CN109475619A - 神经元蜡样脂褐质沉积症的基因治疗 - Google Patents

神经元蜡样脂褐质沉积症的基因治疗 Download PDF

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Publication number
CN109475619A
CN109475619A CN201780043899.7A CN201780043899A CN109475619A CN 109475619 A CN109475619 A CN 109475619A CN 201780043899 A CN201780043899 A CN 201780043899A CN 109475619 A CN109475619 A CN 109475619A
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China
Prior art keywords
cell
polynucleotide
promoter
virus
tpp1
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Pending
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CN201780043899.7A
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English (en)
Chinese (zh)
Inventor
肯德里克·A·戈斯
杰弗里·B·帕森斯
阿西娅·吉尼亚图林
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Bluebird Bio Inc
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Bluebird Bio Inc
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Publication date
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Publication of CN109475619A publication Critical patent/CN109475619A/zh
Pending legal-status Critical Current

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    • 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
    • A61P3/00—Drugs for disorders of the metabolism
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P31/00—Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
    • 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
    • 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
    • C12N2740/00—Reverse transcribing RNA viruses
    • C12N2740/00011—Details
    • C12N2740/10011—Retroviridae
    • C12N2740/16011—Human Immunodeficiency Virus, HIV
    • C12N2740/16041—Use of virus, viral particle or viral elements as a vector
    • C12N2740/16043—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Biomedical Technology (AREA)
  • Biotechnology (AREA)
  • Zoology (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Biochemistry (AREA)
  • Molecular Biology (AREA)
  • Hematology (AREA)
  • Diabetes (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Communicable Diseases (AREA)
  • Neurosurgery (AREA)
  • Oncology (AREA)
  • Virology (AREA)
  • Microbiology (AREA)
  • Obesity (AREA)
  • Neurology (AREA)
  • Plant Pathology (AREA)
  • Epidemiology (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
CN201780043899.7A 2016-06-13 2017-06-13 神经元蜡样脂褐质沉积症的基因治疗 Pending CN109475619A (zh)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201662349505P 2016-06-13 2016-06-13
US62/349,505 2016-06-13
US201762457498P 2017-02-10 2017-02-10
US62/457,498 2017-02-10
PCT/US2017/037230 WO2017218519A1 (en) 2016-06-13 2017-06-13 Gene therapy of neuronal ceroid lipofuscinoses

Publications (1)

Publication Number Publication Date
CN109475619A true CN109475619A (zh) 2019-03-15

Family

ID=60663744

Family Applications (1)

Application Number Title Priority Date Filing Date
CN201780043899.7A Pending CN109475619A (zh) 2016-06-13 2017-06-13 神经元蜡样脂褐质沉积症的基因治疗

Country Status (7)

Country Link
US (1) US20220323612A1 (enExample)
EP (1) EP3468594A4 (enExample)
JP (1) JP2019517281A (enExample)
CN (1) CN109475619A (enExample)
AU (1) AU2017283474A1 (enExample)
CA (1) CA3058735A1 (enExample)
WO (1) WO2017218519A1 (enExample)

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN112522321A (zh) * 2020-12-16 2021-03-19 北京艺妙神州医药科技有限公司 一种基因治疗载体及其用途

Families Citing this family (11)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US10704021B2 (en) 2012-03-15 2020-07-07 Flodesign Sonics, Inc. Acoustic perfusion devices
CA2935960C (en) 2014-01-08 2023-01-10 Bart Lipkens Acoustophoresis device with dual acoustophoretic chamber
US11377651B2 (en) 2016-10-19 2022-07-05 Flodesign Sonics, Inc. Cell therapy processes utilizing acoustophoresis
US11708572B2 (en) 2015-04-29 2023-07-25 Flodesign Sonics, Inc. Acoustic cell separation techniques and processes
CN109641063B (zh) 2016-04-20 2022-11-15 能源环境和技术研究中心O.A., M.P. 用于增强pklr的基因表达的组合物和方法
US11214789B2 (en) 2016-05-03 2022-01-04 Flodesign Sonics, Inc. Concentration and washing of particles with acoustics
SG11202002263TA (en) 2017-10-16 2020-04-29 Centro De Investigaciones Energeticas Medioambientales Y Tecnologicas Lentiviral vectors for delivery of pklr to treat pyruvate kinase deficiency
SG11202003907WA (en) 2017-12-14 2020-05-28 Flodesign Sonics Inc Acoustic transducer drive and controller
JP7576754B2 (ja) 2018-04-11 2024-11-01 スペースクラフト セブン リミテッド ライアビリティ カンパニー 幹細胞移植のための組成物および方法
MX2021001292A (es) * 2018-07-30 2021-07-15 Centro De Investig Energeticas Medioambientales Y Tecnologicas O A M P Metodos para la modificacion genica de celulas hematopoyeticas.
AU2020245415B2 (en) * 2019-03-22 2025-06-26 Rush University Medical Center Combination of nasal gene delivery and oral cinnamic acid, oleamide or gemfibrozil for lysosomal stoarge disorders

Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20130004471A1 (en) * 2011-06-10 2013-01-03 Bluebird Bio, Inc. Gene therapy vectors for adrenoleukodystrophy and adrenomyeloneuropathy
WO2016028879A1 (en) * 2014-08-19 2016-02-25 The Children's Hospital Of Philadelphia Compositions and methods for modulating immune response

Family Cites Families (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2014089449A1 (en) * 2012-12-07 2014-06-12 Rush University Nedical Center Composition and method for treating neuronal ceroid lipofuscinosis

Patent Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20130004471A1 (en) * 2011-06-10 2013-01-03 Bluebird Bio, Inc. Gene therapy vectors for adrenoleukodystrophy and adrenomyeloneuropathy
CN103717240A (zh) * 2011-06-10 2014-04-09 蓝鸟生物公司 用于肾上腺脑白质营养不良症和肾上腺脊髓神经病的基因治疗载体
WO2016028879A1 (en) * 2014-08-19 2016-02-25 The Children's Hospital Of Philadelphia Compositions and methods for modulating immune response

Non-Patent Citations (1)

* Cited by examiner, † Cited by third party
Title
RE HASKELL等: "Viral-mediated delivery of the late-infantile neuronal ceroid lipofuscinosis gene,TPP-I to the mouse central nervous system", 《GENE THERAPY》 *

Cited By (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN112522321A (zh) * 2020-12-16 2021-03-19 北京艺妙神州医药科技有限公司 一种基因治疗载体及其用途
CN112522321B (zh) * 2020-12-16 2022-04-08 北京艺妙神州医药科技有限公司 一种基因治疗载体及其用途

Also Published As

Publication number Publication date
EP3468594A1 (en) 2019-04-17
WO2017218519A1 (en) 2017-12-21
AU2017283474A1 (en) 2019-01-03
CA3058735A1 (en) 2017-12-21
EP3468594A4 (en) 2020-01-22
US20220323612A1 (en) 2022-10-13
JP2019517281A (ja) 2019-06-24

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