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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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)
  • General Health & Medical Sciences (AREA)
  • Biochemistry (AREA)
  • Biotechnology (AREA)
  • Molecular Biology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • Biophysics (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Medicinal Chemistry (AREA)
  • General Engineering & Computer Science (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Toxicology (AREA)
  • Microbiology (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Virology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Epidemiology (AREA)
  • Public Health (AREA)
  • 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)
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
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

Publications (1)

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

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

Country Status (9)

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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=)

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

Families Citing this family (4)

* Cited by examiner, † Cited by third party
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

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
WO1998009524A1 (en) * 1996-09-06 1998-03-12 Chiron Corporation Methods and compositions for liver specific delivery of therapeutic molecules using recombinant aav vectors
CA2722238C (en) 2008-04-22 2017-11-28 Life Sciences Research Partners Vzw Liver-specific nucleic acid regulatory elements and methods and use thereof
US8815779B2 (en) * 2009-09-16 2014-08-26 SwitchGear Genomics, Inc. Transcription biomarkers of biological responses and methods
EP2479278A1 (en) * 2011-01-25 2012-07-25 Synpromics Ltd. Method for the construction of specific promoters
US11007280B2 (en) * 2015-03-17 2021-05-18 Vrije Universiteit Brussel Optimized liver-specific expression systems for FVIII and FIX
MX2018012537A (es) * 2016-04-15 2019-02-25 Univ Pennsylvania Terapia de genes para tratar hemofilia a.
WO2018144709A2 (en) * 2017-02-01 2018-08-09 The Trustees Of The University Of Pennsylvania Gene therapy for treating citrullenemia
EP3630160A1 (en) * 2017-05-24 2020-04-08 Universitat Autònoma De Barcelona Viral expression construct comprising a fibroblast growth factor 21 (fgf21) coding sequence
JP2020533971A (ja) * 2017-08-28 2020-11-26 アルター・バイオサイエンス・コーポレーション Ｉｌ−１５をベースとするｉｌ−７及びｉｌ−２１への融合物

Patent Citations (5)

* Cited by examiner, † Cited by third party
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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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 *
吕新跃 等: ""载脂蛋白CI的分子生物学研究进展"", 中国动脉硬化杂志, vol. 6, no. 4, 31 December 1998 (1998-12-31), pages 356 - 362 *

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 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
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
JP7489161B2 (ja) 2024-05-23
JP2024096349A (ja) 2024-07-12
US20220009981A1 (en) 2022-01-13
IL283219A (en) 2021-06-30
GB201818816D0 (en) 2019-01-02

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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