JP2023509118A - 調節性核酸配列 - Google Patents

調節性核酸配列 Download PDF

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JP2023509118A
JP2023509118A JP2022539145A JP2022539145A JP2023509118A JP 2023509118 A JP2023509118 A JP 2023509118A JP 2022539145 A JP2022539145 A JP 2022539145A JP 2022539145 A JP2022539145 A JP 2022539145A JP 2023509118 A JP2023509118 A JP 2023509118A
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seq
promoter
functional variant
specific
crm
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JP2023509118A5 (enExample
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ホルヘ・オマール・ジャニェス－クニャ
フアン・マヌエル・イグレシアス
シンクレア・クーパー
ケイティ・ベイカー
ポリュクセニ・カツォウピ
リンク・ラジャン
イリアナ・ゲリーニ
アントニア・イヴリピオティ
キラ・モウラオ
マイケル・エル・ロバーツ
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アスクレピオス バイオファーマシューティカル， インコーポレイテッド
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Priority claimed from GBGB1919269.9A external-priority patent/GB201919269D0/en
Priority claimed from GBGB2012192.7A external-priority patent/GB202012192D0/en
Application filed by アスクレピオス バイオファーマシューティカル， インコーポレイテッド filed Critical アスクレピオス バイオファーマシューティカル， インコーポレイテッド
Publication of JP2023509118A publication Critical patent/JP2023509118A/ja
Publication of JP2023509118A5 publication Critical patent/JP2023509118A5/ja
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    • 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
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    • 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
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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
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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
    • C12N2750/14171—Demonstrated in vivo effect
    • 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
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    • 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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  • Wood Science & Technology (AREA)
  • Biochemistry (AREA)
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  • Animal Behavior & Ethology (AREA)
  • Medicinal Chemistry (AREA)
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  • Microbiology (AREA)
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  • 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)
JP2022539145A 2019-12-24 2020-12-24 調節性核酸配列 Pending JP2023509118A (ja)

Applications Claiming Priority (5)

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

Publications (2)

Publication Number Publication Date
JP2023509118A true JP2023509118A (ja) 2023-03-07
JP2023509118A5 JP2023509118A5 (enExample) 2024-01-05

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JP2022539145A Pending JP2023509118A (ja) 2019-12-24 2020-12-24 調節性核酸配列

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

Families Citing this family (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
FI4103214T3 (fi) 2020-02-13 2026-07-03 Tenaya Therapeutics Inc Geeniterapian vektorit sydänsairauden hoitoon
CN116406297A (zh) * 2020-08-05 2023-07-07 阿斯克肋匹奥生物制药公司 治疗心脏疾患和充血性心力衰竭以及施用aav载体的方法
CA3221707A1 (en) * 2021-06-23 2022-12-29 Jorge Omar YANEZ-CUNA Regulatory nucleic acid sequences
JP2025512574A (ja) * 2022-04-22 2025-04-17 スペースクラフト セブン リミテッド ライアビリティ カンパニー ＡＡＶベクターを使用するＢ細胞リンパ腫２関連Ａｎｔｈａｎｏｇｅｎｅ ３ （ＢＡＧ３）遺伝子治療
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
CN122396772A (zh) * 2023-12-18 2026-07-14 豪夫迈·罗氏有限公司 骨骼肌特异性启动子

Citations (3)

* Cited by examiner, † Cited by third party
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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
JP2018522595A (ja) * 2015-08-03 2018-08-16 ミョドパ・リミテッド Ｌ−ｄｏｐａの全身合成及び調節
US20190169691A1 (en) * 2016-03-01 2019-06-06 Washington State University Heritable epigenetic modifications as markers of chemotherapy exposure

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US4683195A (en) 1986-01-30 1987-07-28 Cetus Corporation Process for amplifying, detecting, and/or-cloning nucleic acid sequences
CN1247785C (zh) 2000-10-13 2006-03-29 希龙公司 巨细胞病毒内含子a片段
US20030074679A1 (en) * 2000-12-12 2003-04-17 Schwartz Robert J. Administration of nucleic acid sequence to female animal to enhance growth in offspring
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
EP2494058B1 (en) * 2009-10-29 2017-04-05 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
AU2015208247B2 (en) * 2014-01-21 2021-05-27 Universiteit Gent Muscle-specific nucleic acid regulatory elements and methods and use thereof
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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Patent Citations (3)

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Publication number Priority date Publication date Assignee Title
JP2018522595A (ja) * 2015-08-03 2018-08-16 ミョドパ・リミテッド Ｌ−ｄｏｐａの全身合成及び調節
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
US20190169691A1 (en) * 2016-03-01 2019-06-06 Washington State University Heritable epigenetic modifications as markers of chemotherapy exposure

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Publication number Publication date
WO2021130503A1 (en) 2021-07-01
ZA202207849B (en) 2023-12-20
IL294238A (en) 2022-08-01
US20230233710A1 (en) 2023-07-27
AU2020412375A1 (en) 2022-06-30
KR20220119703A (ko) 2022-08-30
EP4081643A1 (en) 2022-11-02
CA3161735A1 (en) 2021-07-01
CN115151646A (zh) 2022-10-04

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