KR20180034467A - L-dopa의 전신 합성 및 조절 - Google Patents

L-dopa의 전신 합성 및 조절 Download PDF

Info

Publication number
KR20180034467A
KR20180034467A KR1020187003356A KR20187003356A KR20180034467A KR 20180034467 A KR20180034467 A KR 20180034467A KR 1020187003356 A KR1020187003356 A KR 1020187003356A KR 20187003356 A KR20187003356 A KR 20187003356A KR 20180034467 A KR20180034467 A KR 20180034467A
Authority
KR
South Korea
Prior art keywords
seq
expression system
ala
leu
glu
Prior art date
Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
Withdrawn
Application number
KR1020187003356A
Other languages
English (en)
Korean (ko)
Inventor
마이클 맥도날드
Original Assignee
마이오도파 리미티드
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by 마이오도파 리미티드 filed Critical 마이오도파 리미티드
Publication of KR20180034467A publication Critical patent/KR20180034467A/ko
Withdrawn legal-status Critical Current

Links

Images

Classifications

    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86Viral vectors
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/33Heterocyclic compounds
    • A61K31/395Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
    • A61K31/495Heterocyclic 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
    • A61K31/505Pyrimidines; Hydrogenated pyrimidines, e.g. trimethoprim
    • A61K31/519Pyrimidines; Hydrogenated pyrimidines, e.g. trimethoprim ortho- or peri-condensed with heterocyclic rings
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/70Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088Compounds having three or more nucleosides or nucleotides
    • A61K31/713Double-stranded nucleic acids or oligonucleotides
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00Medicinal preparations containing peptides
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K45/00Medicinal preparations containing active ingredients not provided for in groups A61K31/00 - A61K41/00
    • A61K45/06Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00Medicinal preparations characterised by special physical form
    • A61K9/0012Galenical forms characterised by the site of application
    • A61K9/0019Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • A61P25/14Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
    • A61P25/16Anti-Parkinson drugs
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N9/00Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/0004Oxidoreductases (1.)
    • C12N9/0071Oxidoreductases (1.) acting on paired donors with incorporation of molecular oxygen (1.14)
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N9/00Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14Hydrolases (3)
    • C12N9/78Hydrolases (3) acting on carbon to nitrogen bonds other than peptide bonds (3.5)
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N9/00Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/88Lyases (4.)
    • C12Y114/15002
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12YENZYMES
    • C12Y114/00Oxidoreductases acting on paired donors, with incorporation or reduction of molecular oxygen (1.14)
    • C12Y114/16Oxidoreductases 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)
    • C12Y114/16002Tyrosine 3-monooxygenase (1.14.16.2)
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12YENZYMES
    • C12Y305/00Hydrolases acting on carbon-nitrogen bonds, other than peptide bonds (3.5)
    • C12Y305/04Hydrolases acting on carbon-nitrogen bonds, other than peptide bonds (3.5) in cyclic amidines (3.5.4)
    • C12Y305/04016GTP cyclohydrolase I (3.5.4.16)
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12YENZYMES
    • C12Y402/00Carbon-oxygen lyases (4.2)
    • C12Y402/03Carbon-oxygen lyases (4.2) acting on phosphates (4.2.3)
    • C12Y402/030126-Pyruvoyltetrahydropterin synthase (4.2.3.12)
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14171Demonstrated in vivo effect
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12YENZYMES
    • C12Y114/00Oxidoreductases acting on paired donors, with incorporation or reduction of molecular oxygen (1.14)
    • C12Y114/13Oxidoreductases 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)
    • C12Y114/131622,5-Diketocamphane 1,2-monooxygenase (1.14.13.162), i.e. camphor 1,2-monooxygenase

Landscapes

  • 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)
  • Zoology (AREA)
  • Wood Science & Technology (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)
  • Animal Behavior & Ethology (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Epidemiology (AREA)
  • Neurosurgery (AREA)
  • Neurology (AREA)
  • Virology (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Psychology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Immunology (AREA)
  • Dermatology (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)
  • Acyclic And Carbocyclic Compounds In Medicinal Compositions (AREA)
KR1020187003356A 2015-08-03 2016-08-01 L-dopa의 전신 합성 및 조절 Withdrawn KR20180034467A (ko)

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 (1)

Publication Number Publication Date
KR20180034467A true KR20180034467A (ko) 2018-04-04

Family

ID=56682101

Family Applications (1)

Application Number Title Priority Date Filing Date
KR1020187003356A Withdrawn KR20180034467A (ko) 2015-08-03 2016-08-01 L-dopa의 전신 합성 및 조절

Country Status (8)

Country Link
US (1) US20190032079A1 (enExample)
EP (1) EP3331570A1 (enExample)
JP (1) JP2018522595A (enExample)
KR (1) KR20180034467A (enExample)
CN (1) CN108136048A (enExample)
CA (1) CA2992511A1 (enExample)
RU (1) RU2018104098A (enExample)
WO (1) WO2017021359A1 (enExample)

Families Citing this family (17)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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
WO2020023922A1 (en) * 2018-07-27 2020-01-30 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
CA3133255A1 (en) * 2019-03-13 2020-09-17 Generation Bio Co. Non-viral dna vectors and uses thereof for expressing fviii therapeutics
CN113747926A (zh) * 2019-04-08 2021-12-03 吉尼松公司 用于肌肉表达的杂合启动子
GB2587319A (en) * 2019-08-12 2021-03-31 Healing Genes Llc Genetic construct
CN115151646A (zh) * 2019-12-24 2022-10-04 阿斯克肋匹奥生物制药公司 调节核酸序列
CN115298307A (zh) * 2020-02-18 2022-11-04 布鲁塞尔自由大学 核酸调节元件的新组合及其方法和用途
CA3169529A1 (en) * 2020-03-04 2021-09-10 Jingjing Jiang Compositions and methods for the treatment of metabolic liver disorders
AU2021263938A1 (en) * 2020-04-29 2022-10-20 Saliogen Therapeutics, Inc. Compositions and methods for treatment of familial hypercholesterolemia and elevated low-density lipoprotein cholesterol
US20230263864A1 (en) * 2020-07-15 2023-08-24 Danmarks Tekniske Universitet Therapeutic microbes
GB2601752A (en) * 2020-12-08 2022-06-15 Maavrx Ltd Expression vector
CN117897492A (zh) * 2021-08-04 2024-04-16 吉尼松公司 用于肌肉和cns中的基因表达的杂合启动子
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

Family Cites Families (11)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US4522811A (en) 1982-07-08 1985-06-11 Syntex (U.S.A.) Inc. Serial injection of muramyldipeptides and liposomes enhances the anti-infective activity of muramyldipeptides
WO1987000201A1 (en) 1985-07-05 1987-01-15 Whitehead Institute For Biomedical Research Epithelial cells expressing foreign genetic material
EP0633318A1 (en) 1987-09-11 1995-01-11 Whitehead Institute For Biomedical Research Transduced fibroblasts and uses therefor
US5399346A (en) 1989-06-14 1995-03-21 The United States Of America As Represented By The Department Of Health And Human Services Gene therapy
US5677158A (en) 1995-06-07 1997-10-14 Research Foundation Of State University Of New York In vitro packaging of adeno-associated virus DNA
US6451306B1 (en) 1998-04-15 2002-09-17 The Regents Of The University Of California Methods for therapy of neurodegenerative disease of the brain
ES2326893T3 (es) 1998-05-27 2009-10-21 Genzyme Corporation Vectores aav para la fabricacion de medicamentos para administracion potenciada por conveccion.
GB0024550D0 (enExample) * 2000-10-06 2000-11-22 Oxford Biomedica Ltd
KR100456062B1 (ko) * 2001-06-18 2004-11-08 박영식 재조합 대장균에 의한 테리딘 화합물의 제조방법
US20120309816A1 (en) * 2009-11-09 2012-12-06 Genepod Therapeutics Ab Novel viral vector construct for neuron specific optimized continuous DOPA synthesis in vivo
GB201118636D0 (en) * 2011-10-28 2011-12-07 Oxford Biomedica Ltd Nucleotide sequence

Also Published As

Publication number Publication date
JP2018522595A (ja) 2018-08-16
RU2018104098A3 (enExample) 2020-08-10
CA2992511A1 (en) 2017-02-09
US20190032079A1 (en) 2019-01-31
WO2017021359A1 (en) 2017-02-09
CN108136048A (zh) 2018-06-08
RU2018104098A (ru) 2019-09-06
EP3331570A1 (en) 2018-06-13

Similar Documents

Publication Publication Date Title
KR20180034467A (ko) L-dopa의 전신 합성 및 조절
JP7601916B2 (ja) 蝸牛および前庭細胞に核酸を送達するための物質および方法
AU2016232146B2 (en) Optimized liver-specific expression systems for FVIII and FIX
KR102729768B1 (ko) 트랜스제닉 선택 방법 및 조성물
CN111344395B (zh) 产生经修饰的自然杀伤细胞的方法及使用方法
AU774643B2 (en) Compositions and methods for use in recombinational cloning of nucleic acids
AU2019261438B2 (en) Expression of FOXP3 in edited CD34+ cells
AU2016229000B2 (en) Recombinant Glut1 adeno-associated viral vector constructs and related methods for restoring Glut1 expression
KR102745604B1 (ko) Glp-1 및 대사성 질환 치료용 조성물에서 이의 용도
CN108753824B (zh) 用于治疗视网膜营养不良的病毒载体
CN112313334A (zh) 用以编辑血红蛋白相关突变的同源定向修复模板设计和递送
KR102628872B1 (ko) 세포의 증식을 제어하기 위해 세포 분열 좌위를 사용하기 위한 도구 및 방법
KR20180097631A (ko) 핵산을 와우 및 전정 세포에 전달하기 위한 물질 및 방법
AU2016343979A1 (en) Delivery of central nervous system targeting polynucleotides
AU2017277647B2 (en) Engineered viral vector reduces induction of inflammatory and immune responses
KR20210151785A (ko) 비바이러스성 dna 벡터 및 fviii 치료제 발현을 위한 이의 용도
AU2018254529B2 (en) Therapeutic genome editing in Wiskott-Aldrich syndrome and X-linked thrombocytopenia
KR20240022575A (ko) 아머링된 키메라 수용체 및 이의 사용 방법
KR20220023962A (ko) 파브리병을 치료하기 위한 조성물, 디바이스 및 방법
CN115298307A (zh) 核酸调节元件的新组合及其方法和用途
KR102712198B1 (ko) 재조합 발생이 최소화된 유전자치료 벡터, 상기 벡터를 포함하는 재조합 레트로바이러스 및 상기 재조합 레트로바이러스를 포함하는 암의 예방 또는 치료용 약학 조성물
RU2812852C2 (ru) Невирусные днк-векторы и варианты их применения для экспрессии терапевтического средства на основе фактора viii (fviii)
HK40082332A (zh) 使用细胞分裂基因座控制细胞增殖的工具和方法
HK40044016A (en) Expression of foxp3 in edited cd34+ cells
HK40024481A (en) Therapeutic genome editing in wiskott-aldrich syndrome and x-linked thrombocytopenia

Legal Events

Date Code Title Description
PA0105 International application

Patent event date: 20180202

Patent event code: PA01051R01D

Comment text: International Patent Application

PG1501 Laying open of application
PC1203 Withdrawal of no request for examination