CN102596255B - 基因载体 - Google Patents

基因载体 Download PDF

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Publication number
CN102596255B
CN102596255B CN201080030337.7A CN201080030337A CN102596255B CN 102596255 B CN102596255 B CN 102596255B CN 201080030337 A CN201080030337 A CN 201080030337A CN 102596255 B CN102596255 B CN 102596255B
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cells
mir
expression
galc
cell
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Chinese (zh)
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CN102596255A (zh
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A·比菲
B·R·金特纳
L·纳尔迪尼
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San Rafael Hospital Co ltd
Fondazione Telethon
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San Rafael Hospital Co ltd
Fondazione Telethon
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    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P7/00—Drugs for disorders of the blood or the extracellular fluid
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00—Antineoplastic agents
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P37/00—Drugs for immunological or allergic disorders
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P37/00—Drugs for immunological or allergic disorders
    • A61P37/02—Immunomodulators
    • A61P37/04—Immunostimulants
    • 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
    • C07K14/52—Cytokines; Lymphokines; Interferons
    • C07K14/555—Interferons [IFN]
    • C07K14/56—IFN-alpha
    • 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
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/111—General methods applicable to biologically active non-coding nucleic acids
    • 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
    • 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
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
    • C12N2310/141—MicroRNAs, miRNAs
    • 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
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/50—Physical structure
    • C12N2310/51—Physical structure in polymeric form, e.g. multimers, concatemers
    • 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
    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/32—Special delivery means, e.g. tissue-specific
    • 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
    • C12N2330/00—Production
    • C12N2330/50—Biochemical production, i.e. in a transformed host cell
    • C12N2330/51—Specially adapted 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
    • C12N2799/00—Uses of viruses
    • C12N2799/02—Uses of viruses as vector
    • C12N2799/021—Uses of viruses as vector for the expression of a heterologous nucleic acid
    • C12N2799/027—Uses of viruses as vector for the expression of a heterologous nucleic acid where the vector is derived from a retrovirus

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Biomedical Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Health & Medical Sciences (AREA)
  • Zoology (AREA)
  • Biotechnology (AREA)
  • Wood Science & Technology (AREA)
  • Molecular Biology (AREA)
  • General Engineering & Computer Science (AREA)
  • Medicinal Chemistry (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Biochemistry (AREA)
  • Biophysics (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Physics & Mathematics (AREA)
  • Immunology (AREA)
  • Toxicology (AREA)
  • Diabetes (AREA)
  • Hematology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Epidemiology (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (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)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
CN201080030337.7A 2009-04-30 2010-04-30 基因载体 Active CN102596255B (zh)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US17412409P 2009-04-30 2009-04-30
US61/174124 2009-04-30
PCT/IB2010/001166 WO2010125471A2 (en) 2009-04-30 2010-04-30 Gene vector

Publications (2)

Publication Number Publication Date
CN102596255A CN102596255A (zh) 2012-07-18
CN102596255B true CN102596255B (zh) 2017-10-13

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CN201080030337.7A Active CN102596255B (zh) 2009-04-30 2010-04-30 基因载体

Country Status (23)

Country Link
US (4) US10287579B2 (https=)
EP (2) EP2424571B1 (https=)
JP (2) JP6144911B2 (https=)
KR (1) KR101793615B1 (https=)
CN (1) CN102596255B (https=)
AU (1) AU2010243276B2 (https=)
BR (1) BRPI1010873B1 (https=)
CA (1) CA2759438C (https=)
CY (1) CY1123218T1 (https=)
DK (1) DK2424571T3 (https=)
EA (1) EA023938B1 (https=)
ES (1) ES2786039T3 (https=)
HR (1) HRP20200647T1 (https=)
HU (1) HUE049845T2 (https=)
IL (1) IL215804A (https=)
LT (1) LT2424571T (https=)
MX (1) MX2011011508A (https=)
PL (1) PL2424571T3 (https=)
PT (1) PT2424571T (https=)
SG (2) SG10201610827RA (https=)
SI (1) SI2424571T1 (https=)
SM (1) SMT202000224T1 (https=)
WO (1) WO2010125471A2 (https=)

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CA2804791C (en) * 2010-07-08 2019-07-30 Duke University Direct reprogramming of cells to cardiac myocyte fate
GB201122458D0 (en) 2011-12-30 2012-02-08 Univ Wageningen Modified cascade ribonucleoproteins and uses thereof
WO2013152230A1 (en) * 2012-04-04 2013-10-10 The Trustees Of Columbia University In The City Of New York Smooth muscle specific inhibition for anti-restenotic therapy
EP3241902B1 (en) 2012-05-25 2018-02-28 The Regents of The University of California Methods and compositions for rna-directed target dna modification and for rna-directed modulation of transcription
WO2014011901A2 (en) * 2012-07-11 2014-01-16 Sangamo Biosciences, Inc. Methods and compositions for delivery of biologics
EP3363902B1 (en) 2012-12-06 2019-11-27 Sigma Aldrich Co. LLC Crispr-based genome modification and regulation
SI3889173T1 (sl) 2013-02-15 2023-11-30 Bioverativ Therapeutics Inc. Optimiran gen dejavnika VIII
MX374090B (es) 2013-03-14 2025-03-05 Caribou Biosciences Inc Composiciones y métodos de ácidos nucleicos dirigidos al ácido nucleico.
ES2747951T3 (es) 2013-10-24 2020-03-12 Ospedale San Raffaele Srl Método
EP3160478A4 (en) 2014-06-30 2018-05-16 Bioverativ Therapeutics Inc. Optimized factor ix gene
JP6960159B6 (ja) * 2015-04-22 2022-06-24 国立大学法人京都大学 組織細胞の選別方法
US20180327820A1 (en) * 2015-06-15 2018-11-15 Murdoch Childrens Research Institute Method of measuring chimerism
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US10188750B1 (en) 2015-10-23 2019-01-29 University Of South Florida Self-replicating cell selective gene delivery compositions, methods, and uses thereof
WO2017136358A1 (en) 2016-02-01 2017-08-10 Bioverativ Therapeutics Inc. Optimized factor viii genes
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CN110036112B (zh) 2016-10-18 2024-05-10 俄勒冈健康与科学大学 引发受主要组织相容性复合体e分子限制的t细胞的巨细胞病毒载体
CN106566838B (zh) * 2016-11-14 2019-11-01 上海伯豪生物技术有限公司 一种基于CRISPR-Cas9技术的miR-126全长基因敲除试剂盒及其应用
PT3612624T (pt) * 2017-04-21 2024-11-29 Ospedale San Raffaele Srl Terapia génica
IT201700086403A1 (it) * 2017-07-27 2019-01-27 Univ Del Piemonte Orientale Endothelial-specific promoter sequences and uses thereof
TW202545984A (zh) 2017-08-09 2025-12-01 美商生物化學醫療公司 核酸分子及其用途
AU2019215063B2 (en) 2018-02-01 2025-10-16 Bioverativ Therapeutics, Inc. Use of lentiviral vectors expressing Factor VIII
WO2020033863A1 (en) 2018-08-09 2020-02-13 Bioverativ Therapeutics Inc. Nucleic acid molecules and uses thereof for non-viral gene therapy
AU2019393880A1 (en) 2018-12-06 2021-07-15 Bioverativ Therapeutics Inc. Use of lentiviral vectors expressing factor IX
JP7803014B2 (ja) 2019-09-30 2026-01-21 バイオベラティブ セラピューティクス インコーポレイテッド レンチウイルスベクター製剤
AU2020384286A1 (en) * 2019-11-12 2022-06-09 The Regents Of The University Of California Lentiviral vectors in hematopoietic stem cells to treat X-linked chronic granulomatous disease
US12173307B2 (en) 2020-06-24 2024-12-24 Bioverativ Therapeutics Inc. Methods for the purification of viral vectors
US20220090130A1 (en) 2020-08-23 2022-03-24 Bioverativ Therapeutics Inc. MODIFIED BACULOVIRUS SYSTEM FOR IMPROVED PRODUCTION OF CLOSED-ENDED DNA (ceDNA)
CA3229323A1 (en) 2021-08-23 2023-03-02 Ajay MAGHODIA Optimized factor viii genes
AU2022334711A1 (en) 2021-08-23 2024-04-04 Bioverativ Therapeutics Inc. Baculovirus expression system
CN115725656B (zh) * 2021-09-01 2026-01-16 联邦生物科技(珠海横琴)有限公司 一种以细胞质rna病毒为载体递送干扰rna的方法
CA3232988A1 (en) 2021-09-30 2023-04-06 Bioverativ Therapeutics Inc. Nucleic acids encoding factor viii polypeptides with reduced immunogenicity
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WO2025122739A1 (en) * 2023-12-05 2025-06-12 University Of Pittsburgh-Of The Commonwealth System Of Higher Education Compositions and methods for treating a heart disease
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Publication number Publication date
JP2016028582A (ja) 2016-03-03
HRP20200647T1 (hr) 2020-10-02
SMT202000224T1 (it) 2020-05-08
EP2424571A2 (en) 2012-03-07
EP2424571B1 (en) 2020-04-08
HUE049845T2 (hu) 2020-10-28
EA201171335A1 (ru) 2012-05-30
AU2010243276A1 (en) 2011-11-17
SI2424571T1 (sl) 2020-10-30
KR20120038403A (ko) 2012-04-23
EP3733214A1 (en) 2020-11-04
IL215804A0 (en) 2012-01-31
BRPI1010873A2 (pt) 2016-03-15
WO2010125471A2 (en) 2010-11-04
US20190367911A1 (en) 2019-12-05
WO2010125471A3 (en) 2010-12-29
JP6370278B2 (ja) 2018-08-08
CA2759438A1 (en) 2010-11-04
EA023938B1 (ru) 2016-07-29
DK2424571T3 (da) 2020-05-04
CY1123218T1 (el) 2021-10-29
IL215804A (en) 2016-10-31
KR101793615B1 (ko) 2017-11-03
SG10201610827RA (en) 2017-02-27
JP2012525141A (ja) 2012-10-22
LT2424571T (lt) 2020-08-10
CA2759438C (en) 2021-03-30
US10287579B2 (en) 2019-05-14
US20220333103A1 (en) 2022-10-20
BRPI1010873B1 (pt) 2020-04-14
PL2424571T3 (pl) 2020-10-19
SG175839A1 (en) 2011-12-29
ES2786039T3 (es) 2020-10-08
AU2010243276B2 (en) 2016-09-15
MX2011011508A (es) 2012-02-13
US20120128643A1 (en) 2012-05-24
US9951328B2 (en) 2018-04-24
US20180044670A1 (en) 2018-02-15
US11407996B2 (en) 2022-08-09
US12577560B2 (en) 2026-03-17
PT2424571T (pt) 2020-05-06
JP6144911B2 (ja) 2017-06-07
CN102596255A (zh) 2012-07-18

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