CN106434648A - 肌强直性营养障碍蛋白激酶(dmpk)表达的调节 - Google Patents

肌强直性营养障碍蛋白激酶(dmpk)表达的调节 Download PDF

Info

Publication number
CN106434648A
CN106434648A CN201610527770.0A CN201610527770A CN106434648A CN 106434648 A CN106434648 A CN 106434648A CN 201610527770 A CN201610527770 A CN 201610527770A CN 106434648 A CN106434648 A CN 106434648A
Authority
CN
China
Prior art keywords
dmpk
modified
antisense
compound
certain embodiments
Prior art date
Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
Pending
Application number
CN201610527770.0A
Other languages
English (en)
Chinese (zh)
Inventor
F·C·贝内特
苏珊·M·弗赖尔
R·A·马克雷德
桑杰·K·潘迪
查尔斯·A·桑顿
瑟曼·惠勒
S·H·程
安德鲁·雷吉尔
布鲁斯·M·文特沃斯
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Individual
Original Assignee
Individual
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=CN106434648(A) "Global patent litigation dataset” by Darts-ip is licensed under a Creative Commons Attribution 4.0 International License.
Application filed by Individual filed Critical Individual
Publication of CN106434648A publication Critical patent/CN106434648A/zh
Pending legal-status Critical Current

Links

Classifications

    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • C12N15/1137—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing against enzymes
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P15/00—Drugs for genital or sexual disorders; Contraceptives
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00—Drugs for disorders of the muscular or neuromuscular system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00—Drugs for disorders of the muscular or neuromuscular system
    • A61P21/04—Drugs for disorders of the muscular or neuromuscular system for myasthenia gravis
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/14—Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/18—Antipsychotics, i.e. neuroleptics; Drugs for mania or schizophrenia
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00—Antineoplastic agents
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y207/00—Transferases transferring phosphorus-containing groups (2.7)
    • C12Y207/11—Protein-serine/threonine kinases (2.7.11)
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/11—Antisense
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/315—Phosphorothioates
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/318—Chemical structure of the backbone where the PO2 is completely replaced, e.g. MMI or formacetal
    • C12N2310/3181—Peptide nucleic acid, PNA
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/321—2'-O-R Modification
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/322—2'-R Modification
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/323—Chemical structure of the sugar modified ring structure
    • C12N2310/3231—Chemical structure of the sugar modified ring structure having an additional ring, e.g. LNA, ENA
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/33—Chemical structure of the base
    • C12N2310/334—Modified C
    • C12N2310/3341—5-Methylcytosine
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/341—Gapmers, i.e. of the type ===---===
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/346—Spatial arrangement of the modifications having a combination of backbone and sugar modifications
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/352—Nature of the modification linked to the nucleic acid via a carbon atom
    • C12N2310/3525—MOE, 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)
  • Medicinal Chemistry (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Molecular Biology (AREA)
  • Biotechnology (AREA)
  • Biochemistry (AREA)
  • Neurology (AREA)
  • Biophysics (AREA)
  • Microbiology (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Virology (AREA)
  • Neurosurgery (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Endocrinology (AREA)
  • Psychiatry (AREA)
  • Reproductive Health (AREA)
  • Epidemiology (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)
CN201610527770.0A 2010-07-19 2011-07-19 肌强直性营养障碍蛋白激酶(dmpk)表达的调节 Pending CN106434648A (zh)

Applications Claiming Priority (7)

Application Number Priority Date Filing Date Title
US36577510P 2010-07-19 2010-07-19
US36576210P 2010-07-19 2010-07-19
US61/365,762 2010-07-19
US61/365,775 2010-07-19
US201161478021P 2011-04-21 2011-04-21
US61/478,021 2011-04-21
CN201180035573.2A CN103167883B (zh) 2010-07-19 2011-07-19 肌强直性营养障碍蛋白激酶(dmpk)表达的调节

Related Parent Applications (1)

Application Number Title Priority Date Filing Date
CN201180035573.2A Division CN103167883B (zh) 2010-07-19 2011-07-19 肌强直性营养障碍蛋白激酶(dmpk)表达的调节

Publications (1)

Publication Number Publication Date
CN106434648A true CN106434648A (zh) 2017-02-22

Family

ID=45497419

Family Applications (2)

Application Number Title Priority Date Filing Date
CN201610527770.0A Pending CN106434648A (zh) 2010-07-19 2011-07-19 肌强直性营养障碍蛋白激酶(dmpk)表达的调节
CN201180035573.2A Active CN103167883B (zh) 2010-07-19 2011-07-19 肌强直性营养障碍蛋白激酶(dmpk)表达的调节

Family Applications After (1)

Application Number Title Priority Date Filing Date
CN201180035573.2A Active CN103167883B (zh) 2010-07-19 2011-07-19 肌强直性营养障碍蛋白激酶(dmpk)表达的调节

Country Status (13)

Country Link
US (7) US20130225659A1 (enExample)
EP (5) EP2595663A4 (enExample)
JP (5) JP5981428B2 (enExample)
KR (2) KR20180105730A (enExample)
CN (2) CN106434648A (enExample)
AU (6) AU2011282243B2 (enExample)
CA (2) CA2805791A1 (enExample)
DK (1) DK3031920T3 (enExample)
ES (1) ES2756326T3 (enExample)
HR (1) HRP20191753T1 (enExample)
IL (2) IL223962B (enExample)
PL (1) PL3031920T3 (enExample)
WO (2) WO2012012467A2 (enExample)

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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

Families Citing this family (121)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
ES2373246T3 (es) 2006-08-11 2012-02-01 Prosensa Technologies B.V. Oligonucleótidos monocatenarios complementarios de elementos repetitivos para el tratamiento de trastornos genéticos asociados a la inestabilidad de repeticiones de adn.
HUE028662T2 (en) 2007-10-26 2016-12-28 Academisch Ziekenhuis Leiden Preparations and methods for controlling muscle disorders
AU2011282243B2 (en) 2010-07-19 2016-09-22 Ionis 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
WO2013033223A1 (en) 2011-08-29 2013-03-07 Isis Pharmaceuticals, Inc. Methods and compounds useful in conditions related to repeat expansion
EP3533873A1 (en) 2011-09-14 2019-09-04 Translate Bio MA, Inc. Multimeric oligonucleotide compounds
EP2794880B1 (en) * 2011-12-22 2018-05-02 Ionis Pharmaceuticals, Inc. Methods for modulating metastasis-associated-in-lung-adenocarcinoma-transcript-1(malat-1) expression
CN111893117B (zh) 2012-01-27 2024-06-04 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
HRP20171254T1 (hr) 2012-04-23 2018-01-12 Biomarin Technologies B.V. Oligonukleotidi koji moduliraju rnk s poboljšanim karakteristikama za liječenje neuromišićnih poremećaja
KR20160074368A (ko) 2012-05-16 2016-06-28 라나 테라퓨틱스, 인크. Utrn 발현을 조절하기 위한 조성물 및 방법
CA2873769A1 (en) 2012-05-16 2013-11-21 Rana Therapeutics Inc. Compositions and methods for modulating hemoglobin gene family expression
EP2850182A4 (en) 2012-05-16 2016-01-20 Rana Therapeutics Inc COMPOSITIONS AND METHODS FOR MODULATING ATP2A2 GENE EXPRESSION
DK2850186T3 (en) 2012-05-16 2019-04-08 Translate Bio Ma Inc COMPOSITIONS AND PROCEDURES FOR MODULATING SMN GENFAMILY EXPRESSION
AU2013262709A1 (en) 2012-05-16 2015-01-22 Rana Therapeutics, Inc. Compositions and methods for modulating MECP2 expression
US10837014B2 (en) 2012-05-16 2020-11-17 Translate Bio Ma, Inc. Compositions and methods for modulating SMN gene family expression
DK2850189T3 (en) 2012-05-16 2019-02-25 Translate Bio Ma Inc COMPOSITIONS AND PROCEDURES FOR MODULATING GENEPRESSION
EP4458361A3 (en) * 2012-06-25 2025-01-22 Ionis Pharmaceuticals, Inc. Modulation of ube3a-ats expression
US20150307937A1 (en) * 2012-08-22 2015-10-29 Wayne State University Activity-dependent gene pairs as therapeutic targets and methods and devices to identify the same
US20150247141A1 (en) 2012-09-14 2015-09-03 Rana Therapeutics, Inc. Multimeric oligonucleotide compounds
US9175291B2 (en) * 2012-10-11 2015-11-03 Isis Pharmaceuticals Inc. Modulation of androgen receptor expression
WO2014062686A1 (en) 2012-10-15 2014-04-24 Isis Pharmaceuticals, Inc. Methods for modulating c9orf72 expression
WO2014062736A1 (en) 2012-10-15 2014-04-24 Isis Pharmaceuticals, Inc. Methods for monitoring c9orf72 expression
AU2013331434B2 (en) * 2012-10-15 2019-08-08 Ionis Pharmaceuticals, Inc. Compositions for modulating C90RF72 expression
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
SI3043827T1 (sl) * 2013-09-13 2019-08-30 Ionis Pharmaceuticals, Inc. Modulatorji komplementnega faktorja 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
EP3052632A4 (en) 2013-10-04 2017-03-29 Rana Therapeutics, Inc. Compositions and methods for treating amyotrophic lateral sclerosis
RU2748426C2 (ru) 2013-10-11 2021-05-25 Ионис Фармасьютикалз, Инк. Композиции для модуляции экспрессии c90rf72
US11162096B2 (en) 2013-10-14 2021-11-02 Ionis Pharmaceuticals, Inc Methods for modulating expression of C9ORF72 antisense transcript
AU2015214141B2 (en) 2014-02-07 2020-07-30 Katholieke Universiteit Leuven, K.U.Leuven R&D Inhibition of NEAT1 for treatment of solid tumors
US10006027B2 (en) 2014-03-19 2018-06-26 Ionis Pharmaceuticals, Inc. Methods for modulating Ataxin 2 expression
PL3119888T3 (pl) 2014-03-19 2021-12-20 Ionis Pharmaceuticals, Inc. Kompozycje do modulacji ekspresji ataksyny 2
WO2015161189A1 (en) * 2014-04-17 2015-10-22 Cornell University Neat1 as a prognostic marker and therapeutic target for prostate cancer
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
WO2016086104A1 (en) 2014-11-25 2016-06-02 Ionis Pharmaceuticals, Inc. Modulation of ube3a-ats expression
US10758558B2 (en) 2015-02-13 2020-09-01 Translate Bio Ma, Inc. Hybrid oligonucleotides and uses thereof
EP3271460A4 (en) 2015-03-17 2019-03-13 The General Hospital Corporation RNA INTERACTOM OF POLYCOMB REPRESSIVE COMPLEX 1 (PRC1)
EP3722424A1 (en) * 2015-04-16 2020-10-14 Ionis Pharmaceuticals, Inc. Compositions for modulating c9orf72 expression
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
EP3353303B1 (en) 2015-09-25 2023-08-02 Ionis Pharmaceuticals, Inc. Compositions and methods for modulating ataxin 3 expression
JP2018528783A (ja) 2015-09-25 2018-10-04 アイオーニス ファーマシューティカルズ， インコーポレーテッドＩｏｎｉｓ Ｐｈａｒｍａｃｅｕｔｉｃａｌｓ，Ｉｎｃ． コンジュゲートアンチセンス化合物及びその使用
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
PL3374509T3 (pl) 2015-11-12 2021-05-04 F. Hoffmann-La Roche Ag Oligonukleotydy do indukowania ojcowskiej ekspresji UBE3A
KR102488323B1 (ko) 2015-12-10 2023-01-12 피티씨 테라퓨틱스, 인크. 헌팅턴병 치료 또는 개선을 위한 조성물
AU2016381174A1 (en) 2015-12-31 2018-05-31 Ionis Pharmaceuticals, Inc. Methods for reducing Ataxin-2 expression
MA45328A (fr) 2016-04-01 2019-02-06 Avidity Biosciences Llc Compositions acide nucléique-polypeptide et utilisations de celles-ci
US11198867B2 (en) 2016-06-16 2021-12-14 Ionis Pharmaceuticals, Inc. Combinations for the modulation of SMN expression
WO2018002812A1 (en) * 2016-06-29 2018-01-04 Crispr Therapeutics Ag Materials and methods for treatment of myotonic dystrophy type 1 (dm1) and other related disorders
EP4206213A1 (en) * 2016-07-15 2023-07-05 Ionis Pharmaceuticals, Inc. Compounds and methods for modulation of smn2
AU2017297624B2 (en) * 2016-07-15 2023-11-02 Ionis Pharmaceuticals, Inc. Compounds and methods for modulation of transcript processing
WO2018014042A1 (en) * 2016-07-15 2018-01-18 Ionis Pharmaceuticals, Inc. Compounds and methods for modulation of dystrophin transcript
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
CN110177544A (zh) 2016-11-29 2019-08-27 普尔泰克健康有限公司 用于递送治疗剂的外泌体
KR20190104381A (ko) 2017-01-06 2019-09-09 어비디티 바이오사이언시스 엘엘씨 핵산-폴리펩티드 조성물 및 엑손 스키핑을 유도하는 방법
US11512312B2 (en) 2017-03-10 2022-11-29 The Board Of Regents Of The University Of Texas System Treatment of Fuchs' endothelial corneal dystrophy
WO2018191153A1 (en) * 2017-04-09 2018-10-18 The Cleveland Clinic Foundation Cancer treatment by malat1 inhibition
KR20200033249A (ko) 2017-06-05 2020-03-27 피티씨 테라퓨틱스, 인크. 헌팅턴병 치료 화합물
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.
MX2019015578A (es) 2017-06-28 2020-07-28 Ptc Therapeutics Inc Metodos para tratar la enfermedad de huntington.
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
US12103926B2 (en) 2018-03-27 2024-10-01 Ptc Therapeutics, Inc. Compounds for treating huntington's disease
UY38225A (es) 2018-05-09 2019-11-29 Ionis Pharmaceuticals Inc Compuestos y métodos para reducir de la expresión de atxn3
MA51938B1 (fr) * 2018-05-15 2022-10-31 Univ Washington Compositions et procédés pour réduire les anomalies d'épissage et traiter des troubles de dominance arn
JP7379387B2 (ja) 2018-06-05 2023-11-14 エフ． ホフマン－ラ ロシュ アーゲー Ａｔｘｎ２発現を制御するためのオリゴヌクレオチド
AU2019294478B2 (en) 2018-06-27 2023-03-23 Ptc Therapeutics, Inc. Heterocyclic and heteroaryl compounds for treating Huntington's disease
WO2020005877A1 (en) 2018-06-27 2020-01-02 Ptc Therapeutics, Inc. Heteroaryl compounds for treating huntington's disease
EP3826645A4 (en) 2018-07-25 2023-05-17 Ionis Pharmaceuticals, Inc. COMPOUNDS AND METHODS FOR REDUCING ATXN2 EXPRESSION
SG11202100928QA (en) 2018-08-02 2021-02-25 Dyne Therapeutics Inc Muscle targeting complexes and uses thereof for treating facioscapulohumeral muscular dystrophy
SG11202100934PA (en) 2018-08-02 2021-02-25 Dyne Therapeutics Inc Muscle targeting complexes and uses thereof for treating 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
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
US12097263B2 (en) 2018-08-02 2024-09-24 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating myotonic dystrophy
US11911484B2 (en) 2018-08-02 2024-02-27 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating myotonic dystrophy
CN112955154A (zh) * 2018-08-02 2021-06-11 达因疗法公司 肌肉靶向复合物及其用于治疗强直性肌营养不良的用途
GB201812972D0 (en) 2018-08-09 2018-09-26 Univ Oxford Innovation Ltd Cell-penetrating peptides
GB201812980D0 (en) 2018-08-09 2018-09-26 Univ Oxford Innovation Ltd Cell-penetrating peptides
WO2020041634A1 (en) * 2018-08-22 2020-02-27 Research Institute At Nationwide Children's Hospital Recombinant virus products and methods for inhibiting expression of dystrophia myotonica protein kinase and/or interfering with a trinucleotide repeat expansion in the 3' untranslated region of the dmpk gene
AU2019385598A1 (en) * 2018-11-19 2021-06-03 Uniqure Ip B.V. RNAi induced reduction of ataxin-3 for the treatment of Spinocerebellar ataxia type 3
EP3890783A1 (en) 2018-12-07 2021-10-13 Oxford University Innovation Limited Linkers
IL319265A (en) 2018-12-21 2025-04-01 Avidity Biosciences Inc Anti-transferrin receptor antibodies and their uses
KR20210134686A (ko) * 2019-02-27 2021-11-10 아이오니스 파마수티컬즈, 인코포레이티드 Malat1 발현의 조절인자
TWI874376B (zh) 2019-03-29 2025-03-01 美商Ionis製藥公司 用於調節ube3a-ats 之化合物及方法
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
AU2020263487A1 (en) 2019-04-25 2021-12-16 Avidity Biosciences, Inc. Nucleic acid compositions and methods of multi-exon skipping
EP3976785A1 (en) * 2019-05-28 2022-04-06 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
WO2021035128A1 (en) * 2019-08-21 2021-02-25 H. Lee Moffitt Cancer Center And Research Institute Inc. Compositions and methods for treating chronic myelomonocytic leukemia
MX2022005254A (es) 2019-11-01 2022-06-29 Novartis Ag El uso de un modulador de empalme para un tratamiento que retrasa la progresion de la enfermedad de huntington.
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
BR112022016238A2 (pt) 2020-02-28 2022-10-11 Ionis Pharmaceuticals Inc Compostos e métodos para modular smn2
CA3172111A1 (en) 2020-03-19 2021-09-23 Barbora MALECOVA Compositions and methods of treating facioscapulohumeral muscular dystrophy
EP4656242A2 (en) 2020-03-27 2025-12-03 Avidity Biosciences, Inc. Compositions and methods of treating muscle dystrophy
CA3181546A1 (en) 2020-05-01 2021-11-04 Ionis Pharmaceuticals, Inc. Compounds and methods for modulating 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
EP4271816A4 (en) 2020-12-31 2025-04-02 Dyne Therapeutics, Inc. MUSCLE TARGETING COMPLEXES AND RELATED USES FOR THE TREATMENT OF 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
EP4304657A2 (en) * 2021-03-12 2024-01-17 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
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
US11969475B2 (en) 2021-07-09 2024-04-30 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating facioscapulohumeral muscular dystrophy
WO2023034870A2 (en) 2021-09-01 2023-03-09 Ionis Pharmaceuticals, Inc. Compounds and methods for reducing dmpk expression
CN118265544A (zh) 2021-09-16 2024-06-28 艾维迪提生物科学公司 治疗面肩肱型肌营养不良的组合物和方法
KR20240133725A (ko) * 2021-12-23 2024-09-04 유니버시티 오브 매사추세츠 취약 x 관련 장애의 치료용 치료제
US12071621B2 (en) 2022-04-05 2024-08-27 Avidity Biosciences, Inc. Anti-transferrin receptor antibody-PMO conjugates for inducing DMD exon 44 skipping
CA3255934A1 (en) 2022-04-15 2023-10-19 Dyne Therapeutics, Inc. MUSCLE TARGETTING COMPLEXES AND FORMULATIONS FOR THE TREATMENT OF 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

Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2004093783A2 (en) * 2003-04-16 2004-11-04 Isis Pharmaceuticals, Inc. Modulation of apolipoprotein c-iii expression
CN101501193A (zh) * 2006-08-11 2009-08-05 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
US20100016215A1 (en) * 2007-06-29 2010-01-21 Avi Biopharma, Inc. Compound and method for treating myotonic dystrophy

Family Cites Families (136)

* 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
CA1340032C (en) 1987-06-24 1998-09-08 Jim Haralambidis Lucleoside 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
ATE190981T1 (de) 1989-10-24 2000-04-15 Isis Pharmaceuticals Inc 2'-modifizierte nukleotide
US5130302A (en) 1989-12-20 1992-07-14 Boron Bilogicals, Inc. Boronated nucleoside, nucleotide and oligonucleotide compounds, compositions and methods for using same
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
US5646265A (en) 1990-01-11 1997-07-08 Isis Pharmceuticals, Inc. Process for the preparation of 2'-O-alkyl purine phosphoramidites
US5670633A (en) 1990-01-11 1997-09-23 Isis Pharmaceuticals, Inc. Sugar modified oligonucleotides that detect and modulate gene expression
US5681941A (en) 1990-01-11 1997-10-28 Isis Pharmaceuticals, Inc. Substituted purines and oligonucleotide cross-linking
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
DE69032425T2 (de) 1990-05-11 1998-11-26 Microprobe Corp., Bothell, Wash. Teststreifen zum Eintauchen für Nukleinsäure-Hybridisierungsassays und Verfahren zur kovalenten Immobilisierung von Oligonucleotiden
US5614617A (en) 1990-07-27 1997-03-25 Isis Pharmaceuticals, Inc. Nuclease resistant, pyrimidine modified oligonucleotides that detect and modulate gene expression
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
ES2103918T3 (es) 1991-10-17 1997-10-01 Ciba Geigy Ag Nucleosidos biciclicos, oligonucleotidos, procedimiento para su obtencion y productos intermedios.
US5594121A (en) 1991-11-07 1997-01-14 Gilead Sciences, Inc. Enhanced triple-helix and double-helix formation with oligomers containing modified purines
US5484908A (en) 1991-11-26 1996-01-16 Gilead Sciences, Inc. Oligonucleotides containing 5-propynyl pyrimidines
TW393513B (en) 1991-11-26 2000-06-11 Isis Pharmaceuticals Inc Enhanced triple-helix and double-helix formation with oligomers containing modified pyrimidines
JP3739785B2 (ja) 1991-11-26 2006-01-25 アイシス ファーマシューティカルズ，インコーポレイティド 修飾されたピリミジンを含有するオリゴマーを使用する増強された三重らせんおよび二重らせんの成形
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
DE69404289T2 (de) 1993-03-30 1998-02-19 Sanofi Sa Acyclische nucleosid analoge und sie enthaltende oligonucleotidsequenzen
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
US5519134A (en) 1994-01-11 1996-05-21 Isis Pharmaceuticals, Inc. Pyrrolidine-containing monomers and oligomers
JP3345700B2 (ja) 1994-01-11 2002-11-18 株式会社リコー 電子写真用感光体
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
WO2005121370A2 (en) 2004-06-03 2005-12-22 Isis Pharmaceuticals, Inc. Oligomeric compounds that facilitate risc loading
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
WO1999014226A2 (en) 1997-09-12 1999-03-25 Exiqon A/S Bi- and tri-cyclic nucleoside, nucleotide and oligonucleotide analogues
US6794499B2 (en) 1997-09-12 2004-09-21 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
CN102180924A (zh) 1999-05-04 2011-09-14 桑塔里斯制药公司 L-核糖-lna类似物
US6525191B1 (en) 1999-05-11 2003-02-25 Kanda S. Ramasamy Conformationally constrained L-nucleosides
US6436909B1 (en) 1999-09-17 2002-08-20 Isis Pharmaceuticals, Inc. Antisense inhibition of transforming growth factor-β expression
EP1244667B1 (en) 1999-12-30 2006-04-05 K.U. Leuven Research & Development Cyclohexene nucleic acids
US20040241651A1 (en) 2000-04-07 2004-12-02 Alexander Olek Detection of single nucleotide polymorphisms (snp's) and cytosine-methylations
AU2001268737A1 (en) * 2000-06-30 2002-01-14 Brown University Research Foundation Methods to screen for antibiotic agents and their use in treatment of opportunistic infections
US7671035B2 (en) * 2001-05-07 2010-03-02 Idera Pharmaceuticals, Inc. Epidermal growth factor receptor antisense oligonucleotides
US20030207804A1 (en) 2001-05-25 2003-11-06 Muthiah Manoharan Modified peptide nucleic acids
US20030158403A1 (en) 2001-07-03 2003-08-21 Isis Pharmaceuticals, Inc. Nuclease resistant chimeric oligonucleotides
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
US7407943B2 (en) * 2001-08-01 2008-08-05 Isis Pharmaceuticals, Inc. Antisense modulation of apolipoprotein B expression
EP1423537A4 (en) 2001-08-07 2006-11-29 Univ Delaware COMPOSITIONS AND METHODS FOR PREVENTING AND TREATING HUNTINGTON'S DISEASE
US20060009409A1 (en) 2002-02-01 2006-01-12 Woolf Tod M Double-stranded oligonucleotides
US20040132063A1 (en) * 2002-09-25 2004-07-08 Gierse James K. Antisense modulation of microsomal prostaglandin E2 synthase expression
WO2004044136A2 (en) 2002-11-05 2004-05-27 Isis Pharmaceuticals, Inc. Compositions comprising alternating 2’-modified nucleosides for use in gene modulation
AU2003291753B2 (en) 2002-11-05 2010-07-08 Isis Pharmaceuticals, Inc. Polycyclic sugar surrogate-containing oligomeric compounds and compositions for use in gene modulation
WO2006006948A2 (en) 2002-11-14 2006-01-19 Dharmacon, Inc. METHODS AND COMPOSITIONS FOR SELECTING siRNA OF IMPROVED FUNCTIONALITY
EP1560931B1 (en) 2002-11-14 2011-07-27 Dharmacon, Inc. Functional and hyperfunctional sirna
US20050019746A1 (en) 2003-01-23 2005-01-27 Eirx Therapeutics Limited Apoptosis-related kinase/GPCRs
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
US7589189B2 (en) 2003-05-14 2009-09-15 Japan Science And Technology Agency Inhibition of the expression of huntingtin gene
WO2004106356A1 (en) 2003-05-27 2004-12-09 Syddansk Universitet Functionalized nucleotide derivatives
ATE555118T1 (de) 2003-08-28 2012-05-15 Takeshi Imanishi Neue synthetische nukleidsäuren vom typ mit quervernetzter n-o-bindung
JP5379347B2 (ja) 2003-09-18 2013-12-25 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 ４’−チオヌクレオシドおよびオリゴマー化合物
US7208174B2 (en) 2003-12-18 2007-04-24 Hoffmann-La Roche Inc. Liposome compositions
AU2003300239A1 (en) 2003-12-29 2005-07-21 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
AU2004320622B2 (en) * 2004-06-03 2012-06-14 Isis Pharmaceuticals, Inc. Chimeric gapped oligomeric compositions
EP1799859B1 (en) * 2004-09-17 2014-07-02 Isis Pharmaceuticals, Inc. Enhanced antisense oligonucleotides
WO2006047842A2 (en) 2004-11-08 2006-05-11 K.U. Leuven Research And Development Modified nucleosides for rna interference
EP2161038B1 (en) * 2006-01-26 2013-12-25 Isis Pharmaceuticals, Inc. Compositions and their uses directed to Huntingtin
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
WO2007121272A2 (en) * 2006-04-11 2007-10-25 The Board Of Regents Of The University Of Texas System Methods and compositions involving nucleotide repeat disorders
US7547684B2 (en) 2006-05-11 2009-06-16 Isis Pharmaceuticals, Inc. 5′-modified bicyclic nucleic acid analogs
ES2373246T3 (es) * 2006-08-11 2012-02-01 Prosensa Technologies B.V. Oligonucleótidos monocatenarios complementarios de elementos repetitivos para el tratamiento de trastornos genéticos asociados a la inestabilidad de repeticiones de adn.
EP3034083B1 (en) 2006-09-21 2020-12-09 University of Rochester Antisense oligonucleotides for use in treating myotonic dystrophy
JP5665317B2 (ja) * 2006-10-18 2015-02-04 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 アンチセンス化合物
ES2547958T3 (es) * 2006-12-20 2015-10-09 Novarx Vacuna de célula tumoral universal para utilización terapéutica y profiláctica anticancerígena
WO2008101157A1 (en) 2007-02-15 2008-08-21 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
CA2691673A1 (en) * 2007-06-29 2009-01-08 Avi Biopharma, Inc. Tissue specific peptide conjugates and methods
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
WO2009067647A1 (en) 2007-11-21 2009-05-28 Isis Pharmaceuticals, Inc. Carbocyclic alpha-l-bicyclic nucleic acid analogs
JP2011515653A (ja) * 2008-02-04 2011-05-19 ガラパゴス・ナムローゼ・フェンノートシャップ 神経変性疾患の治療に有用な標的配列及びそれらの同定方法
WO2009099326A1 (en) * 2008-02-08 2009-08-13 Prosensa Holding Bv Methods and means for treating dna repeat instability associated genetic disorders
CA2732343C (en) * 2008-07-29 2017-05-09 The Board Of Regents Of The University Of Texas System Selective inhibition of polyglutamine protein expression
GB0816778D0 (en) * 2008-09-12 2008-10-22 Isis Innovation Gene silencing
EP2361256B1 (en) 2008-09-24 2013-04-10 Isis Pharmaceuticals, Inc. Cyclohexenyl nucleic acid analogs
WO2010036698A1 (en) 2008-09-24 2010-04-01 Isis Pharmaceuticals, Inc. Substituted alpha-l-bicyclic nucleosides
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
CN102625840A (zh) * 2009-04-10 2012-08-01 肌肉学研究协会 用于治疗疾病的三环dna反义寡核苷酸、组合物和方法
EP2462153B1 (en) 2009-08-06 2015-07-29 Isis Pharmaceuticals, Inc. Bicyclic cyclohexose nucleic acid analogs
US9574191B2 (en) 2010-02-03 2017-02-21 The Board Of Regents Of The University Of Texas System Selective inhibition of polyglutamine protein expression
US20130059902A1 (en) 2010-02-08 2013-03-07 Isis Pharmaceuticals, Inc. Methods and compositions useful in treatment of diseases or conditions related to repeat expansion
US9080170B2 (en) 2010-03-17 2015-07-14 Association Institut De Myologie Modified U7 snRNAs for treatment of neuromuscular diseases
AU2011282243B2 (en) * 2010-07-19 2016-09-22 Ionis Pharmaceuticals, Inc. Modulation of nuclear-retained RNA
KR20160074368A (ko) 2012-05-16 2016-06-28 라나 테라퓨틱스, 인크. Utrn 발현을 조절하기 위한 조성물 및 방법
WO2013173637A1 (en) 2012-05-16 2013-11-21 Rana Therapeutics, Inc. Compositions and methods for modulating gene expression
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
US20250177433A1 (en) 2021-09-01 2025-06-05 Ionis Pharmaceuticals, Inc. Compounds and methods for reducing dmpk expression
WO2023034870A2 (en) 2021-09-01 2023-03-09 Ionis Pharmaceuticals, Inc. Compounds and methods for reducing dmpk expression

Patent Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2004093783A2 (en) * 2003-04-16 2004-11-04 Isis Pharmaceuticals, Inc. Modulation of apolipoprotein c-iii expression
CN101501193A (zh) * 2006-08-11 2009-08-05 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
US20100016215A1 (en) * 2007-06-29 2010-01-21 Avi Biopharma, Inc. Compound and method for treating myotonic dystrophy

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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

Also Published As

Publication number Publication date
AU2016269447A1 (en) 2017-01-05
CN103167883B (zh) 2016-08-03
KR20130106811A (ko) 2013-09-30
CN103167883A (zh) 2013-06-19
US10526604B2 (en) 2020-01-07
US20230174987A1 (en) 2023-06-08
US20180127758A1 (en) 2018-05-10
ES2756326T3 (es) 2020-04-27
EP3031920B1 (en) 2019-08-21
IL257936A (en) 2018-05-31
US20160068845A1 (en) 2016-03-10
WO2012012467A3 (en) 2012-04-26
AU2016201288A1 (en) 2016-03-31
EP3489360A2 (en) 2019-05-29
EP3633038A3 (en) 2020-07-29
AU2011282243A1 (en) 2013-02-21
JP2020183379A (ja) 2020-11-12
AU2011282217B2 (en) 2015-12-03
WO2012012443A3 (en) 2013-05-23
JP2013538560A (ja) 2013-10-17
DK3031920T3 (da) 2019-10-14
WO2012012467A2 (en) 2012-01-26
JP5981428B2 (ja) 2016-08-31
HRP20191753T1 (hr) 2019-12-27
EP2595664B1 (en) 2018-10-17
AU2011282217A1 (en) 2013-02-21
US20200362353A1 (en) 2020-11-19
EP2595663A2 (en) 2013-05-29
AU2016269447B2 (en) 2018-12-13
US20180163209A1 (en) 2018-06-14
JP2013538561A (ja) 2013-10-17
AU2020202315A1 (en) 2020-04-23
US9765338B2 (en) 2017-09-19
AU2016269447C1 (en) 2019-06-20
AU2018200825A1 (en) 2018-02-22
EP3031920A1 (en) 2016-06-15
EP3633038A2 (en) 2020-04-08
JP6059141B2 (ja) 2017-01-11
JP2017014235A (ja) 2017-01-19
JP6343308B2 (ja) 2018-06-13
IL223962B (en) 2018-03-29
AU2011282243B2 (en) 2016-09-22
US12480126B2 (en) 2025-11-25
JP2018150344A (ja) 2018-09-27
EP2595664A2 (en) 2013-05-29
KR101900770B1 (ko) 2018-09-20
PL3031920T3 (pl) 2020-01-31
EP2595664A4 (en) 2014-09-17
JP6698740B2 (ja) 2020-05-27
KR20180105730A (ko) 2018-09-28
CA2805791A1 (en) 2012-01-26
AU2011282243A2 (en) 2013-10-10
US20130237585A1 (en) 2013-09-12
US20130225659A1 (en) 2013-08-29
CA2805765A1 (en) 2012-01-26
WO2012012443A2 (en) 2012-01-26
AU2011282217A9 (en) 2015-12-03
EP3489360A3 (en) 2019-08-28
EP2595663A4 (en) 2014-03-05

Similar Documents

Publication Publication Date Title
US12480126B2 (en) Modulation of dystrophia myotonica-protein kinase (DMPK) expression
US11981897B2 (en) Compounds and methods for modulation of dystrophia myotonica-protein kinase (DMPK) expression
JP6974386B2 (ja) Ｃ９ｏｒｆ７２発現を調節するための組成物
JP6679476B2 (ja) Ｃ９ｏｒｆ７２発現を調節するための組成物

Legal Events

Date Code Title Description
C06 Publication
PB01 Publication
C10 Entry into substantive examination
SE01 Entry into force of request for substantive examination
WD01 Invention patent application deemed withdrawn after publication
WD01 Invention patent application deemed withdrawn after publication

Application publication date: 20170222