JP2022544004A - 操作された核酸調節エレメントならびにその使用方法 - Google Patents

操作された核酸調節エレメントならびにその使用方法 Download PDF

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JP2022544004A
JP2022544004A JP2022503959A JP2022503959A JP2022544004A JP 2022544004 A JP2022544004 A JP 2022544004A JP 2022503959 A JP2022503959 A JP 2022503959A JP 2022503959 A JP2022503959 A JP 2022503959A JP 2022544004 A JP2022544004 A JP 2022544004A
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aav
seq
promoter
expression
transgene
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シャオ，チュンピン
マクドゥーガルド，デヴィン
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リジェネックスバイオ インコーポレイテッド
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    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • 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
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    • 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
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    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/005—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from viruses
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • 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
    • C12N2830/007—Vector systems having a special element relevant for transcription cell cycle specific enhancer/promoter combination
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • 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
    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/15—Vector systems having a special element relevant for transcription chimeric 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
    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/30—Vector systems having a special element relevant for transcription being an enhancer not forming part of the promoter region

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  • Genetics & Genomics (AREA)
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  • Wood Science & Technology (AREA)
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  • Chemical & Material Sciences (AREA)
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  • General Health & Medical Sciences (AREA)
  • Physics & Mathematics (AREA)
  • Virology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines Containing Antibodies Or Antigens For Use As Internal Diagnostic Agents (AREA)
JP2022503959A 2019-07-26 2020-07-24 操作された核酸調節エレメントならびにその使用方法 Pending JP2022544004A (ja)

Applications Claiming Priority (7)

Application Number Priority Date Filing Date Title
US201962879358P 2019-07-26 2019-07-26
US62/879,358 2019-07-26
US202062967480P 2020-01-29 2020-01-29
US62/967,480 2020-01-29
US202063023152P 2020-05-11 2020-05-11
US63/023,152 2020-05-11
PCT/US2020/043578 WO2021021661A1 (en) 2019-07-26 2020-07-24 Engineered nucleic acid regulatory element and methods of uses thereof

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JP2022544004A true JP2022544004A (ja) 2022-10-17
JP2022544004A5 JP2022544004A5 (https=) 2023-08-02

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US (1) US20230042103A1 (https=)
EP (1) EP4004214A1 (https=)
JP (1) JP2022544004A (https=)
CA (1) CA3145112A1 (https=)
IL (1) IL289922A (https=)
WO (1) WO2021021661A1 (https=)

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CN113322281B (zh) * 2021-05-12 2024-01-05 成都金唯科生物科技有限公司 一种高效组织特异性表达rs1蛋白的重组腺相关病毒及应用
US20250025574A1 (en) * 2021-10-28 2025-01-23 Regenxbio Inc. Engineered nucleic acid regulatory elements and methods and uses thereof
CN114470241A (zh) * 2022-03-03 2022-05-13 中日友好医院(中日友好临床医学研究所) 肝脏靶向性γ-干扰素表达载体在制备治疗抗肝脏肿瘤的药物中的应用
WO2023178053A1 (en) 2022-03-13 2023-09-21 Regenxbio Inc. Modified muscle-specific promoters
US20250277004A1 (en) 2022-04-18 2025-09-04 Regenxbio Inc. Hybrid aav capsids
CN119213133B (zh) 2022-05-16 2026-01-06 上海天泽云泰生物医药有限公司 用于治疗i型戊二酸尿症的重组aav载体
JP2025535114A (ja) * 2022-10-11 2025-10-22 メイラグティーエックス ユーケー アイアイ リミティド 中枢神経系における遺伝子発現のための核酸調節エレメント及び使用方法
EP4601710A2 (en) * 2022-10-11 2025-08-20 RegenxBio Inc. Engineered nucleic acid regulatory elements and methods and uses thereof
CN117947040A (zh) * 2022-10-31 2024-04-30 苏州荷光科汇生物科技有限公司 用于目的基因的表达盒及其应用
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KR20260005328A (ko) * 2023-04-28 2026-01-09 청두 오리젠 바이오테크놀로지 씨오., 엘티디. 항vegf 단백질을 코딩하는 핵산, 폴리뉴클레오타이드 발현 카세트 및 재조합 아데노 관련 바이러스
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Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US12290574B2 (en) 2018-08-24 2025-05-06 Spark Therapeutics, Inc. Optimized promoter sequences, intron-free expression constructs and methods of use

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IL289922A (en) 2022-03-01
CA3145112A1 (en) 2021-02-04
US20230042103A1 (en) 2023-02-09
WO2021021661A1 (en) 2021-02-04
EP4004214A1 (en) 2022-06-01

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