuleringer af bendamustin
DK2526425T3 (da) Fremgangsmåde til diagnosticering af endometriel receptivitet
DK2929999T3 (da) Fremstilling af afstivere til sammensatte ventilatorhuse
DK2595948T3 (da) Fremgangsmåde til fremstilling af ingenol-3-angelat
DK2459129T3 (da) Forbedringer af karstøtteanordninger
DK2822568T3 (da) Anvendelser af caseinsammensætninger
DK2260873T3 (da) Pcylering af proteiner
DK2833884T3 (da) Anvendelse af ccr3-inhibitorer
DK2802204T3 (da) Modulering af plantebiologi
DK2506840T3 (da) Anvendelser af hæmmere af hypoksi-fremkaldende faktorer
DK2870252T3 (da) Fremgangsmåder ved anvendelse af patatin