CN117264929A - 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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Publication number
CN117264929A
CN117264929A CN202311029425.0A CN202311029425A CN117264929A CN 117264929 A CN117264929 A CN 117264929A CN 202311029425 A CN202311029425 A CN 202311029425A CN 117264929 A CN117264929 A CN 117264929A
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meganuclease
engineered meganuclease
seq
engineered
acid sequence
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Chinese (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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Precision Biosciences Inc
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    • C12N9/14—Hydrolases (3)
    • C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
    • C12N9/22—Ribonucleases [RNase]; Deoxyribonucleases [DNase]
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    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
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    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
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    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/48—Preparations in capsules, e.g. of gelatin, of chocolate
    • A61K9/50—Microcapsules having a gas, liquid or semi-solid filling; Solid microparticles or pellets surrounded by a distinct coating layer, e.g. coated microspheres, coated drug crystals
    • A61K9/51—Nanocapsules; Nanoparticles
    • A61K9/5107—Excipients; Inactive ingredients
    • A61K9/5123—Organic compounds, e.g. fats, sugars
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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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
    • C12N15/63—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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    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • 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)
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
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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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    • C07K—PEPTIDES
    • 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
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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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  • Obesity (AREA)
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  • Immunology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Nanotechnology (AREA)
  • Optics & Photonics (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Enzymes And Modification Thereof (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
CN202311029425.0A 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 Pending CN117264929A (zh)

Applications Claiming Priority (6)

Application Number Priority Date Filing Date Title
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
CN201880038349.0A CN110769845B (zh) 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
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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CN201880038349.0A Active CN110769845B (zh) 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

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US (4) US11680254B2 (enExample)
EP (2) EP3612205B9 (enExample)
JP (4) JP2020517255A (enExample)
KR (2) KR20230170125A (enExample)
CN (2) CN117264929A (enExample)
AU (2) AU2018254576B2 (enExample)
CA (1) CA3060112A1 (enExample)
ES (1) ES2953153T3 (enExample)
IL (2) IL270020B2 (enExample)
MX (2) MX2019012452A (enExample)
WO (1) WO2018195449A1 (enExample)

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WO2021222249A1 (en) * 2020-04-27 2021-11-04 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
US20230183664A1 (en) 2020-05-11 2023-06-15 Precision Biosciences, Inc. Self-limiting viral vectors encoding nucleases
KR20230010670A (ko) 2020-05-12 2023-01-19 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 이식유전자 발현의 drg-특이적 감소를 위한 조성물
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JP2025066725A (ja) 2025-04-23
MX2023001877A (es) 2023-03-10
EP3612205B1 (en) 2023-06-07
US20200131489A1 (en) 2020-04-30
JP2022188058A (ja) 2022-12-20
MX2019012452A (es) 2020-08-03
EP3612205B9 (en) 2023-10-04
IL270020B1 (en) 2023-04-01
US20250154481A1 (en) 2025-05-15
EP4269596A3 (en) 2024-02-14
IL301059A (en) 2023-05-01
CA3060112A1 (en) 2018-10-25
US20240101986A1 (en) 2024-03-28
JP2023058509A (ja) 2023-04-25
KR20230170125A (ko) 2023-12-18
IL270020B2 (en) 2023-08-01
US20240101985A1 (en) 2024-03-28
KR20190140982A (ko) 2019-12-20
IL270020A (enExample) 2019-12-31
JP2020517255A (ja) 2020-06-18
JP7214915B2 (ja) 2023-01-30
EP3612205A1 (en) 2020-02-26
CN110769845A (zh) 2020-02-07
EP4269596A2 (en) 2023-11-01
US11680254B2 (en) 2023-06-20
ES2953153T3 (es) 2023-11-08
EP3612205C0 (en) 2023-06-07
AU2018254576B2 (en) 2022-12-22
AU2018254576A1 (en) 2019-11-07
KR102620399B1 (ko) 2024-01-04
AU2023201658A1 (en) 2023-06-22
CN110769845B (zh) 2023-08-15
WO2018195449A1 (en) 2018-10-25
IL301059B1 (en) 2025-12-01

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