JP2016521556A - Ｆｏｘｐ３発現を調節するための組成物及び方法 - Google Patents

Ｆｏｘｐ３発現を調節するための組成物及び方法 Download PDF

Info

Publication number
JP2016521556A
JP2016521556A JP2016518041A JP2016518041A JP2016521556A JP 2016521556 A JP2016521556 A JP 2016521556A JP 2016518041 A JP2016518041 A JP 2016518041A JP 2016518041 A JP2016518041 A JP 2016518041A JP 2016521556 A JP2016521556 A JP 2016521556A
Authority
JP
Japan
Prior art keywords
oligonucleotide
stranded oligonucleotide
nucleotide
nucleotides
single stranded
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
JP2016518041A
Other languages
English (en)
Japanese (ja)
Other versions
JP2016521556A5 (https=
Inventor
ジェイムズ バーサム，
ジェイムズ バーサム，
マリアノ セヴェルニーニ，
マリアノ セヴェルニーニ，
ジェイムズ マクスウィゲン，
ジェイムズ マクスウィゲン，
Original Assignee
ラナ セラピューティクス インコーポレイテッド
ラナ セラピューティクス インコーポレイテッド
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by ラナ セラピューティクス インコーポレイテッド, ラナ セラピューティクス インコーポレイテッド filed Critical ラナ セラピューティクス インコーポレイテッド
Publication of JP2016521556A publication Critical patent/JP2016521556A/ja
Publication of JP2016521556A5 publication Critical patent/JP2016521556A5/ja
Pending legal-status Critical Current

Links

Classifications

    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P1/00—Drugs for disorders of the alimentary tract or the digestive system
    • A61P1/02—Stomatological preparations, e.g. drugs for caries, aphtae, periodontitis
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P1/00—Drugs for disorders of the alimentary tract or the digestive system
    • A61P1/04—Drugs for disorders of the alimentary tract or the digestive system for ulcers, gastritis or reflux esophagitis, e.g. antacids, inhibitors of acid secretion, mucosal protectants
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P1/00—Drugs for disorders of the alimentary tract or the digestive system
    • A61P1/16—Drugs for disorders of the alimentary tract or the digestive system for liver or gallbladder disorders, e.g. hepatoprotective agents, cholagogues, litholytics
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P1/00—Drugs for disorders of the alimentary tract or the digestive system
    • A61P1/18—Drugs for disorders of the alimentary tract or the digestive system for pancreatic disorders, e.g. pancreatic enzymes
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P11/00—Drugs for disorders of the respiratory system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P11/00—Drugs for disorders of the respiratory system
    • A61P11/04—Drugs for disorders of the respiratory system for throat disorders
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P13/00—Drugs for disorders of the urinary system
    • A61P13/02—Drugs for disorders of the urinary system of urine or of the urinary tract, e.g. urine acidifiers
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P13/00—Drugs for disorders of the urinary system
    • A61P13/10—Drugs for disorders of the urinary system of the bladder
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P13/00—Drugs for disorders of the urinary system
    • A61P13/12—Drugs for disorders of the urinary system of the kidneys
    • 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
    • A61P17/00—Drugs for dermatological disorders
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P17/00—Drugs for dermatological disorders
    • A61P17/04—Antipruritics
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P17/00—Drugs for dermatological disorders
    • A61P17/06—Antipsoriatics
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P17/00—Drugs for dermatological disorders
    • A61P17/08—Antiseborrheics
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P17/00—Drugs for dermatological disorders
    • A61P17/14—Drugs for dermatological disorders for baldness or alopecia
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P19/00—Drugs for skeletal disorders
    • A61P19/02—Drugs for skeletal disorders for joint disorders, e.g. arthritis, arthrosis
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P19/00—Drugs for skeletal disorders
    • A61P19/08—Drugs for skeletal disorders for bone diseases, e.g. rachitism, Paget's disease
    • 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
    • A61P25/00—Drugs for disorders of the nervous system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/02—Drugs for disorders of the nervous system for peripheral neuropathies
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P27/00—Drugs for disorders of the senses
    • A61P27/02—Ophthalmic agents
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P27/00—Drugs for disorders of the senses
    • A61P27/16—Otologicals
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P29/00—Non-central analgesic, antipyretic or antiinflammatory agents, e.g. antirheumatic agents; Non-steroidal antiinflammatory drugs [NSAID]
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00—Drugs for disorders of the metabolism
    • A61P3/06—Antihyperlipidemics
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00—Drugs for disorders of the metabolism
    • A61P3/08—Drugs for disorders of the metabolism for glucose homeostasis
    • A61P3/10—Drugs for disorders of the metabolism for glucose homeostasis for hyperglycaemia, e.g. antidiabetics
    • 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
    • A61P37/00—Drugs for immunological or allergic disorders
    • A61P37/02—Immunomodulators
    • A61P37/04—Immunostimulants
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P37/00—Drugs for immunological or allergic disorders
    • A61P37/02—Immunomodulators
    • A61P37/06—Immunosuppressants, e.g. drugs for graft rejection
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P5/00—Drugs for disorders of the endocrine system
    • A61P5/14—Drugs for disorders of the endocrine system of the thyroid hormones, e.g. T3, T4
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P7/00—Drugs for disorders of the blood or the extracellular fluid
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P7/00—Drugs for disorders of the blood or the extracellular fluid
    • A61P7/06—Antianaemics
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P7/00—Drugs for disorders of the blood or the extracellular fluid
    • A61P7/10—Antioedematous agents; Diuretics
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00—Drugs for disorders of the cardiovascular system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00—Drugs for disorders of the cardiovascular system
    • A61P9/10—Drugs for disorders of the cardiovascular system for treating ischaemic or atherosclerotic diseases, e.g. antianginal drugs, coronary vasodilators, drugs for myocardial infarction, retinopathy, cerebrovascula insufficiency, renal arteriosclerosis
    • 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
    • C12N2310/32—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
    • C12N2310/323—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
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/343—Spatial arrangement of the modifications having patterns, e.g. ==--==--==--
    • 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
    • C12N2310/351—Conjugate

Landscapes

  • Health & Medical Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Chemical & Material Sciences (AREA)
  • General Health & Medical Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • General Chemical & Material Sciences (AREA)
  • Animal Behavior & Ethology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Genetics & Genomics (AREA)
  • Biomedical Technology (AREA)
  • Diabetes (AREA)
  • Immunology (AREA)
  • Hematology (AREA)
  • Dermatology (AREA)
  • Zoology (AREA)
  • Biotechnology (AREA)
  • Wood Science & Technology (AREA)
  • Molecular Biology (AREA)
  • General Engineering & Computer Science (AREA)
  • Urology & Nephrology (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Rheumatology (AREA)
  • Pulmonology (AREA)
  • Neurology (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Endocrinology (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Heart & Thoracic Surgery (AREA)
  • Neurosurgery (AREA)
JP2016518041A 2013-06-07 2014-06-06 Ｆｏｘｐ３発現を調節するための組成物及び方法 Pending JP2016521556A (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201361832677P 2013-06-07 2013-06-07
US61/832,677 2013-06-07
PCT/US2014/041345 WO2014197826A1 (en) 2013-06-07 2014-06-06 Compositions and methods for modulating foxp3 expression

Publications (2)

Publication Number Publication Date
JP2016521556A true JP2016521556A (ja) 2016-07-25
JP2016521556A5 JP2016521556A5 (https=) 2017-07-20

Family

ID=52008624

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2016518041A Pending JP2016521556A (ja) 2013-06-07 2014-06-06 Ｆｏｘｐ３発現を調節するための組成物及び方法

Country Status (7)

Country Link
US (1) US20160122760A1 (https=)
EP (1) EP3004354A4 (https=)
JP (1) JP2016521556A (https=)
KR (1) KR20160027968A (https=)
AU (1) AU2014274730A1 (https=)
CA (1) CA2914536A1 (https=)
WO (1) WO2014197826A1 (https=)

Cited By (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2017509631A (ja) * 2014-03-13 2017-04-06 エフ・ホフマン−ラ・ロシュ・アクチェンゲゼルシャフト エストロゲン受容体突然変異体を調節する方法及び組成物
JP2022502062A (ja) * 2018-09-26 2022-01-11 オーム ライフテック インコーポレイテッド ２’ｆａｎａ修飾ｆｏｘｐ３アンチセンスオリゴヌクレオチドおよびその使用方法
JP2022507425A (ja) * 2018-11-14 2022-01-18 アイオニス・ファーマシューティカルズ・インコーポレイテッド Ｆｏｘｐ３発現のモジュレータ
JP2022518929A (ja) * 2019-01-29 2022-03-17 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 Ａｐｐの発現を低減するための化合物及び方法
US11584932B2 (en) 2016-11-01 2023-02-21 The Research Foundation For The State University Of New York 5-halouracil-modified microRNAs and their use in the treatment of cancer
US12319913B2 (en) 2018-10-31 2025-06-03 The Research Foundation For The State University Of New York 5-halouracil-modified microRNAs and their use in the treatment of cancer

Families Citing this family (19)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EA201492122A1 (ru) 2012-05-16 2015-10-30 Рана Терапьютикс, Инк. Композиции и способы для модулирования экспрессии utrn
AU2013262699A1 (en) 2012-05-16 2015-01-22 Rana Therapeutics, Inc. Compositions and methods for modulating ATP2A2 expression
BR112014028631A2 (pt) 2012-05-16 2017-10-17 Rana Therapeutics Inc composições e métodos para modulação da expressão da família de genes da hemoglobina
SG11201407483YA (en) 2012-05-16 2014-12-30 Rana Therapeutics Inc Compositions and methods for modulating smn gene family expression
US10837014B2 (en) 2012-05-16 2020-11-17 Translate Bio Ma, Inc. Compositions and methods for modulating SMN gene family expression
EA201492116A1 (ru) 2012-05-16 2015-05-29 Рана Терапьютикс, Инк. Композиции и способы для модулирования экспрессии mecp2
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
WO2017181026A1 (en) * 2016-04-15 2017-10-19 Translate Bio Ma, Inc. Selective modulation of foxp3 expression
WO2017184082A1 (en) * 2016-04-22 2017-10-26 Nanyang Technological University A lymphocyte permeating chimeric oligonucleotide, methods and uses thereof
WO2018031871A1 (en) * 2016-08-12 2018-02-15 Translate Bio Ma, Inc. Ex vivo modulation of foxp3 expression
CA3043768A1 (en) 2016-11-29 2018-06-07 PureTech Health LLC Exosomes for delivery of therapeutic agents
US12024715B2 (en) * 2017-11-07 2024-07-02 Temple University-Of The Commonwealth System Of Higher Education Compositions and methods for improved T cells
CN111378622B (zh) * 2018-12-29 2022-12-02 华东师范大学 核酸编码的car-t细胞及其制备方法和应用
CA3136172A1 (en) * 2019-04-18 2020-10-22 Proqr Therapeutics Ii B.V. Antisense oligonucleotides for the treatment of usher syndrome
US11866708B2 (en) * 2019-10-22 2024-01-09 Board Of Regents, The University Of Texas System Tailored modulation of gene regulation programs via functional enhancer RNA
CA3189922A1 (en) 2020-07-28 2022-02-03 Ionis Pharmaceuticals, Inc. Compounds and methods for reducing app expression
WO2022088342A1 (zh) * 2020-10-28 2022-05-05 苏州吉玛基因股份有限公司 一种靶向FOXP3基因的siRNA及其修饰方法
US20250188476A1 (en) * 2021-06-18 2025-06-12 Ionis Pharmaceuticals, Inc. Compounds and methods for reducing ifnar1 expression
WO2023235838A2 (en) * 2022-06-02 2023-12-07 Skyhawk Therapeutics, Inc. Rna-targeting splicing modifiers for treatment of foxp3-associated conditions and diseases

Citations (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2008103763A2 (en) * 2007-02-20 2008-08-28 Sequenom, Inc. Methods and compositions for cancer diagnosis and treatment based on nucleic acid methylation
US20120004278A1 (en) * 2010-06-18 2012-01-05 The Board Of Trustees Of The Leland Stanford Junior University Linc rnas in cancer diagnosis and treatment
WO2012020839A1 (ja) * 2010-08-12 2012-02-16 塩野義製薬株式会社 癌治療用医薬組成物
WO2012075114A2 (en) * 2010-12-01 2012-06-07 Ablitech, Inc. Nucleic acid-polymer conjugates and uses thereof

Family Cites Families (13)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US14008A (en) * 1856-01-01 Improvement in furnaces for soldering
EP1967525B1 (en) * 2001-05-08 2012-11-14 Darwin Molecular Corporation A method for regulating immune function in primates using the foxp3 protein
US8029985B2 (en) * 2004-09-01 2011-10-04 Vybion, Inc. Amplified bioassay
US8629108B2 (en) * 2006-06-27 2014-01-14 Opexa Therapeutics, Inc. Rheumatoid arthritis T cell vaccine
EP2064350B1 (en) * 2006-11-27 2013-01-02 Ludwig Institute for Cancer Research Ltd. Expression of foxp3 by cancer cells
US8158596B2 (en) * 2007-05-11 2012-04-17 The Regents Of The University Of Michigan Materials and methods for FOXP3 tumor suppression
EP2591797A4 (en) * 2010-07-09 2014-01-15 Inst Pasteur Of Shanghai Cas REGULATORY FACTOR OF FOXP3 AND REGULATORY T CELLS AND USE THEREOF
BR112013005806B1 (pt) * 2010-09-10 2022-05-10 Epizyme, Inc Métodos para detectar se um indivíduo é um candidato para o tratamento com ou responsivo a um inibidor de ezh2 e usos terapêuticos do dito inibidor de ezh2
US9920317B2 (en) * 2010-11-12 2018-03-20 The General Hospital Corporation Polycomb-associated non-coding RNAs
EP2850183A4 (en) * 2012-05-16 2016-02-10 Rana Therapeutics Inc COMPOSITIONS AND METHOD FOR MODULATING GENE EXPRESSION
WO2013173652A1 (en) * 2012-05-16 2013-11-21 Rana Therapeutics, Inc. Compositions and methods for modulating gene expression
EA201492117A1 (ru) * 2012-05-16 2015-04-30 Рана Терапьютикс, Инк. Композиции и способы для модулирования экспрессии bdnf
US9567581B2 (en) * 2012-08-07 2017-02-14 The General Hospital Corporation Selective reactivation of genes on the inactive X chromosome

Patent Citations (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2008103763A2 (en) * 2007-02-20 2008-08-28 Sequenom, Inc. Methods and compositions for cancer diagnosis and treatment based on nucleic acid methylation
US20120004278A1 (en) * 2010-06-18 2012-01-05 The Board Of Trustees Of The Leland Stanford Junior University Linc rnas in cancer diagnosis and treatment
WO2012020839A1 (ja) * 2010-08-12 2012-02-16 塩野義製薬株式会社 癌治療用医薬組成物
WO2012075114A2 (en) * 2010-12-01 2012-06-07 Ablitech, Inc. Nucleic acid-polymer conjugates and uses thereof

Non-Patent Citations (4)

* Cited by examiner, † Cited by third party
Title
J. BIOL. CHEM., 2012, 287(41), PP.34372-34385, JPN6018018454, ISSN: 0003952236 *
PHARMACOGENOMICS, 2007, 8(11), PP.1485-1487, JPN6018018453, ISSN: 0003952235 *
SARMA K. ET AL., PROC. NATL. ACAD. USA, vol. 107(51)(2010), JPN6017005742, pages 22196 - 22201, ISSN: 0003952237 *
神武洋二郎 他: "長鎖ｎｃＲＮＡによるクロマチン修飾因子のリクルートメント機構", 実験医学, vol. Vol.29 No.11(2011), JPN7017000587, pages 1716 - 1721, ISSN: 0003952238 *

Cited By (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2017509631A (ja) * 2014-03-13 2017-04-06 エフ・ホフマン−ラ・ロシュ・アクチェンゲゼルシャフト エストロゲン受容体突然変異体を調節する方法及び組成物
US11584932B2 (en) 2016-11-01 2023-02-21 The Research Foundation For The State University Of New York 5-halouracil-modified microRNAs and their use in the treatment of cancer
US12371693B2 (en) 2016-11-01 2025-07-29 The Research Foundation For The State University Of New York 5-halouracil-modified micrornas and their use in the treatment of cancer
JP2022502062A (ja) * 2018-09-26 2022-01-11 オーム ライフテック インコーポレイテッド ２’ｆａｎａ修飾ｆｏｘｐ３アンチセンスオリゴヌクレオチドおよびその使用方法
US12319913B2 (en) 2018-10-31 2025-06-03 The Research Foundation For The State University Of New York 5-halouracil-modified microRNAs and their use in the treatment of cancer
JP2022507425A (ja) * 2018-11-14 2022-01-18 アイオニス・ファーマシューティカルズ・インコーポレイテッド Ｆｏｘｐ３発現のモジュレータ
JP2022518929A (ja) * 2019-01-29 2022-03-17 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 Ａｐｐの発現を低減するための化合物及び方法
JP7557469B2 (ja) 2019-01-29 2024-09-27 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 Ａｐｐの発現を低減するための化合物及び方法

Also Published As

Publication number Publication date
EP3004354A4 (en) 2017-01-11
KR20160027968A (ko) 2016-03-10
CA2914536A1 (en) 2014-12-11
US20160122760A1 (en) 2016-05-05
WO2014197826A1 (en) 2014-12-11
AU2014274730A1 (en) 2016-01-21
EP3004354A1 (en) 2016-04-13

Similar Documents

Publication Publication Date Title
JP2016521556A (ja) Ｆｏｘｐ３発現を調節するための組成物及び方法
EP2850190B1 (en) Compositions and methods for modulating mecp2 expression
EP2850186B1 (en) Compositions and methods for modulating smn gene family expression
US10041074B2 (en) Euchromatic region targeting methods for modulating gene expression
JP2015523853A (ja) Ａｔｐ２ａ２発現を調節するための組成物及び方法
JP2015518710A (ja) ヘモグロビン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
JP2015518713A (ja) Ｕｔｒｎ発現を調節するための組成物及び方法
US20150232847A1 (en) Targeting oligonucleotides and related methods for modulating fxn rna
JP2015519057A (ja) Ｐｔｅｎ発現を調節するための組成物及び方法
JP2015518711A (ja) Ｂｄｎｆ発現を調節するための組成物及び方法
JP2015523855A (ja) Ａｐｏａ１及びａｂｃａ１発現を調節するための組成物及び方法
US20150225722A1 (en) Methods for selective targeting of heterochromatin forming non-coding rna
JP2015518714A (ja) 遺伝子発現を調節するための組成物及び方法
JP2016534035A (ja) 筋萎縮性側索硬化症を治療するための組成物及び方法
EP3033425A1 (en) Compositions and methods for modulating expression of frataxin
WO2015023938A1 (en) Epigenetic regulators of frataxin
US20150232836A1 (en) Compositions and methods for modulating gene expression
US20180030452A1 (en) Targeting oligonucleotides and uses thereof to modulate gene expression
AU2022203361A1 (en) Compositions and methods for modulating RNA

Legal Events

Date Code Title Description
A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20170605

A621 Written request for application examination

Free format text: JAPANESE INTERMEDIATE CODE: A621

Effective date: 20170605

A131 Notification of reasons for refusal

Free format text: JAPANESE INTERMEDIATE CODE: A131

Effective date: 20180522

A601 Written request for extension of time

Free format text: JAPANESE INTERMEDIATE CODE: A601

Effective date: 20180821

A02 Decision of refusal

Free format text: JAPANESE INTERMEDIATE CODE: A02

Effective date: 20190107