IL283219A - Liver-specific regulatory nucleic acid sequences - Google Patents

Liver-specific regulatory nucleic acid sequences

Info

Publication number
IL283219A
IL283219A IL283219A IL28321921A IL283219A IL 283219 A IL283219 A IL 283219A IL 283219 A IL283219 A IL 283219A IL 28321921 A IL28321921 A IL 28321921A IL 283219 A IL283219 A IL 283219A
Authority
IL
Israel
Prior art keywords
liver
nucleic acid
acid sequences
specific regulatory
regulatory nucleic
Prior art date
Application number
IL283219A
Other languages
English (en)
Hebrew (he)
Inventor
Roberts Michael
Manuel Iglesias Juan
Omar YANEZ-CUNA Jorge
Kippen Nicolle
Fraser Ross
Baker Katie
Original Assignee
Synpromics Ltd
Roberts Michael
Manuel Iglesias Juan
Jorge Omar Yanez Cuna
Kippen Nicolle
Fraser Ross
Baker Katie
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Synpromics Ltd, Roberts Michael, Manuel Iglesias Juan, Jorge Omar Yanez Cuna, Kippen Nicolle, Fraser Ross, Baker Katie filed Critical Synpromics Ltd
Publication of IL283219A publication Critical patent/IL283219A/en

Links

Classifications

    • 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

Landscapes

  • 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)
IL283219A 2018-11-19 2021-05-16 Liver-specific regulatory nucleic acid sequences IL283219A (en)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
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
IL283219A true IL283219A (en) 2021-06-30

Family

ID=64740036

Family Applications (1)

Application Number Title Priority Date Filing Date
IL283219A IL283219A (en) 2018-11-19 2021-05-16 Liver-specific regulatory nucleic acid sequences

Country Status (9)

Country Link
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=)

Families Citing this family (5)

* 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
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

Family Cites Families (18)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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
US7351813B2 (en) * 2000-06-20 2008-04-01 The Board Of Trustees Of The Leland Stanford Junior University Liver-specific gene expression cassettes, and methods of use
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
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
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

Also Published As

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

Similar Documents

Publication Publication Date Title
IL264966B (en) De novo synthesized nucleic acid libraries
IL271342A (en) Nucleic acid-guided nucleases
EP3784799C0 (en) METHOD FOR DETECTING NUCLEIC ACIDS
DK3523430T3 (da) Manipulerede nukleinsyremålrettede nukleinsyrer
PL3507366T3 (pl) Chemicznie modyfikowane jednoniciowe oligonukleotydy edytujące rna
PL3565905T5 (pl) Sekwencjonowanie kwasów nukleinowych z zastosowaniem odczynników powinowactwa
DK3433368T3 (da) Transreplikerende rna
EP3545076A4 (en) NUCLEIC ACID SEQUENCING DEVICE CONTAINING GRAPHEN
DK3337902T3 (da) 3'-utr-sekvenser til stabilisering af rna
DK3341482T3 (da) Kunstige nukleinsyremolekyler
IL283219A (en) Liver-specific regulatory nucleic acid sequences
IL254000B (en) Nucleic acid sequence assembly
DK4008782T3 (da) Fremgangsmåder til tilvejebringelse af enkeltstrenget rna
DK3707723T3 (da) Teknikker til indeksering af nukleinsyrer
HUE047175T2 (hu) Eljárások nukleinsavak tanulmányozására
DK3448990T3 (da) Forbedrede fremgangsmåder til modificering af målnukleinsyrer
DK3371310T3 (da) Nucleinsyrekonstruerede targetnucleinsyrer
DK3351633T3 (da) Antisense-nukleinsyre
PL3118311T3 (pl) Antysensowny kwas nukleinowy
EP3775210C0 (en) NUCLEIC ACID MOLECULES FOR PSEUDOURIDYLATION
EP3997205A4 (en) Rna sequencing methods
EP3808783A4 (en) Novel crosslinked alginic acid
EP3769769A4 (en) NUCLEIC ACID WITH REDUCED TOXICITY
DK3645717T3 (da) Modulære nukleinsyreadaptere
EP3366773A4 (en) NUCLEIC COMPLEX