CN115151646A - 调节核酸序列 - Google Patents

调节核酸序列 Download PDF

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Publication number
CN115151646A
CN115151646A CN202080097425.2A CN202080097425A CN115151646A CN 115151646 A CN115151646 A CN 115151646A CN 202080097425 A CN202080097425 A CN 202080097425A CN 115151646 A CN115151646 A CN 115151646A
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China
Prior art keywords
seq
promoter
functional variant
crm
specific
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Pending
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CN202080097425.2A
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English (en)
Chinese (zh)
Inventor
乔治·奥马尔·亚尼兹库纳
胡安·曼努埃尔·伊格莱西亚斯
辛克莱·库珀
卡蒂·贝克
波利克塞妮·卡特索皮
林库·拉扬
伊利安娜·格里尼
安东尼娅·埃夫里皮奥蒂
基拉·穆拉欧
迈克尔·L·罗伯茨
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AskBio Inc
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Asklepios Biopharmaceutical Inc
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Filing date
Publication date
Priority claimed from GBGB1919269.9A external-priority patent/GB201919269D0/en
Priority claimed from GBGB2012192.7A external-priority patent/GB202012192D0/en
Application filed by Asklepios Biopharmaceutical Inc filed Critical Asklepios Biopharmaceutical Inc
Publication of CN115151646A publication Critical patent/CN115151646A/zh
Pending legal-status Critical Current

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    • 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
    • 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
    • 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
    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/32—Special delivery means, e.g. tissue-specific
    • 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
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • 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
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14171—Demonstrated in vivo effect
    • 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
    • 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
    • C12N5/0652—Cells of skeletal and connective tissues; Mesenchyme
    • C12N5/0657—Cardiomyocytes; Heart cells

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Biotechnology (AREA)
  • Biomedical Technology (AREA)
  • General Health & Medical Sciences (AREA)
  • Molecular Biology (AREA)
  • Zoology (AREA)
  • General Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • Wood Science & Technology (AREA)
  • Biochemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Epidemiology (AREA)
  • Medicinal Chemistry (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Virology (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)
CN202080097425.2A 2019-12-24 2020-12-24 调节核酸序列 Pending CN115151646A (zh)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
GBGB1919269.9A GB201919269D0 (en) 2019-12-24 2019-12-24 Regulatory nucleic acid sequences
GB1919269.9 2019-12-24
GBGB2012192.7A GB202012192D0 (en) 2020-08-05 2020-08-05 Regulatory nucleic acid sequences
GB2012192.7 2020-08-05
PCT/GB2020/053371 WO2021130503A1 (en) 2019-12-24 2020-12-24 Regulatory nucleic acid sequences

Publications (1)

Publication Number Publication Date
CN115151646A true CN115151646A (zh) 2022-10-04

Family

ID=74130280

Family Applications (1)

Application Number Title Priority Date Filing Date
CN202080097425.2A Pending CN115151646A (zh) 2019-12-24 2020-12-24 调节核酸序列

Country Status (10)

Country Link
US (1) US20230233710A1 (https=)
EP (1) EP4081643A1 (https=)
JP (1) JP2023509118A (https=)
KR (1) KR20220119703A (https=)
CN (1) CN115151646A (https=)
AU (1) AU2020412375A1 (https=)
CA (1) CA3161735A1 (https=)
IL (1) IL294238A (https=)
WO (1) WO2021130503A1 (https=)
ZA (1) ZA202207849B (https=)

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2024138812A1 (zh) * 2022-12-30 2024-07-04 广州派真生物技术有限公司 在哺乳动物肌肉中特异性启动基因的启动子序列及其应用

Families Citing this family (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP4103214B1 (en) 2020-02-13 2026-04-29 Tenaya Therapeutics, Inc. Gene therapy vectors for treating heart disease
EP4192580A4 (en) * 2020-08-05 2025-06-18 AskBio Inc. Methods for treating heart disease and congestive heart failure and administration of AAV vectors
AU2022297795A1 (en) * 2021-06-23 2024-01-04 AskBio Inc. Regulatory nucleic acid sequences
KR20250012749A (ko) * 2022-04-22 2025-01-24 스페이스크래프트 세븐, 엘엘씨 Aav 벡터를 사용한 b-세포 림프종 2-관련 안타노겐 3(bag3) 유전자 요법
AU2024406119A1 (en) * 2023-12-18 2026-04-02 F. Hoffmann-La Roche Ag Skeletal muscle-specific promoters

Citations (4)

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Publication number Priority date Publication date Assignee Title
CN102747082A (zh) * 2011-12-29 2012-10-24 华中农业大学 猪肌肉特异性itgb1bp2启动子及其应用
CN103074373A (zh) * 2011-10-26 2013-05-01 南京医科大学 肌肉组织特异性表达Follistatin以提高牛瘦肉率的转基因载体
US20170253927A1 (en) * 2016-03-01 2017-09-07 Washington State University Heritable epigenetic modifications as markers of chemotherapy exposure
CN108018311A (zh) * 2016-11-04 2018-05-11 中国科学院上海生命科学研究院 通过基因编辑特异靶向肌肉组织mstn治疗恶病质

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US4683195A (en) 1986-01-30 1987-07-28 Cetus Corporation Process for amplifying, detecting, and/or-cloning nucleic acid sequences
ES2261489T3 (es) 2000-10-13 2006-11-16 Chiron Corporation Fragmentos de intron a de citomegalovirus.
US8106180B2 (en) 2003-08-07 2012-01-31 Whitehead Institute For Biomedical Research Methods and products for expression of micro RNAs
US20050142581A1 (en) 2003-09-04 2005-06-30 Griffey Richard H. Microrna as ligands and target molecules
WO2005056761A2 (en) 2003-12-05 2005-06-23 Massachusetts Institute Of Technology REAGENTS AND METHODS FOR IDENTIFICATION OF RNAi PATHWAY GENES AND CHEMICAL MODULATORS OF RNAi
KR100614827B1 (ko) 2004-05-06 2006-08-25 재단법인서울대학교산학협력재단 양방향 은닉 마코프 모델을 이용한 완숙한마이크로알엔에이 위치예측방법 및 이를 구현하기 위한컴퓨터 프로그램을 기록한 저장매체
WO2011051450A1 (en) * 2009-10-29 2011-05-05 Vib Vzw Cardiac-specific nucleic acid regulatory elements and methods and use thereof
US20190032079A1 (en) * 2015-08-03 2019-01-31 Myodopa Limited Systemic synthesis and regulation of l-dopa
CN105063079A (zh) * 2015-08-15 2015-11-18 内蒙古大学 一种三顺反子肌肉特异双向共表达基因转移体及制备方法
EP3596112A2 (en) * 2017-03-17 2020-01-22 Newcastle University Adeno-associated virus vector delivery of a fragment of micro-dystrophin to treat muscular dystrophy

Patent Citations (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN103074373A (zh) * 2011-10-26 2013-05-01 南京医科大学 肌肉组织特异性表达Follistatin以提高牛瘦肉率的转基因载体
CN102747082A (zh) * 2011-12-29 2012-10-24 华中农业大学 猪肌肉特异性itgb1bp2启动子及其应用
US20170253927A1 (en) * 2016-03-01 2017-09-07 Washington State University Heritable epigenetic modifications as markers of chemotherapy exposure
CN108018311A (zh) * 2016-11-04 2018-05-11 中国科学院上海生命科学研究院 通过基因编辑特异靶向肌肉组织mstn治疗恶病质

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EUKARYOTA; METAZOA ET AL: "Homo sapiens Sharpr-MPRA regulatory region 4357 (LOC112067713) on chromosome 11", Retrieved from the Internet <URL:GENEBANK> *

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2024138812A1 (zh) * 2022-12-30 2024-07-04 广州派真生物技术有限公司 在哺乳动物肌肉中特异性启动基因的启动子序列及其应用

Also Published As

Publication number Publication date
WO2021130503A1 (en) 2021-07-01
AU2020412375A1 (en) 2022-06-30
IL294238A (en) 2022-08-01
ZA202207849B (en) 2023-12-20
CA3161735A1 (en) 2021-07-01
KR20220119703A (ko) 2022-08-30
US20230233710A1 (en) 2023-07-27
JP2023509118A (ja) 2023-03-07
EP4081643A1 (en) 2022-11-02

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