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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    • 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
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    • 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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  • Health & Medical Sciences (AREA)
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  • Bioinformatics & Cheminformatics (AREA)
  • Biomedical Technology (AREA)
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  • General Engineering & Computer Science (AREA)
  • Biotechnology (AREA)
  • Biochemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Microbiology (AREA)
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  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Virology (AREA)
  • Physics & Mathematics (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Immunology (AREA)
  • Crystallography & Structural Chemistry (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)

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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)

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EP15306678 2015-10-20
EP15306678.2 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
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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 (87)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2010045659A1 (en) 2008-10-17 2010-04-22 American Gene Technologies International Inc. 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
US9388430B2 (en) 2013-09-06 2016-07-12 President And Fellows Of Harvard College Cas9-recombinase fusion proteins and uses thereof
US9737604B2 (en) 2013-09-06 2017-08-22 President And Fellows Of Harvard College Use of cationic lipids to deliver CAS9
US11053481B2 (en) 2013-12-12 2021-07-06 President And Fellows Of Harvard College Fusions of Cas9 domains and nucleic acid-editing domains
AU2015298571B2 (en) 2014-07-30 2020-09-03 President And Fellows Of Harvard College Cas9 proteins including ligand-dependent inteins
EP3319631A4 (en) 2015-07-08 2019-01-09 American Gene Technologies International Inc. HIV PREMUNICATION AND IMMUNOTHERAPY
US12043852B2 (en) 2015-10-23 2024-07-23 President And Fellows Of Harvard College Evolved Cas9 proteins for gene editing
US10137144B2 (en) 2016-01-15 2018-11-27 American Gene Technologies International Inc. Methods and compositions for the activation of gamma-delta T-cells
CA3011529A1 (en) 2016-01-15 2017-07-20 American Gene Technologies International Inc. Methods and compositions for the activation of gamma-delta t-cells
US10888613B2 (en) 2016-02-08 2021-01-12 American Gene Technologies International Inc. Method of producing cells resistant to HIV infection
ES2911448T3 (es) 2016-03-09 2022-05-19 American Gene Tech Int Inc Vectores combinados y métodos para el tratamiento del cáncer
EP3468617A4 (en) 2016-06-08 2020-01-22 American Gene Technologies International Inc. INTEGRATED VIRAL ADMINISTRATION SYSTEM AND RELATED METHODS
IL300730A (en) 2016-07-08 2023-04-01 American Gene Tech Int Inc Pre-HIV vaccine and immunotherapy
JP7176756B2 (ja) 2016-07-21 2022-11-22 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 パーキンソン病を処置するためのウイルスベクター
KR20250103795A (ko) 2016-08-03 2025-07-07 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 아데노신 핵염기 편집제 및 그의 용도
CN109804066A (zh) 2016-08-09 2019-05-24 哈佛大学的校长及成员们 可编程cas9-重组酶融合蛋白及其用途
US11542509B2 (en) 2016-08-24 2023-01-03 President And Fellows Of Harvard College Incorporation of unnatural amino acids into proteins using base editing
AU2017342543B2 (en) 2016-10-14 2024-06-27 President And Fellows Of 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
EP3592853A1 (en) 2017-03-09 2020-01-15 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
KR20190127797A (ko) 2017-03-10 2019-11-13 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 시토신에서 구아닌으로의 염기 편집제
CA3057192A1 (en) 2017-03-23 2018-09-27 President And Fellows Of Harvard College Nucleobase editors comprising nucleic acid programmable dna binding proteins
US11820999B2 (en) 2017-04-03 2023-11-21 American Gene Technologies International Inc. Compositions and methods for treating phenylketonuria
CN110785179A (zh) * 2017-04-21 2020-02-11 西雅图儿童医院（Dba西雅图儿童研究所） Wiskott-Aldrich综合征和X连锁血小板减少症中的治疗性基因组编辑
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
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
WO2019139645A2 (en) 2017-08-30 2019-07-18 President And Fellows Of Harvard College High efficiency base editors comprising gam
CA3082251A1 (en) 2017-10-16 2019-04-25 The Broad Institute, Inc. Uses of adenosine base editors
EP3724214A4 (en) 2017-12-15 2021-09-01 The Broad Institute Inc. SYSTEMS AND METHODS FOR PREDICTING REPAIR RESULTS IN GENETIC ENGINEERING
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
WO2019226953A1 (en) 2018-05-23 2019-11-28 The Broad Institute, Inc. Base editors and uses thereof
BR112021000408A2 (pt) * 2018-07-10 2021-06-29 Alia Therapeutics S.R.L. vesículas para dispensação sem traço de moléculas de rna guia e/ou complexo(s) de nuclease guiada por rna/molécula de rna guia e método de produção das mesmas
JP7306721B2 (ja) * 2018-07-13 2023-07-11 国立大学法人京都大学 ウイルス様粒子及びその使用
KR20200011899A (ko) * 2018-07-25 2020-02-04 주식회사 툴젠 인위적인 유전자 조작을 통한 자가면역질환 치료
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
US12281338B2 (en) 2018-10-29 2025-04-22 The Broad Institute, Inc. Nucleobase editors comprising GeoCas9 and uses thereof
WO2020097049A1 (en) * 2018-11-05 2020-05-14 American Gene Technologies International Inc. Vector system for expressing regulatory rna
CN113330023A (zh) * 2018-11-08 2021-08-31 代表亚利桑那州立大学的亚利桑那校董会 体内具有crispr超级阻遏子的合成免疫调控
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
US20220194990A1 (en) * 2019-01-31 2022-06-23 Board Of Regents Of The University Of Nebraska Virus-like particles and methods of use thereof
DE112020001306T5 (de) 2019-03-19 2022-01-27 Massachusetts Institute Of Technology Verfahren und zusammensetzungen zur editierung von nukleotidsequenzen
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
US20220280644A1 (en) 2019-08-09 2022-09-08 University Of Utah Research Foundation "TRPswitch" - A STEP FUNCTION CHEMO-OPTOGENETIC LIGAND
EP4025698A1 (en) 2019-09-03 2022-07-13 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
JP7696335B2 (ja) * 2019-09-09 2025-06-20 スクライブ・セラピューティクス・インコーポレイテッド 免疫療法に使用するための組成物および方法
JP2022548308A (ja) * 2019-09-20 2022-11-17 ザ・ブロード・インスティテュート・インコーポレイテッド カーゴを標的細胞に送達するための組成物及び方法
WO2021069593A1 (en) 2019-10-09 2021-04-15 INSERM (Institut National de la Santé et de la Recherche Médicale) 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
KR20220113442A (ko) * 2019-12-06 2022-08-12 스크라이브 테라퓨틱스 인크. 입자 전달 시스템
EP4069845A1 (en) * 2019-12-06 2022-10-12 Scribe Therapeutics Inc. Compositions and methods for the targeting of rhodopsin
WO2021113769A1 (en) * 2019-12-07 2021-06-10 Scribe Therapeutics Inc. Compositions and methods for the targeting of htt
JP2023525304A (ja) 2020-05-08 2023-06-15 ザ ブロード インスティテュート，インコーポレーテッド 標的二本鎖ヌクレオチド配列の両鎖同時編集のための方法および組成物
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
US20220403379A1 (en) * 2021-05-28 2022-12-22 The Regents Of The University Of California Compositions and methods for targeted delivery of crispr-cas effector polypeptides and transgenes
JP2024521811A (ja) 2021-05-28 2024-06-04 サナ バイオテクノロジー，インコーポレイテッド 短縮型ヒヒ内在性レトロウイルス（ＢａＥＶ）エンベロープ糖タンパク質を含む脂質粒子、ならびに関連する方法及び使用
EP4351660A2 (en) 2021-06-09 2024-04-17 Scribe Therapeutics Inc. Particle delivery systems
US20250230511A1 (en) 2021-10-29 2025-07-17 Sana Biotechnology, Inc. Methods and reagents for amplifying viral vector nucleic acid products
AU2022400961A1 (en) * 2021-12-03 2024-05-30 President And Fellows Of Harvard College Self-assembling virus-like particles for delivery of nucleic acid programmable fusion proteins and methods of making and using same
KR20240130158A (ko) * 2021-12-03 2024-08-28 더 브로드 인스티튜트, 인코퍼레이티드 효율적인 생체내 전달을 위한 조성물 및 방법
IL313146A (en) * 2021-12-03 2024-07-01 Harvard College Self-assembling virus-like particles for delivery of prime editors and methods of making and using same
US20250059560A1 (en) 2021-12-16 2025-02-20 Sana Biotechnology, Inc. Methods and systems of particle production
EP4448549A2 (en) 2021-12-17 2024-10-23 Sana Biotechnology, Inc. Modified paramyxoviridae fusion glycoproteins
WO2023115041A1 (en) 2021-12-17 2023-06-22 Sana Biotechnology, Inc. Modified paramyxoviridae attachment glycoproteins
EP4463135A2 (en) 2022-01-10 2024-11-20 Sana Biotechnology, Inc. Methods of ex vivo dosing and administration of lipid particles or viral vectors and related systems and uses
US20250152709A1 (en) 2022-02-01 2025-05-15 Sana Biotechnology, Inc. Cd3-targeted lentiviral vectors and uses thereof
US20250144235A1 (en) 2022-02-02 2025-05-08 Sana Biotechnology, Inc. Methods of repeat dosing and administration of lipid particles or viral vectors and related systems and uses
WO2024026377A1 (en) 2022-07-27 2024-02-01 Sana Biotechnology, Inc. Methods of transduction using a viral vector and inhibitors of antiviral restriction factors
WO2024044655A1 (en) 2022-08-24 2024-02-29 Sana Biotechnology, Inc. Delivery of heterologous proteins
EP4590688A1 (en) 2022-09-21 2025-07-30 Sana Biotechnology, Inc. Lipid particles comprising variant paramyxovirus attachment glycoproteins and uses thereof
EP4602174A1 (en) 2022-10-13 2025-08-20 Sana Biotechnology, Inc. Viral particles targeting hematopoietic stem cells
EP4627096A1 (en) 2022-12-02 2025-10-08 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
WO2024220560A1 (en) 2023-04-18 2024-10-24 Sana Biotechnology, Inc. Engineered protein g fusogens and related lipid particles and methods 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
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

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
CA2742295A1 (en) * 2008-11-03 2010-06-03 Ligocyte Pharmaceuticals, 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
EP4234696A3 (en) 2012-12-12 2023-09-06 The Broad Institute Inc. Crispr-cas component systems, methods and compositions for sequence manipulation
DK3019619T3 (da) * 2013-07-11 2021-10-11 Modernatx Inc Sammensætninger, der omfatter syntetiske polynukleotider, som koder for crispr-beslægtede proteiner, og syntetiske sgrna'er, og anvendelsesfremgangsmåder
EP3080271B1 (en) 2013-12-12 2020-02-12 The Broad Institute, Inc. Systems, methods and compositions for sequence manipulation with optimized functional crispr-cas systems
BR112016013201B1 (pt) * 2013-12-12 2023-01-31 The Broad Institute, Inc. Uso de uma composição compreendendo um sistema crispr-cas no tratamento de uma doença genética ocular
JP2016539998A (ja) 2013-12-12 2016-12-22 ダウ アグロサイエンシィズ エルエルシー ハラウキシフェンによる選択的雑草防除
US20160060655A1 (en) * 2014-05-30 2016-03-03 The Board Of Trustees Of The Leland Stanford Junior University Compositions and methods to treat latent viral infections
WO2016037163A1 (en) * 2014-09-07 2016-03-10 Selecta Biosciences, Inc. Methods and compositions for attenuating gene therapy anti-viral transfer vector immune responses
KR102424721B1 (ko) * 2014-11-06 2022-07-25 이 아이 듀폰 디 네모아 앤드 캄파니 Rna-유도 엔도뉴클레아제의 세포 내로의 펩티드 매개성 전달
CN107849565A (zh) 2015-06-30 2018-03-27 埃泽瑞斯公司 Atp结合盒家族的编码多核糖核苷酸及其制剂
JP7059179B2 (ja) 2015-10-20 2022-04-25 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
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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