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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expression
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JPWO2021231579A5 (https=
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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
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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
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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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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 (https=) 2024-05-22
JPWO2021231579A5 JPWO2021231579A5 (https=) 2024-05-22

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EP (1) EP4162059A1 (https=)
JP (1) JP2023526310A (https=)
KR (1) KR20230010670A (https=)
CN (1) CN115803064A (https=)
AU (1) AU2021270447A1 (https=)
BR (1) BR112022022212A2 (https=)
CA (1) CA3177407A1 (https=)
CO (1) CO2022017959A2 (https=)
IL (1) IL298138A (https=)
MX (1) MX2022014258A (https=)
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AR125406A1 (es) 2021-04-23 2023-07-12 Univ Pennsylvania Nuevas composiciones con motivos selectivos para el cerebro y composiciones que las contienen
EP4409010A1 (en) 2021-10-02 2024-08-07 The Trustees of The University of Pennsylvania Novel aav capsids and compositions containing same
AR128239A1 (es) 2022-01-10 2024-04-10 Univ Pennsylvania Composiciones y métodos útiles para el tratamiento de trastornos mediados por c9orf72
JP2025512969A (ja) 2022-04-06 2025-04-22 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 Ｈｅｒ２陽性転移性乳がん及び他のがんを治療するための組成物及び方法
US20250250326A1 (en) 2022-04-06 2025-08-07 The Trustees Of The University Of Pennsylvania Passive immunization with anti-aav neutralizing antibodies to prevent off-target transduction of intrathecally delivered aav vectors
MA71383A (fr) * 2022-07-08 2025-04-30 Sensorion Séquences régulatrices comprenant des sites cibles de micro-arn
WO2024015966A2 (en) 2022-07-15 2024-01-18 The Trustees Of The University Of Pennsylvania Recombinant aav having aav clade d and clade e capsids and compositions containing same
AR133098A1 (es) 2023-06-29 2025-08-27 Univ Pennsylvania Aav mutante con motivos dirigidos al sistema nervioso central y composiciones que lo contienen
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
WO2021231579A1 (en) 2021-11-18
CO2022017959A2 (es) 2023-03-07
EP4162059A1 (en) 2023-04-12
CN115803064A (zh) 2023-03-14
AU2021270447A1 (en) 2023-01-05
IL298138A (en) 2023-01-01
CA3177407A1 (en) 2021-11-18
MX2022014258A (es) 2023-02-22
BR112022022212A2 (pt) 2022-12-13
US20230304034A1 (en) 2023-09-28

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