JP7489161B2 - 肝臓特異的調節性核酸配列 - Google Patents

肝臓特異的調節性核酸配列 Download PDF

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JP7489161B2
JP7489161B2 JP2021527948A JP2021527948A JP7489161B2 JP 7489161 B2 JP7489161 B2 JP 7489161B2 JP 2021527948 A JP2021527948 A JP 2021527948A JP 2021527948 A JP2021527948 A JP 2021527948A JP 7489161 B2 JP7489161 B2 JP 7489161B2
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マイケル・ロバーツ
フアン・マヌエル・イグレシアス
ホルヘ・オマール・ジャニェス－クニャ
ニコル・キッペン
ロス・フレイザー
ケイティ・ベイカー
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アスクバイオ・ユーケー・リミテッド
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    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • C07K14/4701—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals not used
    • C07K14/4702—Regulators; Modulating activity
    • C07K14/4705—Regulators; Modulating activity stimulating, promoting or activating activity
    • 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
    • A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • A61K48/0058—Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
    • 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
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86—Viral vectors
    • 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
    • C12N5/00—Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
    • C12N5/06—Animal cells or tissues; Human cells or tissues
    • C12N5/0602—Vertebrate cells
    • C12N5/067—Hepatocytes
    • 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
    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/008—Vector systems having a special element relevant for transcription cell type or tissue specific enhancer/promoter combination

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
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  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
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  • General Engineering & Computer Science (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
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  • Plant Pathology (AREA)
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  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Cell Biology (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Preparation Of Compounds By Using Micro-Organisms (AREA)
JP2021527948A 2018-11-19 2019-11-19 肝臓特異的調節性核酸配列 Active JP7489161B2 (ja)

Priority Applications (1)

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JP2024075266A JP2024096349A (ja) 2018-11-19 2024-05-07 肝臓特異的調節性核酸配列

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GB1818816.9 2018-11-19
GBGB1818816.9A GB201818816D0 (en) 2018-11-19 2018-11-19 Regulatory nucleic acid sequences
PCT/GB2019/053267 WO2020104782A1 (en) 2018-11-19 2019-11-19 Liver-Specific Regulatory Nucleic Acid Sequences

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JP2022507828A JP2022507828A (ja) 2022-01-18
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JP7489161B2 true JP7489161B2 (ja) 2024-05-23

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US (2) US12590129B2 (https=)
EP (2) EP3883951A1 (https=)
JP (2) JP7489161B2 (https=)
CN (1) CN113454104A (https=)
AU (2) AU2019382955B2 (https=)
CA (1) CA3120253A1 (https=)
GB (1) GB201818816D0 (https=)
IL (1) IL283219A (https=)
WO (2) WO2020104782A1 (https=)

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Publication number Priority date Publication date Assignee Title
CN117202925A (zh) 2021-01-20 2023-12-08 科罗根科技有限公司 用于治疗和诊断肝癌的Ssu72蛋白或多核苷酸
CN116744961A (zh) 2021-01-29 2023-09-12 科罗根科技有限公司 使用Ssu72治疗肝病的药物组合物
WO2023008925A1 (ko) * 2021-07-29 2023-02-02 주식회사 리보퓨틱스 융합 프로모터, 이를 포함하는 재조합 벡터 및 이를 이용하는 간암 치료용 조성물
CN121358865A (zh) * 2023-04-20 2026-01-16 阿斯克生物公司 肝脏特异性调控核酸序列
CN116555266B (zh) * 2023-06-09 2024-11-12 呈诺再生医学科技(北京)有限公司 肝脏特异性表达的重组启动子和载体及其在肝脏相关疾病治疗中的应用

Citations (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2001500376A (ja) 1996-09-06 2001-01-16 カイロン コーポレイション 組換えａａｖベクターを使用する治療分子の肝臓特異的送達のための方法および組成物
JP2004500880A (ja) 2000-06-20 2004-01-15 ザ、ボード、オブ、トラスティーズ、オブ、ザ、リーランド、スタンフォード、ジュニア、ユニバーシティ 肝臓特異的な遺伝子発現カセット、及びその使用法
JP2011517955A (ja) 2008-04-22 2011-06-23 フエー・イー・ベー・フエー・ゼツト・ウエー 肝特異的核酸調節要素ならびにその方法および用途
JP2014506456A (ja) 2011-01-25 2014-03-17 シンプロミクス リミテッド 特異的プロモーターの構築のための方法

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US5721096A (en) * 1991-01-24 1998-02-24 Children's Medical Center Corporation Methods of screening for compounds with ability to alter apolipoprotein AI gene expression
US5578444A (en) 1991-06-27 1996-11-26 Genelabs Technologies, Inc. Sequence-directed DNA-binding molecules compositions and methods
US6268212B1 (en) 1993-10-18 2001-07-31 Amgen Inc. Tissue specific transgene expression
ATE437221T1 (de) 2001-05-14 2009-08-15 Gbp Ip Llc Lentivirale vektoren kodierend für gerinnungsfaktoren für die gentherapie
CN102634515B (zh) * 2008-02-14 2013-11-06 财团法人牧岩生命工学研究所 适于表达用于基因治疗的编码序列的表达载体
CN102628041B (zh) * 2008-02-14 2013-09-11 财团法人牧岩生命工学研究所 适于表达用于基因治疗的编码序列的表达载体
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WO2018144709A2 (en) * 2017-02-01 2018-08-09 The Trustees Of The University Of Pennsylvania Gene therapy for treating citrullenemia
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CN108588097B (zh) * 2018-04-28 2021-07-13 北京锦篮基因科技有限公司 改造后的hbv基因组和相关组合物及其应用

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JP2001500376A (ja) 1996-09-06 2001-01-16 カイロン コーポレイション 組換えａａｖベクターを使用する治療分子の肝臓特異的送達のための方法および組成物
JP2004500880A (ja) 2000-06-20 2004-01-15 ザ、ボード、オブ、トラスティーズ、オブ、ザ、リーランド、スタンフォード、ジュニア、ユニバーシティ 肝臓特異的な遺伝子発現カセット、及びその使用法
JP2011517955A (ja) 2008-04-22 2011-06-23 フエー・イー・ベー・フエー・ゼツト・ウエー 肝特異的核酸調節要素ならびにその方法および用途
JP2014506456A (ja) 2011-01-25 2014-03-17 シンプロミクス リミテッド 特異的プロモーターの構築のための方法

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Publication number Publication date
AU2019382955A1 (en) 2021-06-10
EP3883952A1 (en) 2021-09-29
US20210388347A1 (en) 2021-12-16
WO2020104782A1 (en) 2020-05-28
CA3120253A1 (en) 2020-05-28
CN113454104A (zh) 2021-09-28
EP3883951A1 (en) 2021-09-29
AU2025287323A1 (en) 2026-03-26
AU2019382955B2 (en) 2025-09-25
JP2022507828A (ja) 2022-01-18
US12590129B2 (en) 2026-03-31
WO2020104783A1 (en) 2020-05-28
JP2024096349A (ja) 2024-07-12
US20220009981A1 (en) 2022-01-13
IL283219A (en) 2021-06-30
GB201818816D0 (en) 2019-01-02

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