PT1880008E - Expressão de proteína mitocondrial através de uma abordagem alotópica melhorada - Google Patents

Expressão de proteína mitocondrial através de uma abordagem alotópica melhorada

Info

Publication number
PT1880008E
PT1880008E PT67619692T PT06761969T PT1880008E PT 1880008 E PT1880008 E PT 1880008E PT 67619692 T PT67619692 T PT 67619692T PT 06761969 T PT06761969 T PT 06761969T PT 1880008 E PT1880008 E PT 1880008E
Authority
PT
Portugal
Prior art keywords
enhanced
expression
mitochondrial protein
allotopic
approach
Prior art date
Application number
PT67619692T
Other languages
English (en)
Inventor
Marisol Corral-Debrinski
José-Alain Sahel
Valérie Kaltimbacher
Crystel Bonnet
Original Assignee
Inserm Inst Nat De La Santé Et De La Rech Médicale
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 Inserm Inst Nat De La Santé Et De La Rech Médicale filed Critical Inserm Inst Nat De La Santé Et De La Rech Médicale
Publication of PT1880008E publication Critical patent/PT1880008E/pt

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Classifications

    • 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
    • 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
    • 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
    • A61K48/0066—Manipulation of the nucleic acid to modify its expression pattern, e.g. enhance its duration of expression, achieved by the presence of particular introns in the delivered nucleic acid
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • 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
    • C12N15/8509—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells for producing genetically modified animals, e.g. transgenic
    • 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.)
    • C12N9/0053—Oxidoreductases (1.) acting on a heme group of donors (1.9)
    • 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.)
    • C12N9/0089—Oxidoreductases (1.) acting on superoxide as acceptor (1.15)
    • 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
    • C12N9/10—Transferases (2.)
    • C12N9/1085—Transferases (2.) transferring alkyl or aryl groups other than methyl groups (2.5)
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y109/00—Oxidoreductases acting on a heme group of donors (1.9)
    • C12Y109/03—Oxidoreductases acting on a heme group of donors (1.9) with oxygen as acceptor (1.9.3)
    • C12Y109/03001—Cytochrome-c oxidase (1.9.3.1)
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/01—Fusion polypeptide containing a localisation/targetting motif
    • C07K2319/07—Fusion polypeptide containing a localisation/targetting motif containing a mitochondrial localisation signal
    • 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
    • C12N2800/00—Nucleic acids vectors
    • C12N2800/22—Vectors comprising a coding region that has been codon optimised for expression in a respective host
    • 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
    • C12N2810/00—Vectors comprising a targeting moiety
    • C12N2810/50—Vectors comprising as targeting moiety peptide derived from defined protein
    • C12N2810/80—Vectors comprising as targeting moiety peptide derived from defined protein from vertebrates

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • Zoology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biotechnology (AREA)
  • Molecular Biology (AREA)
  • Biochemistry (AREA)
  • Biomedical Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Medicinal Chemistry (AREA)
  • Microbiology (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Epidemiology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Toxicology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (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)
  • Peptides Or Proteins (AREA)
PT67619692T 2005-05-03 2006-05-03 Expressão de proteína mitocondrial através de uma abordagem alotópica melhorada PT1880008E (pt)

Applications Claiming Priority (1)

Application Number Priority Date Filing Date Title
US67693305P 2005-05-03 2005-05-03

Publications (1)

Publication Number Publication Date
PT1880008E true PT1880008E (pt) 2015-08-27

Family

ID=37308339

Family Applications (1)

Application Number Title Priority Date Filing Date
PT67619692T PT1880008E (pt) 2005-05-03 2006-05-03 Expressão de proteína mitocondrial através de uma abordagem alotópica melhorada

Country Status (7)

Country Link
US (10) US9017999B2 (pt)
EP (2) EP2913403A1 (pt)
DK (1) DK1880008T3 (pt)
ES (1) ES2541771T3 (pt)
PL (1) PL1880008T3 (pt)
PT (1) PT1880008E (pt)
WO (1) WO2006117250A2 (pt)

Families Citing this family (22)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
ES2541771T3 (es) 2005-05-03 2015-07-24 INSERM (Institut National de la Santé et de la Recherche Médicale) Expresión de una proteína mitocondrial mediante un enfoque alotópico mejorado
PL2528621T3 (pl) 2010-01-27 2017-07-31 Glaxosmithkline Biologicals S.A. Zmodyfikowane antygeny gruźlicy
CN102634527B (zh) * 2012-04-11 2013-11-06 华中科技大学同济医学院附属同济医院 重组人nadh脱氢酶亚单位4基因及其表达载体构建方法
CN103571846B (zh) * 2012-07-18 2017-07-18 中国人民解放军总医院 Atp6v1b2基因突变体及其应用
EP2712630B1 (de) 2012-09-26 2015-12-09 Universität Leipzig Mitochondrialer Expressionsvektor und Verfahren zur Transformation von Mitochondrien
US10287331B2 (en) 2013-04-15 2019-05-14 Yissum Research Development Company Of The Hebrew University Of Jerusalem Ltd. Mitochondrial proteins constructs and uses thereof
ITRM20130538A1 (it) * 2013-10-03 2015-04-04 Aldo Mancini Esapeptide e usi di esso
US11814640B2 (en) 2015-11-26 2023-11-14 Luca Science Inc. Recombinant expression vector and lipid membrane structure having said vector encapsulated therein
TW202000240A (zh) 2018-02-23 2020-01-01 日商盧卡科學股份有限公司 用於在粒線體內使蛋白質表現之核酸、封入前述核酸之脂質膜結構體及其等之用途
CN108640970B (zh) * 2018-03-29 2021-07-23 苏州大学附属第一医院 靶向异位atp5b通路的多肽及其应用
WO2019241206A1 (en) * 2018-06-11 2019-12-19 Gensight Biologics Sa Recombinant aav vectors and methods of using the same
CN111073899B (zh) * 2018-10-19 2021-01-01 武汉纽福斯生物科技有限公司 一种编码人nadh脱氢酶亚单位4蛋白的核酸及其应用
EP3814492A4 (en) * 2018-06-29 2022-02-23 Wuhan Neurophth Biotechnology Limited Company COMPOSITIONS AND METHODS OF TREATING LEBER'S HEREDITARY OPTIC NEUROPATHY
CN113476484A (zh) * 2018-06-29 2021-10-08 武汉纽福斯生物科技有限公司 治疗遗传性视神经病变的组合物和方法
WO2020001657A1 (en) * 2018-06-29 2020-01-02 Wuhan Neurophth Biological Technology Limited Company Compositions and methods for treating leber's hereditary optic neuropathy
CN110699367B (zh) * 2018-07-09 2021-06-11 武汉纽福斯生物科技有限公司 编码人nadh脱氢酶亚单位4蛋白的核酸及其应用
CA3109432A1 (en) * 2018-08-20 2020-02-27 Wuhan Neurophth Biotechnology Limited Company Compositions and methods for treating leber's hereditary optic neuropathy
CN110857440B (zh) * 2018-08-23 2021-02-19 武汉纽福斯生物科技有限公司 重组人ⅱ型线粒体动力蛋白样gtp酶基因序列及其应用
CN109207520B (zh) * 2018-09-25 2022-05-10 北京锦篮基因科技有限公司 Leber遗传性视神经病变的基因药物
EP3942029A1 (en) * 2019-03-21 2022-01-26 Cambridge Enterprise Limited Methods of optimising expression and delivery of mitochondrial proteins
CN113025633A (zh) * 2019-12-09 2021-06-25 武汉纽福斯生物科技有限公司 编码人nadh脱氢酶亚单位1蛋白的核酸及其应用
CN111072011B (zh) * 2020-01-15 2021-05-28 郑州大学 一种线粒体-核仁可逆迁移荧光碳点的制备及在监测细胞活性中的应用

Family Cites Families (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US7488485B2 (en) * 2003-09-16 2009-02-10 University Of Kansas Medical Center DNA vaccine compositions and methods of use
US8021875B2 (en) * 2001-08-27 2011-09-20 Roche Madison Inc. Methods for expression of transgenes
US20040072774A1 (en) * 2002-02-23 2004-04-15 Giovanni Manfredi Methods for expressing and targeting mitochondrial-DNA-encoded peptides and uses thereof
ES2541771T3 (es) 2005-05-03 2015-07-24 INSERM (Institut National de la Santé et de la Recherche Médicale) Expresión de una proteína mitocondrial mediante un enfoque alotópico mejorado

Also Published As

Publication number Publication date
US20230220414A1 (en) 2023-07-13
EP1880008B1 (en) 2015-04-08
US11702671B2 (en) 2023-07-18
US20150087054A1 (en) 2015-03-26
US20200277623A1 (en) 2020-09-03
US20140377869A1 (en) 2014-12-25
ES2541771T3 (es) 2015-07-24
US20210292785A1 (en) 2021-09-23
US9017999B2 (en) 2015-04-28
US20220389446A1 (en) 2022-12-08
US20240093229A1 (en) 2024-03-21
PL1880008T3 (pl) 2015-10-30
US20090306188A1 (en) 2009-12-10
DK1880008T3 (en) 2015-07-13
WO2006117250A2 (en) 2006-11-09
WO2006117250A3 (en) 2007-05-03
EP1880008A2 (en) 2008-01-23
EP2913403A1 (en) 2015-09-02
US20180355372A1 (en) 2018-12-13
US20150225740A1 (en) 2015-08-13

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