JP7355382B2 - Ａｒｃカプシドの組成物及び使用方法 - Google Patents

Ａｒｃカプシドの組成物及び使用方法 Download PDF

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JP7355382B2
JP7355382B2 JP2019561875A JP2019561875A JP7355382B2 JP 7355382 B2 JP7355382 B2 JP 7355382B2 JP 2019561875 A JP2019561875 A JP 2019561875A JP 2019561875 A JP2019561875 A JP 2019561875A JP 7355382 B2 JP7355382 B2 JP 7355382B2
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arc
protein
mrna
capsid
nucleic acid
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JP2020523983A5 (enExample
JP2020523983A (ja
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ジェイソン ディー シェパード
キャメロン デイ
エリッサ パストゥジン
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ユニヴァーシティー オブ ユタ リサーチ ファウンデーション
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    • C12N15/09—Recombinant DNA-technology
    • C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C12N15/88—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation using microencapsulation, e.g. using amphiphile liposome vesicle
    • C—CHEMISTRY; METALLURGY
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    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/005—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from viruses
    • C—CHEMISTRY; METALLURGY
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    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • C07K14/4701—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals not used
    • C07K14/4702—Regulators; Modulating activity
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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
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/12—Type of nucleic acid catalytic nucleic acids, e.g. ribozymes
    • C12N2310/122—Hairpin
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2740/00—Reverse transcribing RNA viruses
    • C12N2740/00011—Details
    • C12N2740/10011—Retroviridae
    • C12N2740/10022—New viral proteins or individual genes, new structural or functional aspects of known viral proteins or genes
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    • C12N2740/00—Reverse transcribing RNA viruses
    • C12N2740/00011—Details
    • C12N2740/10011—Retroviridae
    • C12N2740/10023—Virus like particles [VLP]

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  • Health & Medical Sciences (AREA)
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  • Wood Science & Technology (AREA)
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  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Immunology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Toxicology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicinal Preparation (AREA)
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JP2019561875A 2017-05-10 2018-05-10 Ａｒｃカプシドの組成物及び使用方法 Active JP7355382B2 (ja)

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JP2023076834A JP7774319B2 (ja) 2017-05-10 2023-05-08 Ａｒｃカプシドの組成物及び使用方法

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US201762504370P 2017-05-10 2017-05-10
US62/504,370 2017-05-10
US201762543130P 2017-08-09 2017-08-09
US62/543,130 2017-08-09
PCT/US2018/032105 WO2018209113A1 (en) 2017-05-10 2018-05-10 Compositions and methods of use of arc capsids

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JP2020523983A JP2020523983A (ja) 2020-08-13
JP2020523983A5 JP2020523983A5 (enExample) 2021-07-26
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US (1) US20210189432A1 (enExample)
EP (1) EP3621660A4 (enExample)
JP (2) JP7355382B2 (enExample)
CN (1) CN110997011A (enExample)
AU (1) AU2018265395B2 (enExample)
CA (1) CA3062614A1 (enExample)
WO (1) WO2018209113A1 (enExample)

Families Citing this family (12)

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Publication number Priority date Publication date Assignee Title
US9340799B2 (en) 2013-09-06 2016-05-17 President And Fellows Of Harvard College MRNA-sensing switchable gRNAs
WO2019118497A1 (en) * 2017-12-11 2019-06-20 University Of Massachusetts Arc protein extracellular vesicle nucleic acid delivery platform
JP7344300B2 (ja) 2018-09-18 2023-09-13 ブイエヌブイ ニューコ インク． Ａｒｃベースのカプシドおよびその使用
US12351837B2 (en) 2019-01-23 2025-07-08 The Broad Institute, Inc. Supernegatively charged proteins and uses thereof
US12473543B2 (en) 2019-04-17 2025-11-18 The Broad Institute, Inc. Adenine base editors with reduced off-target effects
CN114258398A (zh) 2019-06-13 2022-03-29 总医院公司 工程化的人内源性病毒样颗粒及使用其递送至细胞的方法
US11129892B1 (en) 2020-05-18 2021-09-28 Vnv Newco Inc. Vaccine compositions comprising endogenous Gag polypeptides
BR112023001272A2 (pt) 2020-07-24 2023-04-04 Massachusetts Gen Hospital Partículas semelhantes a vírus aprimoradas e métodos de uso das mesmas para entrega às células
CN117580949A (zh) * 2021-05-04 2024-02-20 康奈尔大学 工程化的胞外囊泡
WO2024030455A2 (en) * 2022-08-01 2024-02-08 University Of Utah Research Foundation Compositions and methods for labeling and detecting binding partners
CN118576725B (zh) * 2024-08-02 2025-06-03 中国人民解放军军事科学院军事医学研究院 一种以arc蛋白为载体的纳米颗粒及其制备方法和应用
CN120248078B (zh) * 2025-05-28 2025-11-28 中国人民解放军军事科学院军事医学研究院 一种基于arc蛋白的修饰多肽及其作为核酸递送载体的应用

Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2009042727A1 (en) 2007-09-24 2009-04-02 The Johns Hopkins University Immediate early gene arc interacts with endocytic machinery and regulates the trafficking and function of presenilin
JP2016526045A (ja) 2013-05-31 2016-09-01 ザ リージェンツ オブ ザ ユニバーシティ オブ カリフォルニア アデノ随伴ウイルス変異体及びその使用方法
US20170087087A1 (en) 2015-09-28 2017-03-30 Northwestern University Targeted extracellular vesicles comprising membrane proteins with engineered glycosylation sites

Family Cites Families (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US6204059B1 (en) * 1994-06-30 2001-03-20 University Of Pittsburgh AAV capsid vehicles for molecular transfer
US7638269B2 (en) * 1997-02-07 2009-12-29 The Regents Of The University Of California Viral capsid assembly intermediates and methods of production
WO2009097691A1 (en) * 2008-02-08 2009-08-13 University Health Network USE OF P27kip1 FOR THE PREVENTION AND TREATMENT OF HEART FAILURE
MX2011007980A (es) * 2009-02-02 2011-08-17 Okairos Ag Secuencias de acido nucleico y aminoacido de adenovirus de simio, vectores que contienen los mismos y usos de las mismas.
GB0911870D0 (en) * 2009-07-08 2009-08-19 Ucl Business Plc Optimised coding sequence and promoter
US20130302364A1 (en) * 2010-11-10 2013-11-14 Laboratorios Del Dr. Esteve, S.A. Highly immunogenic hiv p24 sequences

Patent Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2009042727A1 (en) 2007-09-24 2009-04-02 The Johns Hopkins University Immediate early gene arc interacts with endocytic machinery and regulates the trafficking and function of presenilin
JP2016526045A (ja) 2013-05-31 2016-09-01 ザ リージェンツ オブ ザ ユニバーシティ オブ カリフォルニア アデノ随伴ウイルス変異体及びその使用方法
US20170087087A1 (en) 2015-09-28 2017-03-30 Northwestern University Targeted extracellular vesicles comprising membrane proteins with engineered glycosylation sites

Non-Patent Citations (4)

* Cited by examiner, † Cited by third party
Title
Biochem. J.，2015年，Vol. 468，pp. 145-158
Biochem. J.，2015年，Vol. 469，pp. e1-e3
Neuron，2013年，Vol. 78，pp. 1024-1035
Neuron，2015年，Vol. 86, Issue 2，pp. 490-500

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US20210189432A1 (en) 2021-06-24
AU2018265395A1 (en) 2019-12-05
EP3621660A4 (en) 2021-01-20
WO2018209113A1 (en) 2018-11-15
CN110997011A (zh) 2020-04-10
CA3062614A1 (en) 2018-11-15
JP2023106451A (ja) 2023-08-01
JP7774319B2 (ja) 2025-11-21
EP3621660A1 (en) 2020-03-18
JP2020523983A (ja) 2020-08-13
AU2018265395B2 (en) 2025-04-10

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