CN113454232B - 封闭端dna（cedna）产生中对rep蛋白活性的调节 - Google Patents

封闭端dna（cedna）产生中对rep蛋白活性的调节 Download PDF

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CN113454232B
CN113454232B CN202080014548.5A CN202080014548A CN113454232B CN 113454232 B CN113454232 B CN 113454232B CN 202080014548 A CN202080014548 A CN 202080014548A CN 113454232 B CN113454232 B CN 113454232B
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itr
protein
rep
cedna
cell
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CN113454232A (zh
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R·M·科廷
A·尤谢
A·K·玛拉基安
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Generational Biology Co
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    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
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    • C12N2750/14011—Parvoviridae
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    • C12N2810/00—Vectors comprising a targeting moiety
    • C12N2810/50—Vectors comprising as targeting moiety peptide derived from defined protein
    • C12N2810/60—Vectors comprising as targeting moiety peptide derived from defined protein from viruses
    • C12N2810/6009—Vectors comprising as targeting moiety peptide derived from defined protein from viruses dsDNA viruses
    • C12N2810/6018—Adenoviridae
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    • C12N2820/00—Vectors comprising a special origin of replication system
    • C12N2820/60—Vectors comprising a special origin of replication system from viruses

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  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Antibodies Or Antigens For Use As Internal Diagnostic Agents (AREA)
CN202080014548.5A 2019-02-15 2020-02-14 封闭端dna（cedna）产生中对rep蛋白活性的调节 Active CN113454232B (zh)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201962806076P 2019-02-15 2019-02-15
US62/806,076 2019-02-15
PCT/US2020/018332 WO2020168222A1 (en) 2019-02-15 2020-02-14 Modulation of rep protein activity in closed-ended dna (cedna) production

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CN113454232A CN113454232A (zh) 2021-09-28
CN113454232B true CN113454232B (zh) 2024-10-29

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US (1) US20220127625A1 (https=)
EP (1) EP3924491A4 (https=)
JP (2) JP7644014B2 (https=)
KR (1) KR20210127935A (https=)
CN (1) CN113454232B (https=)
AU (1) AU2020221312A1 (https=)
CA (1) CA3129321A1 (https=)
IL (1) IL285415A (https=)
MA (1) MA54958A (https=)
SG (1) SG11202106491VA (https=)
WO (1) WO2020168222A1 (https=)

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NZ762100A (en) 2017-08-09 2025-12-19 Bioverativ Therapeutics Inc Nucleic acid molecules and uses thereof
EP3833766A1 (en) 2018-08-09 2021-06-16 Bioverativ Therapeutics Inc. Nucleic acid molecules and uses thereof for non-viral gene therapy
WO2020251954A1 (en) * 2019-06-10 2020-12-17 Homology Medicines, Inc. Adeno-associated virus compositions for arsa gene transfer and methods of use thereof
JP2023535632A (ja) 2020-07-27 2023-08-18 アンジャリウム バイオサイエンシズ エージー Ｄｎａ分子の組成物、その作製方法、及びその使用方法
EP4214309A4 (en) * 2020-09-16 2025-07-16 Generation Bio Co CLOSED-END DNA VECTORS AND RELATED USES FOR EXPRESSING PHENYLALANINE HYDROXYLASE (PAH)
WO2022136616A1 (en) * 2020-12-23 2022-06-30 Vivet Therapeutics Minimal bile acid inducible promoters for gene therapy
EP4308705A4 (en) * 2021-03-16 2025-06-04 Wisconsin Alumni Research Foundation INSULIN GENE THERAPY TO TREAT DIABETES
MX2024002333A (es) * 2021-08-23 2024-03-07 Bioverativ Therapeutics Inc Sistema de expresion de baculovirus.
CN114703203B (zh) * 2022-02-11 2024-08-06 上海渤因生物科技有限公司 杆状病毒载体及其用途
KR102737146B1 (ko) * 2023-07-10 2024-12-09 대한민국 신규 발현 벡터 및 이의 용도
WO2025114932A1 (en) * 2023-11-30 2025-06-05 Sanofi Purification of closed-ended dna molecules
US12509711B1 (en) * 2024-08-09 2025-12-30 Martin Williams System and process for in vivo manufacturing nanostructure-ended double-stranded covalently-closed linear DNA, the resulting molecules and their uses

Citations (1)

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CN109195636A (zh) * 2016-03-03 2019-01-11 马萨诸塞大学 用于非病毒基因转移的末端封闭型线性双链体dna

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CA2596303C (en) * 1997-03-21 2013-09-24 Enzo Therapeutics, Inc. Vectors and viral vectors and packaging cell lines for propagating same
WO2001032899A1 (en) 1999-10-29 2001-05-10 Takara Shuzo Co., Ltd. Gene transfer method
CN103849629B (zh) * 2006-06-21 2017-06-09 尤尼克尔Ip股份有限公司 具有经修饰的用于在昆虫细胞中产生aav的aav‑rep78翻译起始密码子的载体
EP2545165B1 (en) * 2010-03-11 2020-07-29 uniQure IP B.V. Mutated rep encoding sequences for use in aav production
EP2500434A1 (en) * 2011-03-12 2012-09-19 Association Institut de Myologie Capsid-free AAV vectors, compositions, and methods for vector production and gene delivery
CA2942289C (en) * 2014-03-10 2024-05-21 Uniqure Ip B.V. Further improved aav vectors produced in insect cells
NZ762100A (en) * 2017-08-09 2025-12-19 Bioverativ Therapeutics Inc Nucleic acid molecules and uses thereof

Patent Citations (1)

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Publication number Priority date Publication date Assignee Title
CN109195636A (zh) * 2016-03-03 2019-01-11 马萨诸塞大学 用于非病毒基因转移的末端封闭型线性双链体dna

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CN113454232A (zh) 2021-09-28
EP3924491A4 (en) 2022-12-14
EP3924491A1 (en) 2021-12-22
JP2024083567A (ja) 2024-06-21
IL285415A (en) 2021-09-30
US20220127625A1 (en) 2022-04-28
MA54958A (fr) 2021-12-22
WO2020168222A1 (en) 2020-08-20
AU2020221312A1 (en) 2021-10-07
JP2022520803A (ja) 2022-04-01
KR20210127935A (ko) 2021-10-25
CA3129321A1 (en) 2020-08-20
JP7644014B2 (ja) 2025-03-11
SG11202106491VA (en) 2021-07-29

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