CN113272428A - 核酸构建体和使用方法 - Google Patents

核酸构建体和使用方法 Download PDF

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Publication number
CN113272428A
CN113272428A CN201980078035.8A CN201980078035A CN113272428A CN 113272428 A CN113272428 A CN 113272428A CN 201980078035 A CN201980078035 A CN 201980078035A CN 113272428 A CN113272428 A CN 113272428A
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Prior art keywords
construct
nucleic acid
cell
polypeptide
segment
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Pending
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CN201980078035.8A
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English (en)
Chinese (zh)
Inventor
J·D·芬恩
H-R·黄
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Intelia Therapeutics Co ltd
Regeneron Pharmaceuticals Inc
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Intelia Therapeutics Co ltd
Regeneron Pharmaceuticals Inc
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Application filed by Intelia Therapeutics Co ltd, Regeneron Pharmaceuticals Inc filed Critical Intelia Therapeutics Co ltd
Publication of CN113272428A publication Critical patent/CN113272428A/zh
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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
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
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    • C12N15/90—Stable introduction of foreign DNA into chromosome
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    • A61K48/0008—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 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition
    • A61K48/0025—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 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition wherein the non-active part clearly interacts with the delivered nucleic acid
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    • 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
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    • C12N9/50—Proteinases, e.g. Endopeptidases (3.4.21-3.4.25)
    • C12N9/64—Proteinases, e.g. Endopeptidases (3.4.21-3.4.25) derived from animal tissue
    • C12N9/6421—Proteinases, e.g. Endopeptidases (3.4.21-3.4.25) derived from animal tissue from mammals
    • C12N9/6424—Serine endopeptidases (3.4.21)
    • C12N9/644—Coagulation factor IXa (3.4.21.22)
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
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CN201980078035.8A 2018-10-18 2019-10-18 核酸构建体和使用方法 Pending CN113272428A (zh)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201862747393P 2018-10-18 2018-10-18
US62/747,393 2018-10-18
US201962840343P 2019-04-29 2019-04-29
US62/840,343 2019-04-29
PCT/US2019/057084 WO2020082041A1 (en) 2018-10-18 2019-10-18 Nucleic acid constructs and methods of use

Publications (1)

Publication Number Publication Date
CN113272428A true CN113272428A (zh) 2021-08-17

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US (2) US20200268906A1 (https=)
EP (2) EP3867376A1 (https=)
JP (2) JP7593920B2 (https=)
CN (1) CN113272428A (https=)
AU (2) AU2019360269A1 (https=)
BR (1) BR112021007323A2 (https=)
CA (1) CA3116580A1 (https=)
CO (1) CO2021006362A2 (https=)
IL (2) IL322336A (https=)
MA (1) MA53919A (https=)
MX (1) MX2021004282A (https=)
MY (1) MY208708A (https=)
PH (1) PH12021550843A1 (https=)
SG (1) SG11202103732RA (https=)
TW (1) TW202028461A (https=)
WO (1) WO2020082041A1 (https=)

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EP4423271A2 (en) 2021-10-28 2024-09-04 Regeneron Pharmaceuticals, Inc. Crispr/cas-related methods and compositions for knocking out c5
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CN119421951A (zh) 2022-05-31 2025-02-11 瑞泽恩制药公司 用于c9orf72重复序列扩增疾病的crispr干扰疗法
IL318625A (en) 2022-07-29 2025-03-01 Regeneron Pharma Compositions and methods for transferrin receptor (TFR)-mediated delivery to brain and muscle
CN120265314A (zh) 2022-09-28 2025-07-04 瑞泽恩制药公司 抗体抗性修饰受体以增强基于细胞的疗法
IL320016A (en) 2022-11-04 2025-06-01 Regeneron Pharma Calcium voltage-gated channel auxiliary subunit gamma 1 (cacng1) binding proteins and cacng1-mediated delivery to skeletal muscle
AU2023379457A1 (en) 2022-11-14 2025-05-15 Regeneron Pharmaceuticals, Inc. Compositions and methods for fibroblast growth factor receptor 3-mediated delivery to astrocytes
AU2024309884A1 (en) 2023-06-30 2025-12-18 Regeneron Pharmaceuticals, Inc. Methods and compositions for increasing homology-directed repair
US20250049896A1 (en) 2023-07-28 2025-02-13 Regeneron Pharmaceuticals, Inc. Anti-tfr:acid sphingomyelinase for treatment of acid sphingomyelinase deficiency
AU2024315073A1 (en) 2023-07-28 2026-01-22 Regeneron Pharmaceuticals, Inc. Use of bgh-sv40l tandem polya to enhance transgene expression during unidirectional gene insertion
AU2024317483A1 (en) 2023-07-28 2026-01-29 Regeneron Pharmaceuticals, Inc. Anti-tfr:gaa and anti-cd63:gaa insertion for treatment of pompe disease
WO2025049524A1 (en) 2023-08-28 2025-03-06 Regeneron Pharmaceuticals, Inc. Cxcr4 antibody-resistant modified receptors
AU2024349204A1 (en) * 2023-09-29 2026-04-09 The Board Of Regents Of The University Of Texas System Endogenous nucleus targeting signal peptides and uses thereof
WO2025128871A2 (en) 2023-12-13 2025-06-19 Renagade Therapeutics Management Inc. Lipid nanoparticles comprising coding rna molecules for use in gene editing and as vaccines and therapeutic agents
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