CN113454104A - 肝特异性调节性核酸序列 - Google Patents

肝特异性调节性核酸序列 Download PDF

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Publication number
CN113454104A
CN113454104A CN201980089303.6A CN201980089303A CN113454104A CN 113454104 A CN113454104 A CN 113454104A CN 201980089303 A CN201980089303 A CN 201980089303A CN 113454104 A CN113454104 A CN 113454104A
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cre0058
crm
cre0065
cre0051
cre0066
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CN201980089303.6A
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English (en)
Chinese (zh)
Inventor
迈克尔·罗伯茨
胡安·曼纽尔·伊格莱西亚斯
豪尔赫·奥马尔·亚涅斯-库纳
尼科尔·基彭
罗斯·弗雷泽
凯蒂·贝克
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AskBio UK Ltd
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Synpromics Ltd
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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)
  • Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Biomedical Technology (AREA)
  • Biochemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Biotechnology (AREA)
  • Molecular Biology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • Biophysics (AREA)
  • Medicinal Chemistry (AREA)
  • Gastroenterology & Hepatology (AREA)
  • General Engineering & Computer Science (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Toxicology (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Virology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Cell Biology (AREA)
  • Epidemiology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (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)
CN201980089303.6A 2018-11-19 2019-11-19 肝特异性调节性核酸序列 Pending CN113454104A (zh)

Applications Claiming Priority (3)

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

Publications (1)

Publication Number Publication Date
CN113454104A true CN113454104A (zh) 2021-09-28

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CN201980089303.6A Pending CN113454104A (zh) 2018-11-19 2019-11-19 肝特异性调节性核酸序列

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US (2) US12590129B2 (enExample)
EP (2) EP3883951A1 (enExample)
JP (3) JP7489161B2 (enExample)
CN (1) CN113454104A (enExample)
AU (2) AU2019382955B2 (enExample)
CA (1) CA3120253A1 (enExample)
GB (1) GB201818816D0 (enExample)
IL (1) IL283219A (enExample)
WO (2) WO2020104783A1 (enExample)

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN116555266A (zh) * 2023-06-09 2023-08-08 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

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Publication number Priority date Publication date Assignee Title
WO2021207526A1 (en) 2020-04-09 2021-10-14 The Regents Of The University Of California Humanized notch receptors with hinge domain
KR102726511B1 (ko) 2021-01-20 2024-11-07 주식회사 큐로젠 Ssu72 단백질 또는 Ssu72 단백질을 코딩하는 뉴클레오티드를 포함하는 간암 예방 또는 치료용 약학적 조성물
AU2022214501B2 (en) 2021-01-29 2025-05-22 Curogen Technology Co., Ltd. Pharmaceutical composition for treating liver diseases using ssu72
WO2023008925A1 (ko) * 2021-07-29 2023-02-02 주식회사 리보퓨틱스 융합 프로모터, 이를 포함하는 재조합 벡터 및 이를 이용하는 간암 치료용 조성물
JP2026516710A (ja) * 2023-04-20 2026-05-26 アスクバイオ インコーポレイテッド 肝臓特異的調節核酸配列

Citations (5)

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Publication number Priority date Publication date Assignee Title
US20020076798A1 (en) * 2000-06-20 2002-06-20 Miao Carol H. Liver-specific gene expression cassettes, and methods of use
US20030077812A1 (en) * 2001-05-14 2003-04-24 Mcarthur James G. Lentiviral vectors encoding clotting factors for gene therapy
CN102628041A (zh) * 2008-02-14 2012-08-08 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
CN102634515A (zh) * 2008-02-14 2012-08-15 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
CN108588097A (zh) * 2018-04-28 2018-09-28 北京五加和分子医学研究所有限公司 改造后的hbv基因组和相关组合物及其应用

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US4683195A (en) 1986-01-30 1987-07-28 Cetus Corporation Process for amplifying, detecting, and/or-cloning nucleic acid sequences
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
EP0933997A4 (en) 1996-09-06 2000-08-16 Chiron Corp METHODS AND COMPOSITIONS FOR LIVER-SPECIFIC ADMINISTRATION OF THERAPEUTIC MOLECULES USING RECOMBINANT AAV VECTORS
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US8815779B2 (en) 2009-09-16 2014-08-26 SwitchGear Genomics, Inc. Transcription biomarkers of biological responses and methods
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JP6879486B2 (ja) * 2015-03-17 2021-06-02 フリーイェ・ユニヴェルシテイト・ブリュッセルＶｒｉｊｅ Ｕｎｉｖｅｒｓｉｅｉｔ Ｂｒｕｓｓｅｌ Ｆｖｉｉｉおよびｆｉｘ用の最適化された肝臓特異的発現系
PE20181922A1 (es) 2016-04-15 2018-12-11 Univ Pennsylvania Terapia genica para tratar la hemofilia a
WO2018144709A2 (en) 2017-02-01 2018-08-09 The Trustees Of The University Of Pennsylvania Gene therapy for treating citrullenemia
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US20020076798A1 (en) * 2000-06-20 2002-06-20 Miao Carol H. Liver-specific gene expression cassettes, and methods of use
US20030077812A1 (en) * 2001-05-14 2003-04-24 Mcarthur James G. Lentiviral vectors encoding clotting factors for gene therapy
CN102628041A (zh) * 2008-02-14 2012-08-08 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
CN102634515A (zh) * 2008-02-14 2012-08-15 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
CN108588097A (zh) * 2018-04-28 2018-09-28 北京五加和分子医学研究所有限公司 改造后的hbv基因组和相关组合物及其应用

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NISHA NAIR 等: ""Computationally designed liver-specific transcriptional modules and hyperactive factor IX improve hepatic gene therapy"", BLOOD, vol. 123, no. 20, 17 March 2014 (2014-03-17), pages 3195 - 3199 *
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Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN116555266A (zh) * 2023-06-09 2023-08-08 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

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JP2024096349A (ja) 2024-07-12
JP2022507828A (ja) 2022-01-18
JP7489161B2 (ja) 2024-05-23
AU2019382955B2 (en) 2025-09-25
US12630595B2 (en) 2026-05-19
CA3120253A1 (en) 2020-05-28
EP3883951A1 (en) 2021-09-29
US12590129B2 (en) 2026-03-31
JP2026124829A (ja) 2026-07-31
WO2020104782A1 (en) 2020-05-28
AU2025287323A1 (en) 2026-03-26
JP7860164B2 (ja) 2026-05-15
IL283219A (en) 2021-06-30
US20210388347A1 (en) 2021-12-16
EP3883952A1 (en) 2021-09-29
GB201818816D0 (en) 2019-01-02
AU2019382955A1 (en) 2021-06-10
WO2020104783A1 (en) 2020-05-28
US20220009981A1 (en) 2022-01-13

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Country or region after: United Kingdom

Address after: Midlothian County, UK

Applicant after: Asbio UK Ltd.

Address before: Midlothian County, UK

Applicant before: Synpromics Ltd.

Country or region before: United Kingdom