ES2895263T3 - Un ácido nucleico que comprende la secuencia de genes IDS y una repetición en tándem de la secuencia de genes de apolipoproteína E - Google Patents

Un ácido nucleico que comprende la secuencia de genes IDS y una repetición en tándem de la secuencia de genes de apolipoproteína E Download PDF

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Publication number
ES2895263T3
ES2895263T3 ES18705462T ES18705462T ES2895263T3 ES 2895263 T3 ES2895263 T3 ES 2895263T3 ES 18705462 T ES18705462 T ES 18705462T ES 18705462 T ES18705462 T ES 18705462T ES 2895263 T3 ES2895263 T3 ES 2895263T3
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ids
sequence
apoeii
nucleic acid
mps
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ES18705462T
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English (en)
Spanish (es)
Inventor
Brian Bigger
Hélène Gleitz
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University of Manchester
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University of Manchester
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    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K35/00—Medicinal preparations containing materials or reaction products thereof with undetermined constitution
    • A61K35/12—Materials from mammals; Compositions comprising non-specified tissues or cells; Compositions comprising non-embryonic stem cells; Genetically modified cells
    • A61K35/28—Bone marrow; Haematopoietic stem cells; Mesenchymal stem cells of any origin, e.g. adipose-derived stem cells
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P5/00—Drugs for disorders of the endocrine system
    • 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
    • C07K14/775—Apolipopeptides
    • 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
    • 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
    • C12N5/00—Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
    • C12N5/10—Cells modified by introduction of foreign genetic material
    • C12N5/12—Fused cells, e.g. hybridomas
    • C12N5/16—Animal cells
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
    • C07H21/00—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
    • C07H21/04—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids with deoxyribosyl as saccharide radical
    • 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
    • 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
    • C12N2510/00—Genetically modified cells

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Zoology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biomedical Technology (AREA)
  • Biotechnology (AREA)
  • Medicinal Chemistry (AREA)
  • Cell Biology (AREA)
  • Immunology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Wood Science & Technology (AREA)
  • Developmental Biology & Embryology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biochemistry (AREA)
  • Epidemiology (AREA)
  • Biophysics (AREA)
  • Microbiology (AREA)
  • Molecular Biology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Virology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Hematology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Physics & Mathematics (AREA)
  • Toxicology (AREA)
  • Plant Pathology (AREA)
  • Diabetes (AREA)
  • Endocrinology (AREA)
  • Enzymes And Modification Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
ES18705462T 2017-02-07 2018-02-07 Un ácido nucleico que comprende la secuencia de genes IDS y una repetición en tándem de la secuencia de genes de apolipoproteína E Active ES2895263T3 (es)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
GBGB1701968.8A GB201701968D0 (en) 2017-02-07 2017-02-07 Gene therapy
PCT/GB2018/050347 WO2018146473A1 (en) 2017-02-07 2018-02-07 Gene therapy

Publications (1)

Publication Number Publication Date
ES2895263T3 true ES2895263T3 (es) 2022-02-18

Family

ID=58462471

Family Applications (2)

Application Number Title Priority Date Filing Date
ES18705462T Active ES2895263T3 (es) 2017-02-07 2018-02-07 Un ácido nucleico que comprende la secuencia de genes IDS y una repetición en tándem de la secuencia de genes de apolipoproteína E
ES21187825T Active ES3035262T3 (en) 2017-02-07 2018-02-07 A nucleic acid comprising ids gene sequence and a tandem repeat of apolipoprotein e gene sequence

Family Applications After (1)

Application Number Title Priority Date Filing Date
ES21187825T Active ES3035262T3 (en) 2017-02-07 2018-02-07 A nucleic acid comprising ids gene sequence and a tandem repeat of apolipoprotein e gene sequence

Country Status (6)

Country Link
US (2) US11701390B2 (enExample)
EP (2) EP3960188B1 (enExample)
JP (1) JP7237364B2 (enExample)
ES (2) ES2895263T3 (enExample)
GB (1) GB201701968D0 (enExample)
WO (1) WO2018146473A1 (enExample)

Families Citing this family (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
BR112020005271A2 (pt) 2017-10-02 2020-09-15 Denali Therapeutics Inc. proteína, polipeptídeo, polinucleotídeo, vetor, célula hospedeira, método para produção de um polipeptídeo, métodos de tratamento de um distúrbio, de diminuição do acúmulo, de monitoramento, de transporte de um agente e de tratamento de um lsd e composição farmacêutica
KR20250110842A (ko) 2022-11-01 2025-07-21 에라스무스 유니버시티 메디컬 센터 로테르담 대사질환을 위한 유전자 요법 구성체

Family Cites Families (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2004108071A2 (en) 2003-06-05 2004-12-16 Salk Institute For Biological Studies Targeting polypeptides to the central nervous system
JP2007513188A (ja) 2003-12-04 2007-05-24 リージェンツ オブ ザ ユニバーシティ オブ ミネソタ リソソーム蓄積症の処置のための組成物および方法
US10227387B2 (en) 2011-05-18 2019-03-12 Children's Hospital Medical Center Targeted delivery of proteins across the blood-brain barrier
RU2014126482A (ru) 2011-12-01 2016-01-27 Ангиочем Инк. Нацеленные соединения ферментов и их применение
EP3218000A2 (en) * 2014-11-10 2017-09-20 Alexion Pharmaceuticals, Inc. Therapeutic compositions of alpha-l-iduronidase, iduronate-2-sulfatase, and alpha-galactosidase a and methods of use thereof

Also Published As

Publication number Publication date
EP3579849B1 (en) 2021-09-08
EP3960188B1 (en) 2025-04-02
EP3960188A1 (en) 2022-03-02
WO2018146473A1 (en) 2018-08-16
JP2020506714A (ja) 2020-03-05
ES3035262T3 (en) 2025-09-01
US20200000854A1 (en) 2020-01-02
EP3579849A1 (en) 2019-12-18
US20230390340A1 (en) 2023-12-07
GB201701968D0 (en) 2017-03-22
JP7237364B2 (ja) 2023-03-13
US11701390B2 (en) 2023-07-18

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