JP7344300B2 - Ａｒｃベースのカプシドおよびその使用 - Google Patents

Ａｒｃベースのカプシドおよびその使用 Download PDF

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JP7344300B2
JP7344300B2 JP2021539499A JP2021539499A JP7344300B2 JP 7344300 B2 JP7344300 B2 JP 7344300B2 JP 2021539499 A JP2021539499 A JP 2021539499A JP 2021539499 A JP2021539499 A JP 2021539499A JP 7344300 B2 JP7344300 B2 JP 7344300B2
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seq
amino acid
arc
acid sequence
gag
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JP2022502077A (ja
JPWO2020061229A5 (https=
JP2022502077A5 (https=
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マローン，コリン
ペイコン，イアン
ギルバート，ザック
ピーサレフ，アンドレイ
フレイツ，アダム
クリスプ，ジェシカ
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ブイエヌブイ ニューコ インク．
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    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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
    • C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • 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
    • C07—ORGANIC CHEMISTRY
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • 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
    • 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
    • A61K9/513—Organic macromolecular compounds; Dendrimers
    • A61K9/5169—Proteins, e.g. albumin, gelatin
    • 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
    • A61K9/5176—Compounds of unknown constitution, e.g. material from plants or animals
    • A61K9/5184—Virus capsids or envelopes enclosing drugs
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • 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
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K16/00—Immunoglobulins [IG], e.g. monoclonal or polyclonal antibodies
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86—Viral vectors
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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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
    • C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C12N15/90—Stable introduction of foreign DNA into chromosome
    • C12N15/902—Stable introduction of foreign DNA into chromosome using homologous recombination
    • C12N15/907—Stable introduction of foreign DNA into chromosome using homologous recombination in mammalian cells
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
    • C12N9/22—Ribonucleases [RNase]; Deoxyribonucleases [DNase]
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K39/00—Medicinal preparations containing antigens or antibodies
    • A61K2039/555—Medicinal preparations containing antigens or antibodies characterised by a specific combination antigen/adjuvant
    • A61K2039/55511—Organic adjuvants
    • A61K2039/55555—Liposomes; Vesicles, e.g. nanoparticles; Spheres, e.g. nanospheres; Polymers
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/20—Type of nucleic acid involving clustered regularly interspaced short palindromic repeats [CRISPR]

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • Molecular Biology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Health & Medical Sciences (AREA)
  • Zoology (AREA)
  • Biomedical Technology (AREA)
  • Biotechnology (AREA)
  • Biochemistry (AREA)
  • Medicinal Chemistry (AREA)
  • Biophysics (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Physics & Mathematics (AREA)
  • Microbiology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Plant Pathology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Epidemiology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Toxicology (AREA)
  • Virology (AREA)
  • Immunology (AREA)
  • Optics & Photonics (AREA)
  • Nanotechnology (AREA)
  • Cell Biology (AREA)
  • Mycology (AREA)
  • Botany (AREA)
  • Peptides Or Proteins (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicinal Preparation (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
JP2021539499A 2018-09-18 2019-09-18 Ａｒｃベースのカプシドおよびその使用 Active JP7344300B2 (ja)

Priority Applications (1)

Application Number Priority Date Filing Date Title
JP2023141593A JP2024007554A (ja) 2018-09-18 2023-08-31 Ａｒｃベースのカプシドおよびその使用

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201862733015P 2018-09-18 2018-09-18
US62/733,015 2018-09-18
PCT/US2019/051786 WO2020061229A2 (en) 2018-09-18 2019-09-18 Arc-based capsids and uses thereof

Related Child Applications (1)

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JP2023141593A Division JP2024007554A (ja) 2018-09-18 2023-08-31 Ａｒｃベースのカプシドおよびその使用

Publications (4)

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JP2022502077A JP2022502077A (ja) 2022-01-11
JPWO2020061229A5 JPWO2020061229A5 (https=) 2022-09-30
JP2022502077A5 JP2022502077A5 (https=) 2022-09-30
JP7344300B2 true JP7344300B2 (ja) 2023-09-13

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JP2021539499A Active JP7344300B2 (ja) 2018-09-18 2019-09-18 Ａｒｃベースのカプシドおよびその使用
JP2023141593A Pending JP2024007554A (ja) 2018-09-18 2023-08-31 Ａｒｃベースのカプシドおよびその使用

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US (3) US20220088224A1 (https=)
EP (1) EP3852813A4 (https=)
JP (2) JP7344300B2 (https=)
KR (2) KR20210070305A (https=)
CN (1) CN113164623A (https=)
AU (1) AU2019343045A1 (https=)
CA (1) CA3113095A1 (https=)
WO (1) WO2020061229A2 (https=)

Families Citing this family (15)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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
US20210403907A1 (en) * 2018-09-18 2021-12-30 Vnv Newco Inc. Arc-based capsids and uses thereof
US12351837B2 (en) 2019-01-23 2025-07-08 The Broad Institute, Inc. Supernegatively charged proteins and uses thereof
US20200347100A1 (en) * 2019-03-15 2020-11-05 The Broad Institute, Inc. Non-naturally occurring capsids for delivery of nucleic acids and/or proteins
JP7657726B2 (ja) 2019-03-19 2025-04-07 ザ ブロード インスティテュート，インコーポレーテッド 編集ヌクレオチド配列を編集するための方法および組成物
US12473543B2 (en) 2019-04-17 2025-11-18 The Broad Institute, Inc. Adenine base editors with reduced off-target effects
WO2020252455A1 (en) 2019-06-13 2020-12-17 The General Hospital Corporation Engineered human-endogenous virus-like particles and methods of use thereof for delivery to cells
US11129892B1 (en) 2020-05-18 2021-09-28 Vnv Newco Inc. Vaccine compositions comprising endogenous Gag polypeptides
EP4185600A4 (en) 2020-07-24 2024-12-04 The General Hospital Corporation Enhanced virus-like particles and methods of use thereof for delivery to cells
US20240102052A1 (en) * 2021-01-27 2024-03-28 Vnv Newco Inc. Pnma2-based capsids and uses thereof
EP4334445A4 (en) * 2021-05-04 2025-09-03 Univ Cornell MODIFIED EXTRACELLULAR VESICLES
WO2024006988A2 (en) * 2022-06-30 2024-01-04 The Broad Institute, Inc. Engineered delivery vesicles and uses thereof
WO2024026295A1 (en) * 2022-07-27 2024-02-01 Aera Therapeutics, Inc. Endogenous gag-based and pnma family capsids and uses thereof
WO2025129158A1 (en) * 2023-12-15 2025-06-19 The Broad Institute, Inc. Engineered arc delivery vesicles and uses thereof
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 (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2018516558A (ja) 2015-05-15 2018-06-28 ブクタリ 少なくとも２つのカプシド形成された非ウイルス性ｒｎａを含む、レトロウイルス粒子

Family Cites Families (120)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US4510245A (en) 1982-11-18 1985-04-09 Chiron Corporation Adenovirus promoter system
US4897355A (en) 1985-01-07 1990-01-30 Syntex (U.S.A.) Inc. N[ω,(ω-1)-dialkyloxy]- and N-[ω,(ω-1)-dialkenyloxy]-alk-1-yl-N,N,N-tetrasubstituted ammonium lipids and uses therefor
US5168062A (en) 1985-01-30 1992-12-01 University Of Iowa Research Foundation Transfer vectors and microorganisms containing human cytomegalovirus immediate-early promoter-regulatory DNA sequence
US4968615A (en) 1985-12-18 1990-11-06 Ciba-Geigy Corporation Deoxyribonucleic acid segment from a virus
US4737323A (en) 1986-02-13 1988-04-12 Liposome Technology, Inc. Liposome extrusion method
US4837028A (en) 1986-12-24 1989-06-06 Liposome Technology, Inc. Liposomes with enhanced circulation time
US5000960A (en) 1987-03-13 1991-03-19 Micro-Pak, Inc. Protein coupling to lipid vesicles
US5989886A (en) 1991-01-02 1999-11-23 The Johns Hopkins University Method for the inhibition and prevention of viral replication using fusions of a virus protein and a destructive enzyme
US5260065A (en) 1991-09-17 1993-11-09 Micro Vesicular Systems, Inc. Blended lipid vesicles
US5543158A (en) 1993-07-23 1996-08-06 Massachusetts Institute Of Technology Biodegradable injectable nanoparticles
US6007845A (en) 1994-07-22 1999-12-28 Massachusetts Institute Of Technology Nanoparticles and microparticles of non-linear hydrophilic-hydrophobic multiblock copolymers
US5985309A (en) 1996-05-24 1999-11-16 Massachusetts Institute Of Technology Preparation of particles for inhalation
US5855913A (en) 1997-01-16 1999-01-05 Massachusetts Instite Of Technology Particles incorporating surfactants for pulmonary drug delivery
US5944710A (en) 1996-06-24 1999-08-31 Genetronics, Inc. Electroporation-mediated intravascular delivery
US5869326A (en) 1996-09-09 1999-02-09 Genetronics, Inc. Electroporation employing user-configured pulsing scheme
GB9710049D0 (en) 1997-05-19 1997-07-09 Nycomed Imaging As Method
GB9710809D0 (en) 1997-05-23 1997-07-23 Medical Res Council Nucleic acid binding proteins
EP1025217B1 (en) 1997-10-24 2006-10-04 Invitrogen Corporation Recombinational cloning using nucleic acids having recombination sites
DE69942334D1 (de) 1998-03-02 2010-06-17 Massachusetts Inst Technology Poly-zinkfinger-proteine mit verbesserten linkern
FR2780069B1 (fr) 1998-06-23 2002-06-28 Inst Nat Sante Rech Med Famille de sequences nucleiques et de sequences proteiques deduites presentant des motifs retroviraux endogenes humains et leurs applications
US6534261B1 (en) 1999-01-12 2003-03-18 Sangamo Biosciences, Inc. Regulation of endogenous gene expression in cells using zinc finger proteins
US7013219B2 (en) 1999-01-12 2006-03-14 Sangamo Biosciences, Inc. Regulation of endogenous gene expression in cells using zinc finger proteins
US20030104526A1 (en) 1999-03-24 2003-06-05 Qiang Liu Position dependent recognition of GNN nucleotide triplets by zinc fingers
US6794136B1 (en) 2000-11-20 2004-09-21 Sangamo Biosciences, Inc. Iterative optimization in the design of binding proteins
US7030215B2 (en) 1999-03-24 2006-04-18 Sangamo Biosciences, Inc. Position dependent recognition of GNN nucleotide triplets by zinc fingers
FR2797889A1 (fr) 1999-09-01 2001-03-02 Bio Merieux Procede de detection de l'expression d'une proteine d'enveloppe d'un retrovirus endogene humain et utilisations d'un gene codant pour cette proteine
DK1385538T3 (da) 2001-04-05 2013-04-29 Univ Johns Hopkins Kimære vacciner omfattende det lumenale domæne fra LAMP-1 eller LAMP-2
AU2002336760A1 (en) 2001-09-26 2003-06-10 Mayo Foundation For Medical Education And Research Mutable vaccines
JP2005535308A (ja) 2002-06-13 2005-11-24 カイロン コーポレイション Ｈｍｌ−２ポリペプチド発現用ベクター
EP1651667B1 (en) 2003-04-04 2012-03-28 Centre National De La Recherche Scientifique Protein with fusogenic activity, nucleic acid sequences encoding said protein and pharmaceutical compositions comprising the same
ZA200608178B (en) 2004-03-30 2008-10-29 Roussy Inst Gustave Polypeptide sequence involved in the modulation of the immunosuppresive effect of viral proteins
GB0418651D0 (en) 2004-08-20 2004-09-22 Medical Res Council Method
CA2891996A1 (en) 2005-10-18 2007-04-26 Duke University Rationally-designed meganucleases with altered sequence specificity and dna-binding affinity
GB0526210D0 (en) * 2005-12-22 2006-02-01 Oxford Biomedica Ltd Vectors
JP2008078613A (ja) 2006-08-24 2008-04-03 Rohm Co Ltd 窒化物半導体の製造方法及び窒化物半導体素子
WO2008149176A1 (en) 2007-06-06 2008-12-11 Cellectis Meganuclease variants cleaving a dna target sequence from the mouse rosa26 locus and uses thereof
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
US20100285056A1 (en) 2008-01-09 2010-11-11 Konkuk University Industrial Cooperation Corp. Baculovirus-Based Vaccines
ES2646630T3 (es) 2008-11-07 2017-12-14 Massachusetts Institute Of Technology Lipidoides aminoalcohólicos y usos de los mismos
US20110027239A1 (en) 2009-07-29 2011-02-03 Tissue Genesis, Inc. Adipose-derived stromal cells (asc) as delivery tool for treatment of cancer
WO2011028929A2 (en) 2009-09-03 2011-03-10 The Regents Of The University Of California Nitrate-responsive promoter
HUE041436T2 (hu) 2009-12-10 2019-05-28 Univ Minnesota Tal-effektor-közvetített DNS-módosítás
US8771985B2 (en) 2010-04-26 2014-07-08 Sangamo Biosciences, Inc. Genome editing of a Rosa locus using zinc-finger nucleases
EP2609135A4 (en) 2010-08-26 2015-05-20 Massachusetts Inst Technology POLY (BETA AMINO ALCOHOLS), THEIR PREPARATION AND USES THEREOF
US9238716B2 (en) 2011-03-28 2016-01-19 Massachusetts Institute Of Technology Conjugated lipomers and uses thereof
EP3069785A1 (en) 2011-10-25 2016-09-21 The University Of British Columbia Limit size lipid nanoparticles and related methods
US20140308304A1 (en) 2011-12-07 2014-10-16 Alnylam Pharmaceuticals, Inc. Lipids for the delivery of active agents
AU2013243948A1 (en) 2012-04-02 2014-10-30 Moderna Therapeutics, Inc. Modified polynucleotides for the production of proteins associated with human disease
US9254311B2 (en) 2012-04-02 2016-02-09 Moderna Therapeutics, Inc. Modified polynucleotides for the production of proteins
JP2015515530A (ja) 2012-04-18 2015-05-28 アローヘッド リサーチ コーポレイション インビボ核酸送達のためのポリ（アクリラート）ポリマー
KR102530118B1 (ko) 2012-07-25 2023-05-08 더 브로드 인스티튜트, 인코퍼레이티드 유도 ｄｎａ 결합 단백질 및 게놈 교란 도구 및 이의 적용
WO2014093718A1 (en) 2012-12-12 2014-06-19 The Broad Institute, Inc. Methods, systems, and apparatus for identifying target sequences for cas enzymes or crispr-cas systems for target sequences and conveying results thereof
US8697359B1 (en) 2012-12-12 2014-04-15 The Broad Institute, Inc. CRISPR-Cas systems and methods for altering expression of gene products
DK2931898T3 (en) 2012-12-12 2016-06-20 Massachusetts Inst Technology CONSTRUCTION AND OPTIMIZATION OF SYSTEMS, PROCEDURES AND COMPOSITIONS FOR SEQUENCE MANIPULATION WITH FUNCTIONAL DOMAINS
DK2931897T3 (en) 2012-12-12 2018-02-05 Broad Inst Inc CONSTRUCTION, MODIFICATION AND OPTIMIZATION OF SYSTEMS, PROCEDURES AND COMPOSITIONS FOR SEQUENCE MANIPULATION AND THERAPEUTICAL APPLICATIONS
CN105121648B (zh) 2012-12-12 2021-05-07 布罗德研究所有限公司 用于序列操纵的系统、方法和优化的指导组合物的工程化
WO2014093709A1 (en) 2012-12-12 2014-06-19 The Broad Institute, Inc. Methods, models, systems, and apparatus for identifying target sequences for cas enzymes or crispr-cas systems for target sequences and conveying results thereof
EP2931899A1 (en) 2012-12-12 2015-10-21 The Broad Institute, Inc. Functional genomics using crispr-cas systems, compositions, methods, knock out libraries and applications thereof
CA2894684A1 (en) 2012-12-12 2014-06-19 The Broad Institute, Inc. Engineering and optimization of improved crispr-cas systems, methods and enzyme compositions for sequence manipulation in eukaryotes
WO2014093694A1 (en) 2012-12-12 2014-06-19 The Broad Institute, Inc. Crispr-cas nickase systems, methods and compositions for sequence manipulation in eukaryotes
AU2013359262C1 (en) 2012-12-12 2021-05-13 Massachusetts Institute Of Technology CRISPR-Cas component systems, methods and compositions for sequence manipulation
WO2014118272A1 (en) 2013-01-30 2014-08-07 Santaris Pharma A/S Antimir-122 oligonucleotide carbohydrate conjugates
US9481905B2 (en) 2013-02-19 2016-11-01 Orizhan Bioscience Limited Method of using neutrilized DNA (N-DNA) as surface probe for high throughput detection platform
US9693958B2 (en) 2013-03-15 2017-07-04 Cureport, Inc. Methods and devices for preparation of lipid nanoparticles
US11332719B2 (en) 2013-03-15 2022-05-17 The Broad Institute, Inc. Recombinant virus and preparations thereof
MX2015017312A (es) 2013-06-17 2017-04-10 Broad Inst Inc Suministro y uso de composiciones, vectores y sistemas crispr-cas para la modificación dirigida y terapia hepáticas.
EP3011030B1 (en) 2013-06-17 2023-11-08 The Broad Institute, Inc. Optimized crispr-cas double nickase systems, methods and compositions for sequence manipulation
KR20250012194A (ko) 2013-06-17 2025-01-23 더 브로드 인스티튜트, 인코퍼레이티드 바이러스 구성성분을 사용하여 장애 및 질환을 표적화하기 위한 crispr-cas 시스템 및 조성물의 전달, 용도 및 치료 적용
KR20160019553A (ko) 2013-06-17 2016-02-19 더 브로드 인스티튜트, 인코퍼레이티드 유사분열 후 세포의 질병 및 장애를 표적화하고 모델링하기 위한 시스템, 방법 및 조성물의 전달, 유전자 조작 및 최적화
WO2014204724A1 (en) 2013-06-17 2014-12-24 The Broad Institute Inc. Delivery, engineering and optimization of tandem guide systems, methods and compositions for sequence manipulation
WO2014204723A1 (en) 2013-06-17 2014-12-24 The Broad Institute Inc. Oncogenic models based on delivery and use of the crispr-cas systems, vectors and compositions
EP3725885A1 (en) 2013-06-17 2020-10-21 The Broad Institute, Inc. Functional genomics using crispr-cas systems, compositions methods, screens and applications thereof
EP3058091B1 (en) 2013-10-18 2020-03-25 The Broad Institute, Inc. Spatial and cellular mapping of biomolecules in situ by high-throughput sequencing
AU2014346559B2 (en) 2013-11-07 2020-07-09 Editas Medicine,Inc. CRISPR-related methods and compositions with governing gRNAs
AU2014361781B2 (en) 2013-12-12 2021-04-01 Massachusetts Institute Of Technology Delivery, use and therapeutic applications of the CRISPR -Cas systems and compositions for genome editing
EP3080259B1 (en) 2013-12-12 2023-02-01 The Broad Institute, Inc. Engineering of systems, methods and optimized guide compositions with new architectures for sequence manipulation
WO2015089364A1 (en) 2013-12-12 2015-06-18 The Broad Institute Inc. Crystal structure of a crispr-cas system, and uses thereof
MX2016007325A (es) 2013-12-12 2017-07-19 Broad Inst Inc Composiciones y metodos de uso de sistemas crispr-cas en desordenes debidos a repeticion de nucleotidos.
JP6793547B2 (ja) 2013-12-12 2020-12-02 ザ・ブロード・インスティテュート・インコーポレイテッド 最適化機能ＣＲＩＳＰＲ−Ｃａｓ系による配列操作のための系、方法および組成物
US20150165054A1 (en) * 2013-12-12 2015-06-18 President And Fellows Of Harvard College Methods for correcting caspase-9 point mutations
BR112016013207A2 (pt) 2013-12-12 2017-09-26 Massachusetts Inst Technology administração, uso e aplicações terapêuticas dos sistemas crispr-cas e composições para o hbv e distúrbios e doenças virais
EP3470089A1 (en) 2013-12-12 2019-04-17 The Broad Institute Inc. Delivery, use and therapeutic applications of the crispr-cas systems and compositions for targeting disorders and diseases using particle delivery components
AU2014361834B2 (en) 2013-12-12 2020-10-22 Massachusetts Institute Of Technology CRISPR-Cas systems and methods for altering expression of gene products, structural information and inducible modular Cas enzymes
WO2016049258A2 (en) 2014-09-25 2016-03-31 The Broad Institute Inc. Functional screening with optimized functional crispr-cas systems
WO2016094874A1 (en) 2014-12-12 2016-06-16 The Broad Institute Inc. Escorted and functionalized guides for crispr-cas systems
WO2016094872A1 (en) 2014-12-12 2016-06-16 The Broad Institute Inc. Dead guides for crispr transcription factors
EP3889260A1 (en) 2014-12-12 2021-10-06 The Broad Institute, Inc. Protected guide rnas (pgrnas)
WO2016106236A1 (en) 2014-12-23 2016-06-30 The Broad Institute Inc. Rna-targeting system
WO2016106244A1 (en) 2014-12-24 2016-06-30 The Broad Institute Inc. Crispr having or associated with destabilization domains
US12060391B2 (en) 2015-03-16 2024-08-13 The Broad Institute, Inc. Constructs for continuous monitoring of live cells
US9738687B2 (en) 2015-04-10 2017-08-22 Feldan Bio Inc. Polypeptide-based shuttle agents for improving the transduction efficiency of polypeptide cargos to the cytosol of target eukaryotic cells, uses thereof, methods and kits relating to same
US10624849B2 (en) 2015-09-28 2020-04-21 Northwestern University Targeted extracellular vesicles comprising membrane proteins with engineered glycosylation sites
WO2017068077A1 (en) 2015-10-20 2017-04-27 Institut National De La Sante Et De La Recherche Medicale (Inserm) Methods and products for genetic engineering
EP3235828A1 (en) 2016-04-21 2017-10-25 Genethon Stable pseudotyped lentiviral particles and uses thereof
EP3452101A2 (en) 2016-05-04 2019-03-13 CureVac AG Rna encoding a therapeutic protein
CA3062614A1 (en) 2017-05-10 2018-11-15 University Of Utah Research Foundation Compositions and methods of use of arc capsids
EP3625342B1 (en) 2017-05-18 2022-08-24 The Broad Institute, Inc. Systems, methods, and compositions for targeted nucleic acid editing
WO2018213708A1 (en) 2017-05-18 2018-11-22 The Broad Institute, Inc. Systems, methods, and compositions for targeted nucleic acid editing
WO2018234576A1 (en) 2017-06-22 2018-12-27 Institut Gustave Roussy HUMAN ENDOGENOUS RETROVIRAL PROTEIN
AU2018290843B2 (en) 2017-06-26 2025-04-24 Massachusetts Institute Of Technology CRISPR/Cas-adenine deaminase based compositions, systems, and methods for targeted nucleic acid editing
WO2019005886A1 (en) 2017-06-26 2019-01-03 The Broad Institute, Inc. CRISPR / CAS-CYTIDINE DEAMINASE COMPOSITIONS, SYSTEMS AND METHODS FOR TARGETED EDITING OF NUCLEIC ACIDS
WO2019018423A1 (en) 2017-07-17 2019-01-24 The Broad Institute, Inc. NEW TYPE VI CRISPR ORTHOLOGISTS AND ASSOCIATED SYSTEMS
AU2018322831B2 (en) 2017-09-01 2023-04-27 Inprother Aps A vaccine for use in the prophylaxis and/or treatment of a disease
JP2020535802A (ja) 2017-09-21 2020-12-10 ザ・ブロード・インスティテュート・インコーポレイテッド 標的化核酸編集のための系、方法、及び組成物
US20190096250A1 (en) 2017-09-28 2019-03-28 Uber Technologies, Inc. Systems and Methods for Determining Whether an Autonomous Vehicle Can Provide a Requested Service for a Rider
WO2019071048A1 (en) 2017-10-04 2019-04-11 The Broad Institute, Inc. SYSTEMS, METHODS AND COMPOSITIONS FOR TARGETED NUCLEIC ACID EDITION
WO2019077149A1 (en) 2017-10-20 2019-04-25 Genethon USE OF SYNCYTINE FOR TARGETING DRUG AND GENE ADMINISTRATION TO REGENERATE MUSCLE TISSUE
JP7208231B2 (ja) 2017-10-20 2023-01-18 ジェネトン 肺組織への標的化薬物及び遺伝子送達のためのシンシチンの使用
US20210163933A1 (en) 2017-12-11 2021-06-03 University Of Massachusetts Arc protein extracellular vesicle nucleic acid delivery platform
WO2019126762A2 (en) 2017-12-22 2019-06-27 The Broad Institute, Inc. Cas12a systems, methods, and compositions for targeted rna base editing
WO2019126709A1 (en) 2017-12-22 2019-06-27 The Broad Institute, Inc. Cas12b systems, methods, and compositions for targeted dna base editing
WO2019213257A1 (en) * 2018-05-01 2019-11-07 Wake Forest University Health Sciences Lentiviral-based vectors and related systems and methods for eukaryotic gene editing
WO2020012335A1 (en) 2018-07-10 2020-01-16 Alia Therapeutics S.R.L. Vesicles for traceless delivery of guide rna molecules and/or guide rna molecule/rna-guided nuclease complex(es) and a production method thereof
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
US20210403907A1 (en) 2018-09-18 2021-12-30 Vnv Newco Inc. Arc-based capsids and uses thereof
CA3124110A1 (en) 2018-12-17 2020-06-25 The Broad Institute, Inc. Crispr-associated transposase systems and methods of use thereof
US20200347100A1 (en) 2019-03-15 2020-11-05 The Broad Institute, Inc. Non-naturally occurring capsids for delivery of nucleic acids and/or proteins
WO2020252455A1 (en) 2019-06-13 2020-12-17 The General Hospital Corporation Engineered human-endogenous virus-like particles and methods of use thereof for delivery to cells
CN114616336B (zh) 2019-09-20 2025-11-14 博德研究所 用于将货物递送至靶细胞的组合物和方法
US11129892B1 (en) * 2020-05-18 2021-09-28 Vnv Newco Inc. Vaccine compositions comprising endogenous Gag polypeptides

Patent Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2018516558A (ja) 2015-05-15 2018-06-28 ブクタリ 少なくとも２つのカプシド形成された非ウイルス性ｒｎａを含む、レトロウイルス粒子

Non-Patent Citations (2)

* Cited by examiner, † Cited by third party
Title
Cell，2018年01月11日，Vol.172, pp.275-288
TREND in Genetics，2006年，Vol.22, No.11, pp.585-589

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