CA3062614A1 - Compositions and methods of use of arc capsids - Google Patents

Compositions and methods of use of arc capsids Download PDF

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Publication number
CA3062614A1
CA3062614A1 CA3062614A CA3062614A CA3062614A1 CA 3062614 A1 CA3062614 A1 CA 3062614A1 CA 3062614 A CA3062614 A CA 3062614A CA 3062614 A CA3062614 A CA 3062614A CA 3062614 A1 CA3062614 A1 CA 3062614A1
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Prior art keywords
arc
mrna
cell
protein
capsid
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Pending
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CA3062614A
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English (en)
French (fr)
Inventor
Jason D. Shepherd
Cameron Day
Elissa PASTUZYN
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University of Utah Research Foundation Inc
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University of Utah Research Foundation Inc
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Publication of CA3062614A1 publication Critical patent/CA3062614A1/en
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    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N7/00Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K14/00Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/005Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from viruses
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K14/00Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/46Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • C07K14/4701Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals not used
    • C07K14/4702Regulators; Modulating activity
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113Non-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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/87Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C12N15/88Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation using microencapsulation, e.g. using amphiphile liposome vesicle
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/12Type of nucleic acid catalytic nucleic acids, e.g. ribozymes
    • C12N2310/122Hairpin
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/14Type of nucleic acid interfering nucleic acids [NA]
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2740/00Reverse transcribing RNA viruses
    • C12N2740/00011Details
    • C12N2740/10011Retroviridae
    • C12N2740/10022New viral proteins or individual genes, new structural or functional aspects of known viral proteins or genes
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2740/00Reverse transcribing RNA viruses
    • C12N2740/00011Details
    • C12N2740/10011Retroviridae
    • C12N2740/10023Virus like particles [VLP]

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • Biomedical Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biotechnology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biochemistry (AREA)
  • Molecular Biology (AREA)
  • Biophysics (AREA)
  • Microbiology (AREA)
  • Medicinal Chemistry (AREA)
  • Virology (AREA)
  • 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)
  • Peptides Or Proteins (AREA)
CA3062614A 2017-05-10 2018-05-10 Compositions and methods of use of arc capsids Pending CA3062614A1 (en)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
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

Publications (1)

Publication Number Publication Date
CA3062614A1 true CA3062614A1 (en) 2018-11-15

Family

ID=64104936

Family Applications (1)

Application Number Title Priority Date Filing Date
CA3062614A Pending CA3062614A1 (en) 2017-05-10 2018-05-10 Compositions and methods of use of arc capsids

Country Status (7)

Country Link
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)

* Cited by examiner, † Cited by third party
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

Family Cites Families (9)

* 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
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
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
ES2897508T3 (es) * 2013-05-31 2022-03-01 Univ California Variantes de virus adenoasociados y métodos de uso de las mismas
US10624849B2 (en) * 2015-09-28 2020-04-21 Northwestern University Targeted extracellular vesicles comprising membrane proteins with engineered glycosylation sites

Also Published As

Publication number Publication date
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
JP7355382B2 (ja) 2023-10-03
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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