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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China
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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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    • C12N15/00—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
    • 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
    • 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
    • A61K48/0058—Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
    • A—HUMAN NECESSITIES
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    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
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    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
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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
    • A01K2217/00—Genetically modified animals
    • A01K2217/07—Animals genetically altered by homologous recombination
    • A01K2217/075—Animals genetically altered by homologous recombination inducing loss of function, i.e. knock out
    • 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
    • A01K2227/00—Animals characterised by species
    • A01K2227/10—Mammal
    • A01K2227/105—Murine
    • 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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    • C12N2750/00011—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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    • C12N2800/00—Nucleic acids vectors
    • C12N2800/10—Plasmid DNA
    • C12N2800/106—Plasmid DNA for vertebrates
    • C12N2800/107—Plasmid DNA for vertebrates for mammalian
    • 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
    • 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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  • Health & Medical Sciences (AREA)
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  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
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  • Bioinformatics & Cheminformatics (AREA)
  • Biomedical Technology (AREA)
  • General Health & Medical Sciences (AREA)
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  • Medicinal Chemistry (AREA)
  • Molecular Biology (AREA)
  • General Engineering & Computer Science (AREA)
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  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Biochemistry (AREA)
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  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
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  • Neurology (AREA)
  • Neurosurgery (AREA)
  • Virology (AREA)
  • 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)
  • Enzymes And Modification Thereof (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (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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CN114127296A CN114127296A (zh) 2022-03-01
CN114127296B true CN114127296B (zh) 2024-10-25

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

Families Citing this family (5)

* Cited by examiner, † Cited by third party
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MX2023006445A (es) * 2020-12-01 2023-08-10 Univ Pennsylvania Composiciones y usos de estas para el tratamiento del síndrome de angelman.
CN120265299A (zh) * 2022-06-07 2025-07-04 吉乔公司 用于调节表达的剂
TW202434734A (zh) * 2023-02-08 2024-09-01 美商銀杏生物工作室有限公司 天使症候群的基因療法
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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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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CA2347129A1 (en) 1998-09-22 2000-03-30 University Of Florida Methods for large-scale production of recombinant aav vectors
EP1127150B1 (en) 1998-11-05 2007-05-16 The Trustees Of The University Of Pennsylvania Adeno-associated virus serotype 1 nucleic acid sequences, vectors and host cells containing same
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CA2410901C (en) 2000-06-01 2012-03-20 University Of North Carolina At Chapel Hill Methods and compounds for controlled release of recombinant parvovirus vectors
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RU2019143627A (ru) * 2017-06-28 2021-07-28 Юниверсити Оф Соус Флорида Модифицированный ген ube3a для генной терапии синдрома ангельмана
JP2022525564A (ja) * 2019-03-21 2022-05-17 ピーティーシー セラピューティクス， インコーポレイテッド アンジェルマン症候群を治療するためのベクターおよび方法
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Publication number Priority date Publication date Assignee Title
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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WO2020237130A1 (en) 2020-11-26
CA3139678A1 (en) 2020-11-26
US12465655B2 (en) 2025-11-11
MX2021014248A (es) 2022-01-06
JP2022533448A (ja) 2022-07-22
SG11202112470YA (en) 2021-12-30
AU2020279387A1 (en) 2021-12-09
EP3973059A4 (en) 2023-10-25
US20260102519A1 (en) 2026-04-16
EA202193214A1 (ru) 2022-03-14
IL287943A (en) 2022-01-01
EP3973059A1 (en) 2022-03-30
JP7616668B2 (ja) 2025-01-17
KR20210158859A (ko) 2021-12-31
CN114127296A (zh) 2022-03-01
US20220241434A1 (en) 2022-08-04

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