CN115803064A - 用于drg特异性降低转基因表达的组合物 - Google Patents

用于drg特异性降低转基因表达的组合物 Download PDF

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CN115803064A
CN115803064A CN202180049076.1A CN202180049076A CN115803064A CN 115803064 A CN115803064 A CN 115803064A CN 202180049076 A CN202180049076 A CN 202180049076A CN 115803064 A CN115803064 A CN 115803064A
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target sequences
sequence
vector
expression
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J·M·威尔逊
J·豪多
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University of Pennsylvania Penn
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University of Pennsylvania Penn
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    • 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
    • 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
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/0012—Galenical forms characterised by the site of application
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    • 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
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • C—CHEMISTRY; METALLURGY
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/11—Antisense
    • C12N2310/113—Antisense targeting other non-coding nucleic acids, e.g. antagomirs
    • 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
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    • C12N2330/00—Production
    • C12N2330/50—Biochemical production, i.e. in a transformed host cell
    • C12N2330/51—Specially adapted vectors
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    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector

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  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • General Preparation And Processing Of Foods (AREA)
  • Coloring Foods And Improving Nutritive Qualities (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
CN202180049076.1A 2020-05-12 2021-05-12 用于drg特异性降低转基因表达的组合物 Pending CN115803064A (zh)

Applications Claiming Priority (11)

Application Number Priority Date Filing Date Title
US202063023593P 2020-05-12 2020-05-12
US63/023593 2020-05-12
US202063038488P 2020-06-12 2020-06-12
US63/038488 2020-06-12
US202063043562P 2020-06-24 2020-06-24
US63/043562 2020-06-24
US202063079299P 2020-09-16 2020-09-16
US63/079299 2020-09-16
US202163152042P 2021-02-22 2021-02-22
US63/152042 2021-02-22
PCT/US2021/032003 WO2021231579A1 (en) 2020-05-12 2021-05-12 Compositions for drg-specific reduction of transgene expression

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Publication Number Publication Date
CN115803064A true CN115803064A (zh) 2023-03-14

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CN202180049076.1A Pending CN115803064A (zh) 2020-05-12 2021-05-12 用于drg特异性降低转基因表达的组合物

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US (1) US20230304034A1 (enExample)
EP (1) EP4162059A1 (enExample)
JP (1) JP2023526310A (enExample)
KR (1) KR20230010670A (enExample)
CN (1) CN115803064A (enExample)
AU (1) AU2021270447A1 (enExample)
BR (1) BR112022022212A2 (enExample)
CA (1) CA3177407A1 (enExample)
CO (1) CO2022017959A2 (enExample)
IL (1) IL298138A (enExample)
MX (1) MX2022014258A (enExample)
WO (1) WO2021231579A1 (enExample)

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KR20230010670A (ko) 2023-01-19
IL298138A (en) 2023-01-01
WO2021231579A1 (en) 2021-11-18
EP4162059A1 (en) 2023-04-12
CO2022017959A2 (es) 2023-03-07
AU2021270447A1 (en) 2023-01-05
JP2023526310A (ja) 2023-06-21
CA3177407A1 (en) 2021-11-18
US20230304034A1 (en) 2023-09-28
BR112022022212A2 (pt) 2022-12-13
MX2022014258A (es) 2023-02-22

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