CN114127296B - Ube3a基因和表达盒及其应用 - Google Patents

Ube3a基因和表达盒及其应用 Download PDF

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Publication number
CN114127296B
CN114127296B CN202080050918.0A CN202080050918A CN114127296B CN 114127296 B CN114127296 B CN 114127296B CN 202080050918 A CN202080050918 A CN 202080050918A CN 114127296 B CN114127296 B CN 114127296B
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vector
aav
expression cassette
ube3a
polynucleotide
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CN114127296A (zh
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B·D·菲尔波特
S·J·格雷
C·邢
M·贾德森
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University of North Carolina at Chapel Hill
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    • 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
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    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
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    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
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    • A01K2217/07—Animals genetically altered by homologous recombination
    • A01K2217/075—Animals genetically altered by homologous recombination inducing loss of function, i.e. knock out
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    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
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    • A01K2227/10—Mammal
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    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
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    • C12N2750/14011—Parvoviridae
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    • C12N2800/00—Nucleic acids vectors
    • C12N2800/10—Plasmid DNA
    • C12N2800/106—Plasmid DNA for vertebrates
    • C12N2800/107—Plasmid DNA for vertebrates for mammalian
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    • C12N2800/00—Nucleic acids vectors
    • C12N2800/22—Vectors comprising a coding region that has been codon optimised for expression in a respective host

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  • General Engineering & Computer Science (AREA)
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  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Enzymes And Modification Thereof (AREA)
CN202080050918.0A 2019-05-22 2020-05-22 Ube3a基因和表达盒及其应用 Active CN114127296B (zh)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201962851411P 2019-05-22 2019-05-22
US62/851,411 2019-05-22
PCT/US2020/034171 WO2020237130A1 (en) 2019-05-22 2020-05-22 Ube3a genes and expression cassettes and their use

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CN114127296B true CN114127296B (zh) 2024-10-25

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US (2) US12465655B2 (https=)
EP (1) EP3973059A4 (https=)
JP (1) JP7616668B2 (https=)
KR (1) KR20210158859A (https=)
CN (1) CN114127296B (https=)
AU (1) AU2020279387A1 (https=)
CA (1) CA3139678A1 (https=)
EA (1) EA202193214A1 (https=)
IL (1) IL287943A (https=)
MX (1) MX2021014248A (https=)
SG (1) SG11202112470YA (https=)
WO (1) WO2020237130A1 (https=)

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IL303239A (en) * 2020-12-01 2023-07-01 Univ Pennsylvania Compositions and their uses for the treatment of Engelmann syndrome
AU2023285684A1 (en) * 2022-06-07 2025-01-02 Kicho Inc. Agents for modulating expression
EP4661916A1 (en) * 2023-02-08 2025-12-17 Ginkgo Bioworks, Inc. Gene therapy for angelman syndrome
WO2025128816A1 (en) * 2023-12-13 2025-06-19 Kicho Inc. Methods of modulating expression of ube3a gene
WO2025213123A1 (en) * 2024-04-05 2025-10-09 Encoded Therapeutics, Inc. Methods and compositions for increasing expression of ube3a

Citations (2)

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CN105793425A (zh) * 2013-06-17 2016-07-20 布罗德研究所有限公司 使用病毒组分靶向障碍和疾病的crispr-cas系统和组合物的递送、用途和治疗应用
CN107530451A (zh) * 2015-05-07 2018-01-02 南佛罗里达大学 用于天使人综合征的基因治疗方法的修饰的ube3a基因

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US6040183A (en) 1995-06-07 2000-03-21 University Of North Carloina At Chapel Hill Helper virus-free AAV production
US6093570A (en) 1995-06-07 2000-07-25 The University Of North Carolina At Chapel Hill Helper virus-free AAV production
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AU774706B2 (en) 1998-09-22 2004-07-08 Johns Hopkins University, The Methods for large-scale production of recombinant AAV vectors
ATE362542T1 (de) 1998-11-05 2007-06-15 Univ Pennsylvania Nukleinsäuresequenzen des adeno-assoziierten virus des serotyps i, und vektoren und wirtszellen, die diese enthalten
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CN105793425A (zh) * 2013-06-17 2016-07-20 布罗德研究所有限公司 使用病毒组分靶向障碍和疾病的crispr-cas系统和组合物的递送、用途和治疗应用
CN107530451A (zh) * 2015-05-07 2018-01-02 南佛罗里达大学 用于天使人综合征的基因治疗方法的修饰的ube3a基因

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Publication number Publication date
US20220241434A1 (en) 2022-08-04
CN114127296A (zh) 2022-03-01
JP2022533448A (ja) 2022-07-22
EP3973059A1 (en) 2022-03-30
CA3139678A1 (en) 2020-11-26
US20260102519A1 (en) 2026-04-16
WO2020237130A1 (en) 2020-11-26
SG11202112470YA (en) 2021-12-30
MX2021014248A (es) 2022-01-06
EA202193214A1 (ru) 2022-03-14
KR20210158859A (ko) 2021-12-31
US12465655B2 (en) 2025-11-11
IL287943A (en) 2022-01-01
AU2020279387A1 (en) 2021-12-09
EP3973059A4 (en) 2023-10-25
JP7616668B2 (ja) 2025-01-17

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