CN104583398A - 用于调节基因表达的组合物和方法 - Google Patents

用于调节基因表达的组合物和方法 Download PDF

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Publication number
CN104583398A
CN104583398A CN201380037158.XA CN201380037158A CN104583398A CN 104583398 A CN104583398 A CN 104583398A CN 201380037158 A CN201380037158 A CN 201380037158A CN 104583398 A CN104583398 A CN 104583398A
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Prior art keywords
nucleotide
oligonucleotide
single stranded
sequence
prc2
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Pending
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CN201380037158.XA
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English (en)
Chinese (zh)
Inventor
阿瑟·M·克里格
罗梅什·苏布拉马尼安
詹姆斯·麦克斯维根
珍妮·T·李
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General Hospital Corp
Translate Bio Inc
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General Hospital Corp
RaNA Therapeutics Inc
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First worldwide family litigation filed litigation Critical https://patents.darts-ip.com/?family=49584310&utm_source=google_patent&utm_medium=platform_link&utm_campaign=public_patent_search&patent=CN104583398(A) "Global patent litigation dataset” by Darts-ip is licensed under a Creative Commons Attribution 4.0 International License.
Application filed by General Hospital Corp, RaNA Therapeutics Inc filed Critical General Hospital Corp
Publication of CN104583398A publication Critical patent/CN104583398A/zh
Pending legal-status Critical Current

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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
    • A61P7/00—Drugs for disorders of the blood or the extracellular fluid
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/11—Antisense
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    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/315—Phosphorothioates
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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
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/322—2'-R Modification
    • C—CHEMISTRY; METALLURGY
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/323—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
    • C12N2310/343—Spatial arrangement of the modifications having patterns, e.g. ==--==--==--
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
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    • C12N2310/3517—Marker; Tag
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    • 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
    • C12N2310/3521—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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  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
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  • General Health & Medical Sciences (AREA)
  • Plant Pathology (AREA)
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  • Physics & Mathematics (AREA)
  • Medicinal Chemistry (AREA)
  • 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)
CN201380037158.XA 2012-05-16 2013-05-16 用于调节基因表达的组合物和方法 Pending CN104583398A (zh)

Applications Claiming Priority (35)

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

Publications (1)

Publication Number Publication Date
CN104583398A true CN104583398A (zh) 2015-04-29

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ID=49584310

Family Applications (1)

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CN201380037158.XA Pending CN104583398A (zh) 2012-05-16 2013-05-16 用于调节基因表达的组合物和方法

Country Status (10)

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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)

Cited By (6)

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CN106636120A (zh) * 2017-02-17 2017-05-10 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
WO2020135677A1 (zh) * 2018-12-29 2020-07-02 中美瑞康核酸技术(南通)研究院有限公司 寡聚核酸分子及其应用
CN113444726A (zh) * 2021-06-28 2021-09-28 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
CN119688749A (zh) * 2024-12-19 2025-03-25 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
US12458604B2 (en) 2020-10-14 2025-11-04 The Trustees Of The University Of Pennsylvania Methods of lipid nanoparticle manufacture and compositions derived therefrom
US12605400B2 (en) 2019-04-30 2026-04-21 Ractigen Therapeutics Oligomeric nucleic acid molecule, and application thereof in an acute intermittent porphyria treatment

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EA201492116A1 (ru) * 2012-05-16 2015-05-29 Рана Терапьютикс, Инк. Композиции и способы для модулирования экспрессии mecp2
US10837014B2 (en) 2012-05-16 2020-11-17 Translate Bio Ma, Inc. Compositions and methods for modulating SMN gene family expression
BR112014028645A2 (pt) * 2012-05-16 2017-06-27 Rana Therapeutics Inc composições e métodos para modulação da expressão de apoa1 e abca1.
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BR112014028631A2 (pt) 2012-05-16 2017-10-17 Rana Therapeutics Inc composições e métodos para modulação da expressão da família de genes da hemoglobina
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JP2016521556A (ja) * 2013-06-07 2016-07-25 ラナ セラピューティクス インコーポレイテッド Ｆｏｘｐ３発現を調節するための組成物及び方法
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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US20200208152A1 (en) 2017-09-08 2020-07-02 Mina Therapeutics Limited Stabilized sarna compositions and methods of use
WO2019048632A1 (en) 2017-09-08 2019-03-14 Mina Therapeutics Limited STABILIZED COMPOSITIONS OF SMALL ACTIVATORY RNA (PARNA) OF HNF4A 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
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
CN113811311A (zh) * 2019-03-15 2021-12-17 马萨诸塞大学 用于组织特异性apoe调节的寡核苷酸
US20220281911A1 (en) 2019-08-19 2022-09-08 Mina Therapeutics Limited Oligonucleotide conjugate compositions and methods of use
WO2021076551A1 (en) * 2019-10-14 2021-04-22 University Of Cincinnati Novel rna aptamer intervenes estrogen receptor interaction with coactivator med1 to overcome breast cancer metastasis
WO2021262919A2 (en) 2020-06-26 2021-12-30 The Research Foundation For The State University Of New York 5-halouracil-modified micrornas and their use in the treatment of cancer
KR20240126870A (ko) 2021-12-22 2024-08-21 캠프4 테라퓨틱스 코포레이션 조절 rna들을 표적으로 하는 안티센스 올리고뉴클레오티드를 사용한 유전자 전사의 조절
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

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