JP2015518714A - 遺伝子発現を調節するための組成物及び方法 - Google Patents

遺伝子発現を調節するための組成物及び方法 Download PDF

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JP2015518714A
JP2015518714A JP2015512864A JP2015512864A JP2015518714A JP 2015518714 A JP2015518714 A JP 2015518714A JP 2015512864 A JP2015512864 A JP 2015512864A JP 2015512864 A JP2015512864 A JP 2015512864A JP 2015518714 A JP2015518714 A JP 2015518714A
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oligonucleotide
nucleotides
nucleotide
sequence
stranded oligonucleotide
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JP2015518714A5 (2
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アーサー エム． クリーグ，
アーサー エム． クリーグ，
ロメシュ スブラマニアン，
ロメシュ スブラマニアン，
ジェイムズ マクスウィゲン，
ジェイムズ マクスウィゲン，
ジェニー ティー． リー，
ジェニー ティー． リー，
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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
ザ ジェネラル ホスピタル コーポレイション
ザ ジェネラル ホスピタル コーポレイション
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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, ザ ジェネラル ホスピタル コーポレイション, ザ ジェネラル ホスピタル コーポレイション 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 JP2015518714A publication Critical patent/JP2015518714A/ja
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    • C—CHEMISTRY; METALLURGY
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    • 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
    • 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
    • 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
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/11—Antisense
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    • C12N2310/00—Structure or type of the nucleic acid
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    • 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
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/32—Chemical structure of the sugar
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    • C12N2310/00—Structure or type of the nucleic acid
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    • 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
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/34—Spatial arrangement of the modifications
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    • C12N2310/3517—Marker; Tag
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    • C12N2310/35—Nature of the modification
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    • C12N2310/3521—Methyl
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/353—Nature of the modification linked to the nucleic acid via an atom other than carbon
    • C12N2310/3533—Halogen

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  • Engineering & Computer Science (AREA)
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  • Bioinformatics & Cheminformatics (AREA)
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  • General Health & Medical Sciences (AREA)
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  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Diabetes (AREA)
  • Hematology (AREA)
  • Epidemiology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
  • Medicinal Preparation (AREA)
JP2015512864A 2012-05-16 2013-05-16 遺伝子発現を調節するための組成物及び方法 Pending JP2015518714A (ja)

Applications Claiming Priority (35)

Application Number Priority Date Filing Date Title
US201261648058P 2012-05-16 2012-05-16
US201261648051P 2012-05-16 2012-05-16
US201261647858P 2012-05-16 2012-05-16
US201261648021P 2012-05-16 2012-05-16
US201261647886P 2012-05-16 2012-05-16
US201261647949P 2012-05-16 2012-05-16
US201261647925P 2012-05-16 2012-05-16
US201261647901P 2012-05-16 2012-05-16
US201261648041P 2012-05-16 2012-05-16
US201261648016P 2012-05-16 2012-05-16
US61/647,949 2012-05-16
US61/647,901 2012-05-16
US61/648,058 2012-05-16
US61/647,925 2012-05-16
US61/648,021 2012-05-16
US61/647,886 2012-05-16
US61/648,016 2012-05-16
US61/648,051 2012-05-16
US61/647,858 2012-05-16
US61/648,041 2012-05-16
US201261719394P 2012-10-27 2012-10-27
US61/719,394 2012-10-27
US201361785529P 2013-03-14 2013-03-14
US201361785778P 2013-03-14 2013-03-14
US201361785956P 2013-03-14 2013-03-14
US201361785832P 2013-03-14 2013-03-14
US201361785885P 2013-03-14 2013-03-14
US201361786232P 2013-03-14 2013-03-14
US61/785,885 2013-03-14
US61/785,832 2013-03-14
US61/785,956 2013-03-14
US61/785,529 2013-03-14
US61/786,232 2013-03-14
US61/785,778 2013-03-14
PCT/US2013/041461 WO2013173652A1 (en) 2012-05-16 2013-05-16 Compositions and methods for modulating gene expression

Publications (2)

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JP2015518714A true JP2015518714A (ja) 2015-07-06
JP2015518714A5 JP2015518714A5 (2) 2016-06-30

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US (2) US20150133362A1 (2)
EP (2) EP3511416A1 (2)
JP (1) JP2015518714A (2)
KR (1) KR102028784B1 (2)
CN (1) CN104583398A (2)
AU (1) AU2013262663A1 (2)
CA (1) CA2873809A1 (2)
DK (1) DK2850189T3 (2)
EA (1) EA201492114A1 (2)
WO (1) WO2013173652A1 (2)

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JP2015523853A (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 Ａｔｐ２ａ２発現を調節するための組成物及び方法
US10059941B2 (en) 2012-05-16 2018-08-28 Translate Bio Ma, Inc. Compositions and methods for modulating SMN gene family expression
US10058623B2 (en) 2012-05-16 2018-08-28 Translate Bio Ma, Inc. Compositions and methods for modulating UTRN expression
US10174315B2 (en) 2012-05-16 2019-01-08 The General Hospital Corporation Compositions and methods for modulating hemoglobin gene family expression
US10655128B2 (en) 2012-05-16 2020-05-19 Translate Bio Ma, 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
JP2022500087A (ja) * 2018-09-14 2022-01-04 元 篠崎 エピジェネティックマーカーを使用してせん妄危険性を検出するためのシステムおよび方法
JP2022525208A (ja) * 2019-03-15 2022-05-11 ユニバーシティ・オブ・マサチューセッツ 組織特異的なａｐｏｅ調節のためのオリゴヌクレオチド
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
US12458604B2 (en) 2020-10-14 2025-11-04 The Trustees Of The University Of Pennsylvania Methods of lipid nanoparticle manufacture and compositions derived therefrom

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EA201492119A1 (ru) * 2012-05-16 2015-05-29 Рана Терапьютикс, Инк. Композиции и способы для модулирования экспрессии apoa1 и abca1
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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
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CN106636120B (zh) * 2017-02-17 2019-08-30 中国人民解放军第三军医大学第三附属医院 可编码ArtRD蛋白基因及其蛋白和应用
WO2019048645A1 (en) 2017-09-08 2019-03-14 Mina Therapeutics Limited STABILIZED COMPOSITIONS OF SMALL ACTIVATOR RNA (PARNA) FROM CEBPA AND METHODS OF USE
CA3075205A1 (en) 2017-09-08 2019-03-14 Mina Therapeutics Limited Stabilized hnf4a sarna compositions and methods of use
WO2020028533A1 (en) * 2018-08-01 2020-02-06 Yale University Compositions and methods for identification of membrane targets for enhancement of t cell activity against cancer
AU2019414608A1 (en) * 2018-12-29 2021-07-22 Ractigen Therapeutics Oligomeric nucleic acid molecule and application thereof
CN111849968A (zh) 2019-04-30 2020-10-30 中美瑞康核酸技术(南通)研究院有限公司 寡核苷酸分子及其在急性间歇性卟啉症治疗中的应用
CN114585633A (zh) 2019-08-19 2022-06-03 米纳治疗有限公司 寡核苷酸缀合物组合物和使用方法
US20240102023A1 (en) * 2019-10-14 2024-03-28 University Of Cincinnati Novel rna aptamer intervenes estrogen receptor interaction with coactivator med1 to overcome breast cancer metastasis
CN113444726B (zh) * 2021-06-28 2022-07-29 甘肃农业大学 一种与仔猪细菌性腹泻相关的lncRNA ALDB-898及其应用
MX2024007790A (es) 2021-12-22 2024-09-06 Camp4 Therapeutics Corp Modulación de la transcripción génica utilizando oligonucleótidos antisentido dirigidos a arn reguladores.
CN114540498A (zh) * 2022-03-09 2022-05-27 厦门飞朔生物技术有限公司 一种基于dna甲基化的浸润性神经胶质瘤的分类装置
KR102923692B1 (ko) * 2023-03-31 2026-02-09 주식회사 어니스트 농업회사법인 적색 또는 황색 구피색 양파 판별용 프라이머 세트 및 이의 용도
CN119688749B (zh) * 2024-12-19 2025-08-26 中山大学孙逸仙纪念医院 一种促进rna晶体生长并提高衍射分辨率的方法及其应用

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AU2013262663A1 (en) 2015-01-22
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