JP2024524270A - 調節性核酸配列 - Google Patents

調節性核酸配列 Download PDF

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Publication number
JP2024524270A
JP2024524270A JP2023579317A JP2023579317A JP2024524270A JP 2024524270 A JP2024524270 A JP 2024524270A JP 2023579317 A JP2023579317 A JP 2023579317A JP 2023579317 A JP2023579317 A JP 2023579317A JP 2024524270 A JP2024524270 A JP 2024524270A
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JP
Japan
Prior art keywords
muscle
promoter
specific
synthetic
gene
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JP2023579317A
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Japanese (ja)
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JP2024524270A5 (https=
Inventor
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
マイケル・エル・ロバーツ
アントニア・イヴリピオティ
Original Assignee
アスクバイオ・ユーケー・リミテッド
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 GBGB2108997.4A external-priority patent/GB202108997D0/en
Application filed by アスクバイオ・ユーケー・リミテッド, 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 アスクバイオ・ユーケー・リミテッド
Publication of JP2024524270A publication Critical patent/JP2024524270A/ja
Publication of JP2024524270A5 publication Critical patent/JP2024524270A5/ja
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
    • C12N15/86—Viral vectors
    • 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
    • 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
    • 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

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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)
  • Molecular Biology (AREA)
  • General Health & Medical Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Biochemistry (AREA)
  • Veterinary Medicine (AREA)
  • Epidemiology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Virology (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (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)
  • Preparation Of Compounds By Using Micro-Organisms (AREA)
JP2023579317A 2021-06-23 2022-06-23 調節性核酸配列 Pending JP2024524270A (ja)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
GBGB2108997.4A GB202108997D0 (en) 2021-06-23 2021-06-23 Regulatory nucleic acid sequences
GB2108997.4 2021-06-23
GB2111317.0 2021-08-05
GB202111317 2021-08-05
PCT/GB2022/051611 WO2022269269A1 (en) 2021-06-23 2022-06-23 Regulatory nucleic acid sequences

Publications (2)

Publication Number Publication Date
JP2024524270A true JP2024524270A (ja) 2024-07-05
JP2024524270A5 JP2024524270A5 (https=) 2025-07-02

Family

ID=82655313

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2023579317A Pending JP2024524270A (ja) 2021-06-23 2022-06-23 調節性核酸配列

Country Status (8)

Country Link
US (1) US20250129381A1 (https=)
EP (1) EP4359548A1 (https=)
JP (1) JP2024524270A (https=)
KR (1) KR20240023643A (https=)
AU (1) AU2022297795A1 (https=)
CA (1) CA3221707A1 (https=)
IL (1) IL309630A (https=)
WO (1) WO2022269269A1 (https=)

Families Citing this family (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
GB202319008D0 (en) * 2023-12-13 2024-01-24 Ucl Business Ltd AAV-mediated gene therapy of diseases caused by impaired function of GLDC

Family Cites Families (12)

* 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
US6221349B1 (en) 1998-10-20 2001-04-24 Avigen, Inc. Adeno-associated vectors for expression of factor VIII by target cells
US6200560B1 (en) 1998-10-20 2001-03-13 Avigen, Inc. Adeno-associated virus vectors for expression of factor VIII by target cells
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
PL3097197T3 (pl) * 2014-01-21 2021-06-28 Vrije Universiteit Brussel Mięśniowo-specyficzne elementy regulatorowe kwasów nukleinowych oraz sposoby i ich zastosowanie
TWI908746B (zh) * 2019-11-28 2025-12-21 美商銳進科斯生物股份有限公司 微小肌縮蛋白基因療法之構築體及其用途
EP4081643A1 (en) * 2019-12-24 2022-11-02 Asklepios Biopharmaceutical, Inc. Regulatory nucleic acid sequences
EP4192580A4 (en) * 2020-08-05 2025-06-18 AskBio Inc. Methods for treating heart disease and congestive heart failure and administration of AAV vectors
CA3195177A1 (en) * 2020-10-07 2022-04-14 Asklepios Biopharmaceutical, Inc. Therapeutic adeno-associated virus delivery of fukutin related protein (fkrp) for treating dystroglycanopathy disorders including limb girdle 2i (lgmd2i)

Non-Patent Citations (3)

* Cited by examiner, † Cited by third party
Title
ACTA NATURAE, vol. 13, no. 1, JPN6026012989, 2021, pages 47 - 58, ISSN: 0005834679 *
MOLECULAR THERAPY, vol. 23, no. 1, JPN6026012990, 2015, pages 43 - 52, ISSN: 0005834680 *
SCIENCE ADVANCES, vol. 7, no. 1444, JPN6026012991, 2021, pages 1 - 23, ISSN: 0005834681 *

Also Published As

Publication number Publication date
WO2022269269A1 (en) 2022-12-29
EP4359548A1 (en) 2024-05-01
KR20240023643A (ko) 2024-02-22
IL309630A (en) 2024-02-01
CA3221707A1 (en) 2022-12-29
AU2022297795A1 (en) 2024-01-04
US20250129381A1 (en) 2025-04-24

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