JP7059179B2 - 遺伝子操作のための方法及び製品 - Google Patents

遺伝子操作のための方法及び製品 Download PDF

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JP7059179B2
JP7059179B2 JP2018520155A JP2018520155A JP7059179B2 JP 7059179 B2 JP7059179 B2 JP 7059179B2 JP 2018520155 A JP2018520155 A JP 2018520155A JP 2018520155 A JP2018520155 A JP 2018520155A JP 7059179 B2 JP7059179 B2 JP 7059179B2
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virus
derived
particles
protein
cas
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JP2018531023A (ja
JP2018531023A6 (ja
JP2018531023A5 (enExample
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オールマン，テオフィル
マンジョ，フィリップ
リッチ，エミリアーノ
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Centre National de la Recherche Scientifique CNRS
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    • 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
    • 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
    • C12N15/10—Processes for the isolation, preparation or purification of DNA or RNA
    • C12N15/102—Mutagenizing nucleic acids
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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
    • 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
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    • C12N7/00—Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof
    • 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]
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/40—Fusion polypeptide containing a tag for immunodetection, or an epitope for immunisation
    • C07K2319/43—Fusion polypeptide containing a tag for immunodetection, or an epitope for immunisation containing a FLAG-tag
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/50—Fusion polypeptide containing protease site
    • 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
    • C12N2310/20—Type of nucleic acid involving clustered regularly interspaced short palindromic repeats [CRISPR]
    • 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
    • C12N2740/10023—Virus like particles [VLP]
    • 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
    • C12N2740/10041—Use of virus, viral particle or viral elements as a vector
    • C12N2740/10042—Use of virus, viral particle or viral elements as a vector virus or viral particle as vehicle, e.g. encapsulating small organic molecule
    • 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
    • C12N2740/00—Reverse transcribing RNA viruses
    • C12N2740/00011—Details
    • C12N2740/10011—Retroviridae
    • C12N2740/13011—Gammaretrovirus, e.g. murine leukeamia virus
    • C12N2740/13023—Virus like particles [VLP]
    • 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
    • C12N2740/00—Reverse transcribing RNA viruses
    • C12N2740/00011—Details
    • C12N2740/10011—Retroviridae
    • C12N2740/13011—Gammaretrovirus, e.g. murine leukeamia virus
    • C12N2740/13041—Use of virus, viral particle or viral elements as a vector
    • C12N2740/13042—Use of virus, viral particle or viral elements as a vector virus or viral particle as vehicle, e.g. encapsulating small organic molecule
    • 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
    • C12N2800/00—Nucleic acids vectors
    • C12N2800/80—Vectors containing sites for inducing double-stranded breaks, e.g. meganuclease restriction sites

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  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
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  • Wood Science & Technology (AREA)
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  • Biotechnology (AREA)
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  • General Health & Medical Sciences (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • Biophysics (AREA)
  • Medicinal Chemistry (AREA)
  • Physics & Mathematics (AREA)
  • Virology (AREA)
  • Plant Pathology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Crystallography & Structural Chemistry (AREA)
  • Immunology (AREA)
  • Mycology (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Peptides Or Proteins (AREA)
  • Enzymes And Modification Thereof (AREA)
  • Apparatus Associated With Microorganisms And Enzymes (AREA)
JP2018520155A 2015-10-20 2016-10-20 遺伝子操作のための方法及び製品 Active JP7059179B2 (ja)

Priority Applications (2)

Application Number Priority Date Filing Date Title
JP2021212300A JP7621930B2 (ja) 2015-10-20 2021-12-27 遺伝子操作のための方法及び製品
JP2025005188A JP2025063185A (ja) 2015-10-20 2025-01-15 遺伝子操作のための方法及び製品

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
EP15306678.2 2015-10-20
EP15306678 2015-10-20
PCT/EP2016/075289 WO2017068077A1 (en) 2015-10-20 2016-10-20 Methods and products for genetic engineering

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JP2018531023A JP2018531023A (ja) 2018-10-25
JP2018531023A6 JP2018531023A6 (ja) 2018-12-13
JP2018531023A5 JP2018531023A5 (enExample) 2019-11-28
JP7059179B2 true JP7059179B2 (ja) 2022-04-25

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Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2022046680A (ja) * 2015-10-20 2022-03-23 アンスティチュ ナショナル ドゥ ラ サンテ エ ドゥ ラ ルシェルシュ メディカル 遺伝子操作のための方法及び製品

Families Citing this family (94)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20100183558A1 (en) 2008-10-17 2010-07-22 Zhennan Lai Safe lentiviral vectors for targeted delivery of multiple therapeutic molecules
WO2013066438A2 (en) 2011-07-22 2013-05-10 President And Fellows Of Harvard College Evaluation and improvement of nuclease cleavage specificity
US20150044192A1 (en) 2013-08-09 2015-02-12 President And Fellows Of Harvard College Methods for identifying a target site of a cas9 nuclease
US9359599B2 (en) 2013-08-22 2016-06-07 President And Fellows Of Harvard College Engineered transcription activator-like effector (TALE) domains and uses thereof
US9340799B2 (en) 2013-09-06 2016-05-17 President And Fellows Of Harvard College MRNA-sensing switchable gRNAs
US9526784B2 (en) 2013-09-06 2016-12-27 President And Fellows Of Harvard College Delivery system for functional nucleases
US9388430B2 (en) 2013-09-06 2016-07-12 President And Fellows Of Harvard College Cas9-recombinase fusion proteins and uses thereof
US20150166984A1 (en) 2013-12-12 2015-06-18 President And Fellows Of Harvard College Methods for correcting alpha-antitrypsin point mutations
WO2016022363A2 (en) 2014-07-30 2016-02-11 President And Fellows Of Harvard College Cas9 proteins including ligand-dependent inteins
WO2017007994A1 (en) 2015-07-08 2017-01-12 American Gene Technologies International Inc. Hiv pre-immunization and immunotherapy
SG10202104041PA (en) 2015-10-23 2021-06-29 Harvard College Nucleobase editors and uses thereof
US10137144B2 (en) 2016-01-15 2018-11-27 American Gene Technologies International Inc. Methods and compositions for the activation of gamma-delta T-cells
CN111569090A (zh) 2016-01-15 2020-08-25 美国基因技术国际有限公司 用于活化γ-δ T细胞的方法和组合物
JP7153332B2 (ja) 2016-02-08 2022-10-14 アメリカン ジーン テクノロジーズ インターナショナル インコーポレイテッド Ｈｉｖワクチン接種および免疫療法
ES2911448T3 (es) 2016-03-09 2022-05-19 American Gene Tech Int Inc Vectores combinados y métodos para el tratamiento del cáncer
US11976292B2 (en) 2016-06-08 2024-05-07 American Gene Technologies International Inc. Non-integrating viral delivery system and methods related thereto
EP3481418A4 (en) 2016-07-08 2020-03-11 American Gene Technologies International Inc. PRE-IMMUNIZATION AND IMMUNOTHERAPY OF HIV
US11583562B2 (en) 2016-07-21 2023-02-21 American Gene Technologies International Inc. Viral vectors for treating Parkinson's disease
KR20250103795A (ko) 2016-08-03 2025-07-07 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 아데노신 핵염기 편집제 및 그의 용도
US11661590B2 (en) 2016-08-09 2023-05-30 President And Fellows Of Harvard College Programmable CAS9-recombinase fusion proteins and uses thereof
WO2018039438A1 (en) 2016-08-24 2018-03-01 President And Fellows Of Harvard College Incorporation of unnatural amino acids into proteins using base editing
SG11201903089RA (en) 2016-10-14 2019-05-30 Harvard College Aav delivery of nucleobase editors
WO2018119359A1 (en) 2016-12-23 2018-06-28 President And Fellows Of Harvard College Editing of ccr5 receptor gene to protect against hiv infection
US11898179B2 (en) 2017-03-09 2024-02-13 President And Fellows Of Harvard College Suppression of pain by gene editing
EP3592381A1 (en) 2017-03-09 2020-01-15 President and Fellows of Harvard College Cancer vaccine
WO2018165629A1 (en) 2017-03-10 2018-09-13 President And Fellows Of Harvard College Cytosine to guanine base editor
WO2018176009A1 (en) 2017-03-23 2018-09-27 President And Fellows Of Harvard College Nucleobase editors comprising nucleic acid programmable dna binding proteins
JP2020512815A (ja) 2017-04-03 2020-04-30 アメリカン ジーン テクノロジーズ インターナショナル インコーポレイテッド フェニルケトン尿症を処置するための組成物および方法
CA3060570A1 (en) * 2017-04-21 2018-10-25 Seattle Children's Hospital (Dba Seattle Children's Research Institute Therapeutic genome editing in wiskott-aldrich syndrome and x-linked thrombocytopenia
WO2018209320A1 (en) 2017-05-12 2018-11-15 President And Fellows Of Harvard College Aptazyme-embedded guide rnas for use with crispr-cas9 in genome editing and transcriptional activation
WO2018232359A1 (en) 2017-06-16 2018-12-20 American Gene Technologies International Inc. Methods and compositions for the activation of tumor cytotoxicity via human gamma-delta t-cells
CN111801345A (zh) 2017-07-28 2020-10-20 哈佛大学的校长及成员们 使用噬菌体辅助连续进化(pace)的进化碱基编辑器的方法和组合物
EP3676376B1 (en) 2017-08-30 2025-01-15 President and Fellows of Harvard College High efficiency base editors comprising gam
US11795443B2 (en) 2017-10-16 2023-10-24 The Broad Institute, Inc. Uses of adenosine base editors
WO2019118949A1 (en) 2017-12-15 2019-06-20 The Broad Institute, Inc. Systems and methods for predicting repair outcomes in genetic engineering
US11422472B2 (en) * 2017-12-22 2022-08-23 Asml Netherlands B.V. Patterning process improvement involving optical aberration
JP2019198250A (ja) * 2018-05-14 2019-11-21 石原産業株式会社 ゲノム編集用キット
US12157760B2 (en) 2018-05-23 2024-12-03 The Broad Institute, Inc. Base editors and uses thereof
EP3820495A4 (en) 2018-07-09 2022-07-20 The Broad Institute Inc. RNA-PROGRAMMABLE EPIGENETIC RNA MODIFIERS AND THEIR USES
US20210261957A1 (en) * 2018-07-10 2021-08-26 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
US12084698B2 (en) 2018-07-13 2024-09-10 Kyoto University Virus-like particles and use thereof
WO2020022802A1 (ko) * 2018-07-25 2020-01-30 주식회사 툴젠 인위적인 유전자 조작을 통한 자가면역질환 치료
US20210403907A1 (en) 2018-09-18 2021-12-30 Vnv Newco Inc. Arc-based capsids and uses thereof
EP3852813A4 (en) 2018-09-18 2022-06-22 VNV Newco Inc. ARC-BASED CAPSIDS AND THEIR USES
WO2020092453A1 (en) 2018-10-29 2020-05-07 The Broad Institute, Inc. Nucleobase editors comprising geocas9 and uses thereof
US11352646B2 (en) 2018-11-05 2022-06-07 American Gene Technologies International Inc. Vector system for expressing regulatory RNA
WO2020097344A1 (en) * 2018-11-08 2020-05-14 Arizona Board of Regents on Behalf Arizona State University Synthetic immunomodulation with a crispr super-repressor in vivo
EP3880717A4 (en) * 2018-11-16 2022-11-23 The Regents of The University of California Compositions and methods for delivering crispr/cas effector polypeptides
US12351837B2 (en) 2019-01-23 2025-07-08 The Broad Institute, Inc. Supernegatively charged proteins and uses thereof
WO2020160418A1 (en) * 2019-01-31 2020-08-06 Board Of Regents Of The University Of Nebraska Virus-like particles and methods of use thereof
EP3942043A2 (en) 2019-03-19 2022-01-26 The Broad Institute, Inc. Methods and compositions for editing nucleotide sequences
WO2020214842A1 (en) 2019-04-17 2020-10-22 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
WO2021030165A1 (en) 2019-08-09 2021-02-18 University Of Utah Research Foundation "trpswitch" − a step function chemo-optogenetic ligand
AU2020341454A1 (en) 2019-09-03 2022-03-10 Sana Biotechnology, Inc. CD24-associated particles and related methods and uses thereof
EP4028522A1 (en) * 2019-09-09 2022-07-20 Scribe Therapeutics Inc. Compositions and methods for the targeting of sod1
AU2020344553A1 (en) * 2019-09-09 2022-04-07 Scribe Therapeutics Inc. Compositions and methods for use in immunotherapy
EP4031561A1 (en) * 2019-09-20 2022-07-27 The Broad Institute, Inc. Compositions and methods for delivering cargo to a target cell
EP4041867A1 (en) 2019-10-09 2022-08-17 Institut National de la Santé et de la Recherche Médicale (INSERM) T cells modified to express mutated cxcr4 or partially deleted and uses thereof
US12435330B2 (en) 2019-10-10 2025-10-07 The Broad Institute, Inc. Methods and compositions for prime editing RNA
EP4069845A1 (en) 2019-12-06 2022-10-12 Scribe Therapeutics Inc. Compositions and methods for the targeting of rhodopsin
EP4069716A1 (en) * 2019-12-06 2022-10-12 Scribe Therapeutics Inc. Particle delivery systems
WO2021113769A1 (en) * 2019-12-07 2021-06-10 Scribe Therapeutics Inc. Compositions and methods for the targeting of htt
BR112022022603A2 (pt) 2020-05-08 2023-01-17 Broad Inst Inc Métodos e composições para edição simultânea de ambas as fitas de sequência alvo de nucleotídeos de fita dupla
CN116670154A (zh) 2020-07-24 2023-08-29 总医院公司 增强的病毒样颗粒及使用其递送至细胞的方法
WO2022251712A1 (en) 2021-05-28 2022-12-01 Sana Biotechnology, Inc. Lipid particles containing a truncated baboon endogenous retrovirus (baev) envelope glycoprotein and related methods and uses
US12565647B2 (en) 2021-05-28 2026-03-03 The Regents Of The University Of California Compositions and methods for targeted delivery of CRISPR-Cas effector polypeptides and transgenes
EP4351660A2 (en) * 2021-06-09 2024-04-17 Scribe Therapeutics Inc. Particle delivery systems
KR20240095525A (ko) 2021-09-21 2024-06-25 스크라이브 테라퓨틱스 인크. 조작된 casx 억제 시스템
US20250230511A1 (en) 2021-10-29 2025-07-17 Sana Biotechnology, Inc. Methods and reagents for amplifying viral vector nucleic acid products
US20250064979A1 (en) * 2021-12-03 2025-02-27 The Broad Institute, Inc. Self-assembling virus-like particles for delivery of prime editors and methods of making and using same
WO2023102537A2 (en) * 2021-12-03 2023-06-08 The Broad Institute, Inc. Self-assembling virus-like particles for delivery of nucleic acid programmable fusion proteins and methods of making and using same
CA3239381A1 (en) * 2021-12-03 2023-06-08 David R. Liu Compositions and methods for efficient in vivo delivery
WO2023114949A1 (en) 2021-12-16 2023-06-22 Sana Biotechnology, Inc. Methods and systems of particle production
WO2023115041A1 (en) 2021-12-17 2023-06-22 Sana Biotechnology, Inc. Modified paramyxoviridae attachment glycoproteins
US20250059239A1 (en) 2021-12-17 2025-02-20 Sana Biotechnology, Inc. Modified paramyxoviridae fusion glycoproteins
WO2023133595A2 (en) 2022-01-10 2023-07-13 Sana Biotechnology, Inc. Methods of ex vivo dosing and administration of lipid particles or viral vectors and related systems and uses
EP4472646A1 (en) 2022-02-01 2024-12-11 Sana Biotechnology, Inc. Cd3-targeted lentiviral vectors and uses thereof
WO2023150647A1 (en) 2022-02-02 2023-08-10 Sana Biotechnology, Inc. Methods of repeat dosing and administration of lipid particles or viral vectors and related systems and uses
US20260027145A1 (en) 2022-07-27 2026-01-29 Sana Biotechnology, Inc. Methods of transduction using a viral vector and inhibitors of antiviral restriction factors
US20260055146A1 (en) 2022-08-24 2026-02-26 Sana Biotechnology, Inc. Delivery of heterologous proteins
WO2024064838A1 (en) 2022-09-21 2024-03-28 Sana Biotechnology, Inc. Lipid particles comprising variant paramyxovirus attachment glycoproteins and uses thereof
WO2024081820A1 (en) 2022-10-13 2024-04-18 Sana Biotechnology, Inc. Viral particles targeting hematopoietic stem cells
WO2024119157A1 (en) 2022-12-02 2024-06-06 Sana Biotechnology, Inc. Lipid particles with cofusogens and methods of producing and using the same
WO2024197008A2 (en) * 2023-03-20 2024-09-26 The Broad Institute, Inc. Nuclease-guided non-ltr retrotransposons and uses thereof
WO2024220574A1 (en) 2023-04-18 2024-10-24 Sana Biotechnology, Inc. Universal protein g fusogens and adapter systems thereof and related lipid particles and uses
WO2024220560A1 (en) 2023-04-18 2024-10-24 Sana Biotechnology, Inc. Engineered protein g fusogens and related lipid particles and methods thereof
AU2024274792A1 (en) 2023-05-23 2025-10-23 Sana Biotechnology, Inc. Tandem fusogens and related lipid particles
WO2025137322A1 (en) * 2023-12-20 2025-06-26 The Board Of Trustees Of The Leland Stanford Junior University Secreted particle information transfer (spit) system for genetic engineering
WO2025151838A1 (en) 2024-01-12 2025-07-17 Sana Biotechnology, Inc. Safety switches to control in vitro and in vivo proliferation of cell therapy products
WO2025184529A1 (en) 2024-03-01 2025-09-04 Sana Biotechnology, Inc. Viral particles with fusogen display and related compositions and methods
CN118547011A (zh) * 2024-06-05 2024-08-27 广州达博生物制品有限公司 一种稳定高表达人源悬浮细胞系的构建方法
WO2025262314A1 (en) 2024-06-21 2025-12-26 Institut National De La Sante Et De La Recherche Medicale Methods and products for prime editing genetic engineering
WO2025262311A1 (en) 2024-06-21 2025-12-26 Institut National De La Sante Et De La Recherche Medicale Methods and products for prime editing genetic engineering

Citations (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2014131833A1 (en) 2013-02-27 2014-09-04 Helmholtz Zentrum München Deutsches Forschungszentrum Für Gesundheit Und Umwelt (Gmbh) Gene editing in the oocyte by cas9 nucleases
WO2014200659A1 (en) 2013-06-11 2014-12-18 Clontech Laboratories, Inc. Protein enriched microvesicles and methods of making and using the same
JP2015505299A (ja) 2011-11-11 2015-02-19 ヴァリエーション バイオテクノロジーズ インコーポレイテッド サイトメガロウイルスの治療のための組成物及び方法
JP2015523856A (ja) 2012-05-25 2015-08-20 ザ リージェンツ オブ ザ ユニバーシティ オブ カリフ Ｒｎａ依存性標的ｄｎａ修飾およびｒｎａ依存性転写調節のための方法および組成物

Family Cites Families (20)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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
GB9227068D0 (en) 1992-12-29 1993-02-24 British Bio Technology Novel proteinaceous particles
FR2716459B1 (fr) 1994-02-22 1996-05-10 Univ Paris Curie Système hôte-vecteur utilisable en thérapie génique.
US6204059B1 (en) 1994-06-30 2001-03-20 University Of Pittsburgh AAV capsid vehicles for molecular transfer
EP1980617A1 (en) * 1998-12-31 2008-10-15 Novartis Vaccines and Diagnostics, Inc. Improved expression of HIV polypeptides and production of virus-like particles
US9296790B2 (en) * 2008-10-03 2016-03-29 The United States Of America, As Represented By The Secretary, Department Of Health And Human Services Methods and compositions for protein delivery
AU2009319979B2 (en) * 2008-11-03 2016-10-20 Takeda Vaccines, Inc. Improved methods for isolating enveloped virus-based VLPs free of infectious agents
DE102010025907A1 (de) 2010-07-02 2012-01-05 Robert Bosch Gmbh Wellenenergiewandler zur Umwandlung von kinetischer Energie in elektrische Energie
WO2013163628A2 (en) 2012-04-27 2013-10-31 Duke University Genetic correction of mutated genes
CN114634950A (zh) 2012-12-12 2022-06-17 布罗德研究所有限公司 用于序列操纵的crispr-cas组分系统、方法以及组合物
HUE056760T2 (hu) * 2013-07-11 2022-03-28 Modernatx Inc A CRISPR-hez kapcsolódó fehérjéket és a szintetikus SGRNS-ket kódoló szintetikus polinukleotidokat tartalmazó készítmények és felhasználási módjaik
CN111206032B (zh) * 2013-12-12 2024-07-19 布罗德研究所有限公司 用于基因组编辑的crispr-cas系统和组合物的递送、用途和治疗应用
WO2015089247A1 (en) 2013-12-12 2015-06-18 Dow Agrosciences Llc Selective weed control with halauxifen
WO2015089486A2 (en) 2013-12-12 2015-06-18 The Broad Institute Inc. Systems, methods and compositions for sequence manipulation with optimized functional crispr-cas systems
KR20170005494A (ko) * 2014-05-30 2017-01-13 더 보드 어브 트러스티스 어브 더 리랜드 스탠포드 주니어 유니버시티 잠복 바이러스 감염에 대한 치료제를 전달하는 조성물 및 방법
WO2016037164A1 (en) * 2014-09-07 2016-03-10 Selecta Biosciences, Inc. Methods and compositions for attenuating gene expression modulating anti-viral transfer vector immune responses
JP6823593B2 (ja) * 2014-11-06 2021-02-03 イー・アイ・デュポン・ドウ・ヌムール・アンド・カンパニーＥ．Ｉ．Ｄｕ Ｐｏｎｔ Ｄｅ Ｎｅｍｏｕｒｓ Ａｎｄ Ｃｏｍｐａｎｙ Ｒｎａ誘導型エンドヌクレアーゼの細胞へのペプチド媒介輸送
WO2017001570A2 (en) 2015-06-30 2017-01-05 Ethris Gmbh Atp-binding cassette family coding polyribonucleotides and formulations thereof
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
CA3040071A1 (en) * 2015-12-17 2017-06-22 The Penn State Research Foundation Paramyxovirus virus-like particles as protein delivery vehicles

Patent Citations (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2015505299A (ja) 2011-11-11 2015-02-19 ヴァリエーション バイオテクノロジーズ インコーポレイテッド サイトメガロウイルスの治療のための組成物及び方法
JP2015523856A (ja) 2012-05-25 2015-08-20 ザ リージェンツ オブ ザ ユニバーシティ オブ カリフ Ｒｎａ依存性標的ｄｎａ修飾およびｒｎａ依存性転写調節のための方法および組成物
WO2014131833A1 (en) 2013-02-27 2014-09-04 Helmholtz Zentrum München Deutsches Forschungszentrum Für Gesundheit Und Umwelt (Gmbh) Gene editing in the oocyte by cas9 nucleases
WO2014200659A1 (en) 2013-06-11 2014-12-18 Clontech Laboratories, Inc. Protein enriched microvesicles and methods of making and using the same

Non-Patent Citations (4)

* Cited by examiner, † Cited by third party
Title
CAI, Y., et al.，"Targeted genome editing by lentiviral protein transduction of zinc-finger and TAL-effector nucleases."，eLife，2014年，Vol. 3，e01911(pp.1-19)，DOI:10.7554/elife.01911
HORII, T., et al.，"Efficient generation of knockin mice by CRISPR/Cas system."，Exp. Anim.，2015年，vol.64, supplement，p.S31(O-7)
KABADI, A.M., et al.，"Multiplex CRISPR/Cas9-based genome engineering from a single lentiviral vector."，Nucleic Acids Research，2014年，Vol. 42, No. 19，e147，Published online 13 August 2014, doi: 10.1093/nar/gku749
KIM, S., et al.，"Highly efficient RNA-guided genome editing in human cells via delivery of purified Cas9 ribonucleoproteins."，Genome Res.，2014年，vol.24, no.6，p.1012-1019, Supplemental Information

Cited By (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2022046680A (ja) * 2015-10-20 2022-03-23 アンスティチュ ナショナル ドゥ ラ サンテ エ ドゥ ラ ルシェルシュ メディカル 遺伝子操作のための方法及び製品
JP7621930B2 (ja) 2015-10-20 2025-01-27 アンスティチュ ナショナル ドゥ ラ サンテ エ ドゥ ラ ルシェルシュ メディカル 遺伝子操作のための方法及び製品

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