JP2024513907A - 筋特異的遺伝子発現用の人工調節カセット - Google Patents

筋特異的遺伝子発現用の人工調節カセット Download PDF

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JP2024513907A
JP2024513907A JP2023561616A JP2023561616A JP2024513907A JP 2024513907 A JP2024513907 A JP 2024513907A JP 2023561616 A JP2023561616 A JP 2023561616A JP 2023561616 A JP2023561616 A JP 2023561616A JP 2024513907 A JP2024513907 A JP 2024513907A
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muscle
enhancer
promoter
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JP2024513907A5 (https=
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エス． チェンバレン，ジェフリー
ディー． ハウシュカ，ステファン
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University of Washington
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University of Washington
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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
    • 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
    • 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
    • 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
    • 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)
  • Genetics & Genomics (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • Biomedical Technology (AREA)
  • Organic Chemistry (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Zoology (AREA)
  • Microbiology (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Molecular Biology (AREA)
  • Biochemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Biophysics (AREA)
  • Virology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
JP2023561616A 2021-04-09 2022-04-07 筋特異的遺伝子発現用の人工調節カセット Pending JP2024513907A (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US202163173295P 2021-04-09 2021-04-09
US63/173,295 2021-04-09
PCT/US2022/023915 WO2022216988A2 (en) 2021-04-09 2022-04-07 Artificial regulatory cassettes for muscle-specific gene expression

Publications (2)

Publication Number Publication Date
JP2024513907A true JP2024513907A (ja) 2024-03-27
JP2024513907A5 JP2024513907A5 (https=) 2025-04-15

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JP2023561616A Pending JP2024513907A (ja) 2021-04-09 2022-04-07 筋特異的遺伝子発現用の人工調節カセット

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Country Link
US (1) US20240200093A1 (https=)
EP (2) EP4707401A2 (https=)
JP (1) JP2024513907A (https=)
WO (1) WO2022216988A2 (https=)

Families Citing this family (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2024216116A1 (en) * 2023-04-14 2024-10-17 Precision Biosciences, Inc. Muscle-specific expression cassettes
WO2024216118A1 (en) * 2023-04-14 2024-10-17 Precision Biosciences, Inc. Muscle-specific expression cassettes
KR20260035966A (ko) * 2023-07-10 2026-03-13 케이트 테라퓨틱스, 인크. Lgmd2b/디스펄린병증을 치료하기 위한 aav 유전자 치료 접근법
WO2025129079A1 (en) * 2023-12-13 2025-06-19 Tenaya Therapeutics, Inc. Cardiac-specific promoters for gene therapy

Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2018503374A (ja) * 2015-01-16 2018-02-08 ユニバーシティ オブ ワシントンＵｎｉｖｅｒｓｉｔｙ ｏｆ Ｗａｓｈｉｎｇｔｏｎ 新規のマイクロジストロフィンおよび使用の関連する方法

Family Cites Families (9)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US6183984B1 (en) * 1991-11-12 2001-02-06 Arch Development Corporation Sequences for promoting epidermal cell-specific transcription
US6967019B2 (en) * 1999-04-06 2005-11-22 The Regents Of The University Of California Production of pancreatic islet cells and delivery of insulin
ATE373095T1 (de) * 2000-07-14 2007-09-15 Univ Michigan Mutierte muskelspezifische enhancer
WO2002077161A2 (en) * 2001-03-12 2002-10-03 Progenetics Llc Production of high levels of trangenic factor ix without gene rescue, and its therapeutic uses
US10196636B2 (en) * 2011-04-21 2019-02-05 Nationwide Children's Hospital, Inc. Recombinant virus products and methods for inhibition of expression of myotilin
US20130136729A1 (en) * 2011-11-11 2013-05-30 University of Virginia Patent Foundation, d/b/a University of Virginia Licensing & Ventures Group Compositions and methods for targeting and treating diseases and injuries using adeno-associated virus vectors
WO2013102904A1 (en) * 2012-01-05 2013-07-11 Hadasit Medical Research Services & Development Ltd. Methods and compositions for gene delivery
EP3405579A1 (en) * 2016-01-22 2018-11-28 Modernatx, Inc. Messenger ribonucleic acids for the production of intracellular binding polypeptides and methods of use thereof
EP4004214A1 (en) * 2019-07-26 2022-06-01 RegenxBio Inc. Engineered nucleic acid regulatory element and methods of uses thereof

Patent Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2018503374A (ja) * 2015-01-16 2018-02-08 ユニバーシティ オブ ワシントンＵｎｉｖｅｒｓｉｔｙ ｏｆ Ｗａｓｈｉｎｇｔｏｎ 新規のマイクロジストロフィンおよび使用の関連する方法

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Publication number Publication date
EP4319874A4 (en) 2025-05-21
US20240200093A1 (en) 2024-06-20
EP4707401A2 (en) 2026-03-11
WO2022216988A3 (en) 2022-11-10
WO2022216988A2 (en) 2022-10-13
EP4319874A2 (en) 2024-02-14
WO2022216988A9 (en) 2023-03-16

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