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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CN
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 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)
  • Pharmacology & Pharmacy (AREA)
  • Medicinal Chemistry (AREA)
  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (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)
  • Communicable Diseases (AREA)
  • Obesity (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Neurology (AREA)
  • Hematology (AREA)
  • Neurosurgery (AREA)
  • Virology (AREA)
  • Microbiology (AREA)
  • Oncology (AREA)
  • Diabetes (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 (cg-RX-API-DMAC7.html)
EP (1) EP3468594A4 (cg-RX-API-DMAC7.html)
JP (1) JP2019517281A (cg-RX-API-DMAC7.html)
CN (1) CN109475619A (cg-RX-API-DMAC7.html)
AU (1) AU2017283474A1 (cg-RX-API-DMAC7.html)
CA (1) CA3058735A1 (cg-RX-API-DMAC7.html)
WO (1) WO2017218519A1 (cg-RX-API-DMAC7.html)

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
CN105939767B (zh) 2014-01-08 2018-04-06 弗洛设计声能学公司 具有双声电泳腔的声电泳装置
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
KR102673203B1 (ko) 2016-04-20 2024-06-10 센트로 데 인베스띠가씨오네스 에너제티까스 메디오암비엔딸레스 와이 테크놀로지까스, 오.에이., 엠.피. Pklr의 유전자 발현 증강을 위한 조성물 및 방법
US11214789B2 (en) 2016-05-03 2022-01-04 Flodesign Sonics, Inc. Concentration and washing of particles with acoustics
AU2018352583B2 (en) 2017-10-16 2025-04-17 Centro de Investigaciones Energeticas, Medioambientales Y Tecnologicas, O.A., M.P. Lentiviral vectors for delivery of PKLR to treat Pyruvate Kinase Deficiency
CN114900773A (zh) 2017-12-14 2022-08-12 弗洛设计声能学公司 声泳系统及其操作方法、控制声换能器及声学系统的方法
US12539316B2 (en) 2018-04-11 2026-02-03 Spacecraft Seven, Llc Compositions and methods for stem cell transplant
IL280465B2 (en) * 2018-07-30 2025-03-01 Centro De Investig Energeticas Medioambientales Y Tecnologicas O A M P Methods for gene modification of hematopoietic cells
EA202192328A1 (ru) * 2019-03-22 2022-02-21 Раш Юниверсити Медикал Сентер Комбинация назальной доставки генов и пероральной доставки коричной кислоты, олеамида или гемфиброзила для лечения лизосомных болезней накопления

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

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