CN110769845B - 对pcsk9基因中的识别序列具有特异性的工程化大范围核酸酶 - Google Patents

对pcsk9基因中的识别序列具有特异性的工程化大范围核酸酶 Download PDF

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CN110769845B
CN110769845B CN201880038349.0A CN201880038349A CN110769845B CN 110769845 B CN110769845 B CN 110769845B CN 201880038349 A CN201880038349 A CN 201880038349A CN 110769845 B CN110769845 B CN 110769845B
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meganuclease
engineered
pcs
pcsk9
seq
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CN110769845A (zh
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V·巴尔特斯维克
D·扬茨
J·J·史密斯
J·莱普
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Precision Biosciences Inc
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    • C12N9/22—Ribonucleases [RNase]; Deoxyribonucleases [DNase]
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    • A—HUMAN NECESSITIES
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    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
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    • C12N9/14—Hydrolases (3)
    • C12N9/48—Hydrolases (3) acting on peptide bonds (3.4)
    • 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)
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    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/01—Fusion polypeptide containing a localisation/targetting motif
    • C07K2319/09—Fusion polypeptide containing a localisation/targetting motif containing a nuclear localisation signal
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    • C07K2319/00—Fusion polypeptide
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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
    • C12N2800/80—Vectors containing sites for inducing double-stranded breaks, e.g. meganuclease restriction sites

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  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
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CN201880038349.0A 2017-04-21 2018-04-20 对pcsk9基因中的识别序列具有特异性的工程化大范围核酸酶 Active CN110769845B (zh)

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US201762488403P 2017-04-21 2017-04-21
US62/488,403 2017-04-21
US201762516966P 2017-06-08 2017-06-08
US62/516,966 2017-06-08
PCT/US2018/028607 WO2018195449A1 (en) 2017-04-21 2018-04-20 Engineered meganucleases specific for recognition sequences in the pcsk9 gene

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US20230175014A1 (en) * 2020-04-27 2023-06-08 The Trustees Of The University Of Pennsylvania Compositions and methods for reducing nuclease expression and off-target activity using a promoter with low transcriptional activity
KR20230003554A (ko) * 2020-04-27 2023-01-06 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 낮은 전사 활성을 갖는 프로모터를 사용하여 뉴클레아제 발현 및 표적-외 활성을 감소시키기 위한 조성물 및 방법
US20230183664A1 (en) 2020-05-11 2023-06-15 Precision Biosciences, Inc. Self-limiting viral vectors encoding nucleases
BR112022022212A2 (pt) 2020-05-12 2022-12-13 Univ Pennsylvania Composições para redução específica de drg de expressão de transgene
TW202233844A (zh) 2020-10-29 2022-09-01 賓州大學委員會 Aav衣殼及含有其之組成物
EP4244342A1 (en) * 2020-11-12 2023-09-20 Precision BioSciences, Inc. Engineered meganucleases having specificity for recognition sequences in the dystrophin gene
TW202309066A (zh) 2021-04-27 2023-03-01 賓州大學委員會 衍生自豬的腺相關病毒衣殼及其用途
WO2023087019A2 (en) 2021-11-15 2023-05-19 The Trustees Of The University Of Pennsylvania Compositions for drg-specific reduction of transgene expression
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EP3612205C0 (en) 2023-06-07
US20240101985A1 (en) 2024-03-28
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JP2022188058A (ja) 2022-12-20
IL270020B1 (en) 2023-04-01
IL301059B1 (en) 2025-12-01
AU2023201658A1 (en) 2023-06-22
CN110769845A (zh) 2020-02-07
EP3612205B9 (en) 2023-10-04
US20200131489A1 (en) 2020-04-30
JP2020517255A (ja) 2020-06-18
EP3612205A1 (en) 2020-02-26
AU2018254576A1 (en) 2019-11-07
EP3612205B1 (en) 2023-06-07
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AU2018254576B2 (en) 2022-12-22
IL270020B2 (en) 2023-08-01
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MX2019012452A (es) 2020-08-03
ES2953153T3 (es) 2023-11-08
WO2018195449A1 (en) 2018-10-25
US20250154481A1 (en) 2025-05-15
CA3060112A1 (en) 2018-10-25
US20240101986A1 (en) 2024-03-28
US11680254B2 (en) 2023-06-20
JP2025066725A (ja) 2025-04-23
KR102620399B1 (ko) 2024-01-04
KR20230170125A (ko) 2023-12-18
CN117264929A (zh) 2023-12-22
IL270020A (enExample) 2019-12-31
KR20190140982A (ko) 2019-12-20
EP4269596A3 (en) 2024-02-14
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