JP2018522595A - Ｌ−ｄｏｐａの全身合成及び調節 - Google Patents

Ｌ−ｄｏｐａの全身合成及び調節 Download PDF

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JP2018522595A
JP2018522595A JP2018526302A JP2018526302A JP2018522595A JP 2018522595 A JP2018522595 A JP 2018522595A JP 2018526302 A JP2018526302 A JP 2018526302A JP 2018526302 A JP2018526302 A JP 2018526302A JP 2018522595 A JP2018522595 A JP 2018522595A
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seq
expression system
promoter
group
polypeptide
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JP2018522595A5 (cg-RX-API-DMAC10.html
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マイケル・マクドナルド
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ミョドパ・リミテッド
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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
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/33—Heterocyclic compounds
    • A61K31/395—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
    • A61K31/495—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with two or more nitrogen atoms as the only ring heteroatoms, e.g. piperazine or tetrazines
    • A61K31/505—Pyrimidines; Hydrogenated pyrimidines, e.g. trimethoprim
    • A61K31/519—Pyrimidines; Hydrogenated pyrimidines, e.g. trimethoprim ortho- or peri-condensed with heterocyclic rings
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/713—Double-stranded nucleic acids or oligonucleotides
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K45/00—Medicinal preparations containing active ingredients not provided for in groups A61K31/00 - A61K41/00
    • A61K45/06—Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/0012—Galenical forms characterised by the site of application
    • A61K9/0019—Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/14—Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
    • A61P25/16—Anti-Parkinson drugs
    • 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
    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/0004—Oxidoreductases (1.)
    • C12N9/0071—Oxidoreductases (1.) acting on paired donors with incorporation of molecular oxygen (1.14)
    • 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
    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • C12N9/78—Hydrolases (3) acting on carbon to nitrogen bonds other than peptide bonds (3.5)
    • 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
    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/88—Lyases (4.)
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y114/00—Oxidoreductases acting on paired donors, with incorporation or reduction of molecular oxygen (1.14)
    • C12Y114/16—Oxidoreductases acting on paired donors, with incorporation or reduction of molecular oxygen (1.14) with reduced pteridine as one donor, and incorporation of one atom of oxygen (1.14.16)
    • C12Y114/16002—Tyrosine 3-monooxygenase (1.14.16.2)
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y305/00—Hydrolases acting on carbon-nitrogen bonds, other than peptide bonds (3.5)
    • C12Y305/04—Hydrolases acting on carbon-nitrogen bonds, other than peptide bonds (3.5) in cyclic amidines (3.5.4)
    • C12Y305/04016—GTP cyclohydrolase I (3.5.4.16)
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y402/00—Carbon-oxygen lyases (4.2)
    • C12Y402/03—Carbon-oxygen lyases (4.2) acting on phosphates (4.2.3)
    • C12Y402/03012—6-Pyruvoyltetrahydropterin synthase (4.2.3.12)
    • 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
    • C12Y—ENZYMES
    • C12Y114/00—Oxidoreductases acting on paired donors, with incorporation or reduction of molecular oxygen (1.14)
    • C12Y114/13—Oxidoreductases acting on paired donors, with incorporation or reduction of molecular oxygen (1.14) with NADH or NADPH as one donor, and incorporation of one atom of oxygen (1.14.13)
    • C12Y114/13162—2,5-Diketocamphane 1,2-monooxygenase (1.14.13.162), i.e. camphor 1,2-monooxygenase

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • General Health & Medical Sciences (AREA)
  • General Engineering & Computer Science (AREA)
  • Biochemistry (AREA)
  • Biomedical Technology (AREA)
  • Medicinal Chemistry (AREA)
  • Biotechnology (AREA)
  • Molecular Biology (AREA)
  • Microbiology (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Epidemiology (AREA)
  • Neurosurgery (AREA)
  • Neurology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Virology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Psychology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Immunology (AREA)
  • Dermatology (AREA)
  • Proteomics, Peptides & Aminoacids (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)
  • Acyclic And Carbocyclic Compounds In Medicinal Compositions (AREA)
  • Peptides Or Proteins (AREA)
JP2018526302A 2015-08-03 2016-08-01 Ｌ−ｄｏｐａの全身合成及び調節 Pending JP2018522595A (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201562200151P 2015-08-03 2015-08-03
US62/200,151 2015-08-03
PCT/EP2016/068315 WO2017021359A1 (en) 2015-08-03 2016-08-01 Systemic synthesis and regulation of l-dopa

Publications (2)

Publication Number Publication Date
JP2018522595A true JP2018522595A (ja) 2018-08-16
JP2018522595A5 JP2018522595A5 (cg-RX-API-DMAC10.html) 2019-09-12

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US (1) US20190032079A1 (cg-RX-API-DMAC10.html)
EP (1) EP3331570A1 (cg-RX-API-DMAC10.html)
JP (1) JP2018522595A (cg-RX-API-DMAC10.html)
KR (1) KR20180034467A (cg-RX-API-DMAC10.html)
CN (1) CN108136048A (cg-RX-API-DMAC10.html)
CA (1) CA2992511A1 (cg-RX-API-DMAC10.html)
RU (1) RU2018104098A (cg-RX-API-DMAC10.html)
WO (1) WO2017021359A1 (cg-RX-API-DMAC10.html)

Cited By (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2022527597A (ja) * 2019-04-08 2022-06-02 ジェネトン 筋肉発現のためのハイブリッドプロモーター
JP2023509118A (ja) * 2019-12-24 2023-03-07 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 調節性核酸配列
JP2023524013A (ja) * 2020-04-29 2023-06-08 サリオジェン セラピューティクス インコーポレイテッド 家族性高コレステロール血症及び上昇した低密度リポタンパク質コレステロールの治療のための組成物及び方法
JP2023533852A (ja) * 2020-07-15 2023-08-04 ダンマールクス・テクニスケ・ウニヴェルシテート 治療用微生物
JP2023554198A (ja) * 2020-12-08 2023-12-26 マーヴルクス・リミテッド 発現ベクター組成物

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GB201420139D0 (en) 2014-11-12 2014-12-24 Ucl Business Plc Factor IX gene therapy
US20210093735A1 (en) * 2018-04-26 2021-04-01 The University Of North Carolina At Chapel Hill Methods and compositions for treatment of hemophilia
US12313635B2 (en) * 2018-07-27 2025-05-27 The Regents Of The University Of California Biomarker for thoracic aortic aneurysm
US10842885B2 (en) 2018-08-20 2020-11-24 Ucl Business Ltd Factor IX encoding nucleotides
GB201813528D0 (en) 2018-08-20 2018-10-03 Ucl Business Plc Factor IX encoding nucleotides
KR20210151785A (ko) * 2019-03-13 2021-12-14 제너레이션 바이오 컴퍼니 비바이러스성 dna 벡터 및 fviii 치료제 발현을 위한 이의 용도
GB2587319A (en) * 2019-08-12 2021-03-31 Healing Genes Llc Genetic construct
EP4107259A1 (en) * 2020-02-18 2022-12-28 Vrije Universiteit Brussel Novel combination of nucleic acid regulatory elements and methods and uses thereof
CN115515646A (zh) 2020-03-04 2022-12-23 波赛达治疗公司 用于治疗代谢性肝病的组合物和方法
GB202107473D0 (en) * 2021-05-26 2021-07-07 Univ Denmark Tech Dtu Therapeutic microbes
IL301255A (en) * 2020-10-09 2023-05-01 UCB Biopharma SRL Nucleic acid structures, viral vectors and viral particles
WO2023012313A1 (en) * 2021-08-04 2023-02-09 Genethon Hybrid promoters for gene expression in muscles and in the cns
WO2023049874A1 (en) * 2021-09-24 2023-03-30 Duke University Compositions for and methods of treating and/or preventing glutaric aciduria type-i
CN117106825B (zh) * 2023-08-28 2024-11-01 康霖生物科技（杭州）有限公司 用于治疗帕金森病的基因治疗载体及其用途

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Cited By (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2022527597A (ja) * 2019-04-08 2022-06-02 ジェネトン 筋肉発現のためのハイブリッドプロモーター
JP7541027B2 (ja) 2019-04-08 2024-08-27 ジェネトン 筋肉発現のためのハイブリッドプロモーター
JP2024159800A (ja) * 2019-04-08 2024-11-08 ジェネトン 筋肉発現のためのハイブリッドプロモーター
JP2023509118A (ja) * 2019-12-24 2023-03-07 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 調節性核酸配列
JP2023524013A (ja) * 2020-04-29 2023-06-08 サリオジェン セラピューティクス インコーポレイテッド 家族性高コレステロール血症及び上昇した低密度リポタンパク質コレステロールの治療のための組成物及び方法
JP2023533852A (ja) * 2020-07-15 2023-08-04 ダンマールクス・テクニスケ・ウニヴェルシテート 治療用微生物
JP2023554198A (ja) * 2020-12-08 2023-12-26 マーヴルクス・リミテッド 発現ベクター組成物
JP7889870B2 (ja) 2020-12-08 2026-07-14 マーヴルクス・リミテッド 発現ベクター組成物

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RU2018104098A (ru) 2019-09-06
EP3331570A1 (en) 2018-06-13
CN108136048A (zh) 2018-06-08
RU2018104098A3 (cg-RX-API-DMAC10.html) 2020-08-10
CA2992511A1 (en) 2017-02-09
KR20180034467A (ko) 2018-04-04
WO2017021359A1 (en) 2017-02-09
US20190032079A1 (en) 2019-01-31

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