KR20220119703A - 조절 핵산 서열 - Google Patents

조절 핵산 서열 Download PDF

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Publication number
KR20220119703A
KR20220119703A KR1020227025726A KR20227025726A KR20220119703A KR 20220119703 A KR20220119703 A KR 20220119703A KR 1020227025726 A KR1020227025726 A KR 1020227025726A KR 20227025726 A KR20227025726 A KR 20227025726A KR 20220119703 A KR20220119703 A KR 20220119703A
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KR
South Korea
Prior art keywords
seq
promoter
functional variant
muscle
specific
Prior art date
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Pending
Application number
KR1020227025726A
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English (en)
Korean (ko)
Inventor
조지 오마르 야네즈-쿠나
후안 마누엘 이글레시아스
싱클레어 쿠퍼
케이티 베이커
폴리세니 카츠피
린쿠 라잔
일레아나 게리니
안토니아 에브리피오티
키라 무라오
마이클 엘. 로버트
Original Assignee
아스클레피오스 바이오파마슈티컬, 인크.
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
Priority claimed from GBGB1919269.9A external-priority patent/GB201919269D0/en
Priority claimed from GBGB2012192.7A external-priority patent/GB202012192D0/en
Application filed by 아스클레피오스 바이오파마슈티컬, 인크. filed Critical 아스클레피오스 바이오파마슈티컬, 인크.
Publication of KR20220119703A publication Critical patent/KR20220119703A/ko
Pending legal-status Critical Current

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    • 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
    • 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
    • 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)
KR1020227025726A 2019-12-24 2020-12-24 조절 핵산 서열 Pending KR20220119703A (ko)

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
KR20220119703A true KR20220119703A (ko) 2022-08-30

Family

ID=74130280

Family Applications (1)

Application Number Title Priority Date Filing Date
KR1020227025726A Pending KR20220119703A (ko) 2019-12-24 2020-12-24 조절 핵산 서열

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=)

Families Citing this family (6)

* 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
CN115948403B (zh) * 2022-12-30 2023-09-15 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
AU2024406119A1 (en) * 2023-12-18 2026-04-02 F. Hoffmann-La Roche Ag Skeletal muscle-specific promoters

Family Cites Families (14)

* 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
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
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
CN102747082B (zh) * 2011-12-29 2014-06-18 华中农业大学 猪肌肉特异性itgb1bp2启动子及其应用
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
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
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

Also Published As

Publication number Publication date
WO2021130503A1 (en) 2021-07-01
AU2020412375A1 (en) 2022-06-30
CN115151646A (zh) 2022-10-04
IL294238A (en) 2022-08-01
ZA202207849B (en) 2023-12-20
CA3161735A1 (en) 2021-07-01
US20230233710A1 (en) 2023-07-27
JP2023509118A (ja) 2023-03-07
EP4081643A1 (en) 2022-11-02

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