JP2023526310A - 導入遺伝子発現のｄｒｇ特異的低減のための組成物 - Google Patents

導入遺伝子発現のｄｒｇ特異的低減のための組成物 Download PDF

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JP2023526310A
JP2023526310A JP2022569265A JP2022569265A JP2023526310A JP 2023526310 A JP2023526310 A JP 2023526310A JP 2022569265 A JP2022569265 A JP 2022569265A JP 2022569265 A JP2022569265 A JP 2022569265A JP 2023526310 A JP2023526310 A JP 2023526310A
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target sequences
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ウイルソン，ジェームス・エム
オルドー，ジュリエット
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ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア
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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
    • A61K9/0019—Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
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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
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    • 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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  • Health & Medical Sciences (AREA)
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  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
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  • Coloring Foods And Improving Nutritive Qualities (AREA)
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JP2022569265A 2020-05-12 2021-05-12 導入遺伝子発現のｄｒｇ特異的低減のための組成物 Pending JP2023526310A (ja)

Applications Claiming Priority (11)

Application Number Priority Date Filing Date Title
US202063023593P 2020-05-12 2020-05-12
US63/023,593 2020-05-12
US202063038488P 2020-06-12 2020-06-12
US63/038,488 2020-06-12
US202063043562P 2020-06-24 2020-06-24
US63/043,562 2020-06-24
US202063079299P 2020-09-16 2020-09-16
US63/079,299 2020-09-16
US202163152042P 2021-02-22 2021-02-22
US63/152,042 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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JP2023526310A true JP2023526310A (ja) 2023-06-21
JP2023526310A5 JP2023526310A5 (enExample) 2024-05-22

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CN (1) CN115803064A (enExample)
AU (1) AU2021270447A1 (enExample)
BR (1) BR112022022212A2 (enExample)
CA (1) CA3177407A1 (enExample)
CO (1) CO2022017959A2 (enExample)
IL (1) IL298138A (enExample)
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EP4409010A1 (en) 2021-10-02 2024-08-07 The Trustees of The University of Pennsylvania Novel aav capsids and compositions containing same
WO2023133574A1 (en) 2022-01-10 2023-07-13 The Trustees Of The University Of Pennsylvania Compositions and methods useful for treatment of c9orf72-mediated disorders
WO2023196892A1 (en) 2022-04-06 2023-10-12 The Trustees Of The University Of Pennsylvania Passive immunization with anti- aav neutralizing antibodies to prevent off-target transduction of intrathecally delivered aav vectors
EP4504797A1 (en) 2022-04-06 2025-02-12 The Trustees of The University of Pennsylvania Compositions and methods for treating her2 positive metastatic breast cancer and other cancers
AU2023303882A1 (en) * 2022-07-08 2025-01-23 Sensorion REGULATORY SEQUENCES COMPRISING MicroRNA TARGET SITES
US20260021205A1 (en) 2022-07-15 2026-01-22 The Trustees Of The University Of Pennsylvania Recombinant aav having aav clade d and clade e capsids and compositions containing same
TW202516019A (zh) 2023-06-29 2025-04-16 賓州大學委員會 具中樞神經系統靶向模體的突變aav及含有其之組成物
IL326390A (en) 2023-08-10 2026-04-01 Univ Pennsylvania Compositions and methods for treating spinal muscular atrophy

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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
CN115803064A (zh) 2023-03-14
CA3177407A1 (en) 2021-11-18
US20230304034A1 (en) 2023-09-28
BR112022022212A2 (pt) 2022-12-13
MX2022014258A (es) 2023-02-22

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