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

Info

Publication number
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
Authority
JP
Japan
Prior art keywords
virus
derived
particles
protein
cas
Prior art date
Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
Active
Application number
JP2018520155A
Other languages
English (en)
Japanese (ja)
Other versions
JP2018531023A6 (ja
JP2018531023A5 (https=
JP2018531023A (ja
Inventor
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
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Centre National de la Recherche Scientifique CNRS
Original Assignee
Centre National de la Recherche Scientifique CNRS
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Centre National de la Recherche Scientifique CNRS filed Critical Centre National de la Recherche Scientifique CNRS
Publication of JP2018531023A publication Critical patent/JP2018531023A/ja
Publication of JP2018531023A6 publication Critical patent/JP2018531023A6/ja
Publication of JP2018531023A5 publication Critical patent/JP2018531023A5/ja
Priority to JP2021212300A priority Critical patent/JP7621930B2/ja
Application granted granted Critical
Publication of JP7059179B2 publication Critical patent/JP7059179B2/ja
Priority to JP2025005188A priority patent/JP2025063185A/ja
Active legal-status Critical Current
Anticipated expiration legal-status Critical

Links

Images

Classifications

    • 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
    • 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
    • C12N15/10Processes for the isolation, preparation or purification of DNA or RNA
    • C12N15/102Mutagenizing nucleic acids
    • 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
    • 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
    • C12N15/90Stable introduction of foreign DNA into chromosome
    • 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
    • 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
    • C12N9/00Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14Hydrolases (3)
    • C12N9/16Hydrolases (3) acting on ester bonds (3.1)
    • C12N9/22Ribonucleases [RNase]; Deoxyribonucleases [DNase]
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K2319/00Fusion polypeptide
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K2319/00Fusion polypeptide
    • C07K2319/40Fusion polypeptide containing a tag for immunodetection, or an epitope for immunisation
    • C07K2319/43Fusion polypeptide containing a tag for immunodetection, or an epitope for immunisation containing a FLAG-tag
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K2319/00Fusion polypeptide
    • C07K2319/50Fusion polypeptide containing protease site
    • 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
    • C12N2310/20Type of nucleic acid involving clustered regularly interspaced short palindromic repeats [CRISPR]
    • 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]
    • 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
    • C12N2740/10041Use of virus, viral particle or viral elements as a vector
    • C12N2740/10042Use of virus, viral particle or viral elements as a vector virus or viral particle as vehicle, e.g. encapsulating small organic molecule
    • 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
    • C12N2740/13011Gammaretrovirus, e.g. murine leukeamia virus
    • C12N2740/13023Virus like particles [VLP]
    • 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
    • C12N2740/13011Gammaretrovirus, e.g. murine leukeamia virus
    • C12N2740/13041Use of virus, viral particle or viral elements as a vector
    • C12N2740/13042Use of virus, viral particle or viral elements as a vector virus or viral particle as vehicle, e.g. encapsulating small organic molecule
    • 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
    • C12N2800/00Nucleic acids vectors
    • C12N2800/80Vectors containing sites for inducing double-stranded breaks, e.g. meganuclease restriction sites

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • Zoology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • Biomedical Technology (AREA)
  • Molecular Biology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biochemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Microbiology (AREA)
  • Biophysics (AREA)
  • Medicinal Chemistry (AREA)
  • Physics & Mathematics (AREA)
  • Virology (AREA)
  • Plant Pathology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (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 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

Related Child Applications (1)

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

Publications (4)

Publication Number Publication Date
JP2018531023A JP2018531023A (ja) 2018-10-25
JP2018531023A6 JP2018531023A6 (ja) 2018-12-13
JP2018531023A5 JP2018531023A5 (https=) 2019-11-28
JP7059179B2 true JP7059179B2 (ja) 2022-04-25

Family

ID=55024029

Family Applications (3)

Application Number Title Priority Date Filing Date
JP2018520155A Active JP7059179B2 (ja) 2015-10-20 2016-10-20 遺伝子操作のための方法及び製品
JP2021212300A Active JP7621930B2 (ja) 2015-10-20 2021-12-27 遺伝子操作のための方法及び製品
JP2025005188A Pending JP2025063185A (ja) 2015-10-20 2025-01-15 遺伝子操作のための方法及び製品

Family Applications After (2)

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

Country Status (3)

Country Link
US (6) US10968253B2 (https=)
JP (3) JP7059179B2 (https=)
WO (1) WO2017068077A1 (https=)

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 (93)

* 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
US10323236B2 (en) 2011-07-22 2019-06-18 President And Fellows Of Harvard College Evaluation and improvement of nuclease cleavage specificity
US9163284B2 (en) 2013-08-09 2015-10-20 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
US9322037B2 (en) 2013-09-06 2016-04-26 President And Fellows Of Harvard College Cas9-FokI fusion proteins and uses thereof
US9228207B2 (en) 2013-09-06 2016-01-05 President And Fellows Of Harvard College Switchable gRNAs comprising aptamers
US9526784B2 (en) 2013-09-06 2016-12-27 President And Fellows Of Harvard College Delivery system for functional nucleases
US20150165054A1 (en) 2013-12-12 2015-06-18 President And Fellows Of Harvard College Methods for correcting caspase-9 point mutations
US10077453B2 (en) 2014-07-30 2018-09-18 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
IL310721B2 (en) 2015-10-23 2025-11-01 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
CN116064678A (zh) 2016-01-15 2023-05-05 美国基因技术国际有限公司 用于活化γ-δT细胞的方法和组合物
WO2017139065A1 (en) 2016-02-08 2017-08-17 American Gene Technologies International Inc. Hiv vaccination and immunotherapy
WO2017156311A2 (en) 2016-03-09 2017-09-14 American Gene Technologies International Inc. Combination vectors and methods for treating cancer
EP3468617A4 (en) 2016-06-08 2020-01-22 American Gene Technologies International Inc. SYSTEM FOR THE NON-INTEGRAL VIRAL ISSUE AND PROCEDURE RELATING TO IT
AU2017292582C1 (en) 2016-07-08 2021-11-11 American Gene Technologies International Inc. HIV pre-immunization and immunotherapy
EP3487507A4 (en) 2016-07-21 2020-04-08 American Gene Technologies International, Inc. VIRAL VECTORS FOR THE TREATMENT OF PARKINSON'S DISEASE
CN110214183A (zh) 2016-08-03 2019-09-06 哈佛大学的校长及成员们 腺苷核碱基编辑器及其用途
WO2018031683A1 (en) 2016-08-09 2018-02-15 President And Fellows Of Harvard College Programmable cas9-recombinase fusion proteins and uses thereof
US11542509B2 (en) 2016-08-24 2023-01-03 President And Fellows Of Harvard College Incorporation of unnatural amino acids into proteins using base editing
KR102622411B1 (ko) 2016-10-14 2024-01-10 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 핵염기 에디터의 aav 전달
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
US12390514B2 (en) 2017-03-09 2025-08-19 President And Fellows Of Harvard College Cancer vaccine
US11542496B2 (en) 2017-03-10 2023-01-03 President And Fellows Of Harvard College Cytosine to guanine base editor
KR20240116572A (ko) 2017-03-23 2024-07-29 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 핵산 프로그램가능한 dna 결합 단백질을 포함하는 핵염기 편집제
US11820999B2 (en) 2017-04-03 2023-11-21 American Gene Technologies International Inc. Compositions and methods for treating phenylketonuria
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
US11560566B2 (en) 2017-05-12 2023-01-24 President And Fellows Of Harvard College Aptazyme-embedded guide RNAs for use with CRISPR-Cas9 in genome editing and transcriptional activation
JP2020524996A (ja) 2017-06-16 2020-08-27 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 ヒトガンマデルタt細胞を介する腫瘍細胞傷害性の活性化のための方法および組成物
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
KR20250107288A (ko) 2017-10-16 2025-07-11 더 브로드 인스티튜트, 인코퍼레이티드 아데노신 염기 편집제의 용도
US12406749B2 (en) 2017-12-15 2025-09-02 The Broad Institute, Inc. Systems and methods for predicting repair outcomes in genetic engineering
CN111512236B (zh) * 2017-12-22 2023-01-24 Asml荷兰有限公司 涉及光学像差的图案化过程改进
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
US12522807B2 (en) 2018-07-09 2026-01-13 The Broad Institute, Inc. RNA programmable epigenetic RNA modifiers and uses thereof
CA3105925A1 (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
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
AU2019343045A1 (en) 2018-09-18 2021-05-13 Vnv Newco Inc. ARC-based capsids and uses thereof
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
SG11202104802RA (en) * 2018-11-08 2021-06-29 Arizona Board Of Regents On Behalf Arizona State Univ Synthetic immunomodulation with a crispr super-repressor in vivo
WO2020102709A1 (en) * 2018-11-16 2020-05-22 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
WO2020191233A1 (en) 2019-03-19 2020-09-24 The Broad Institute, Inc. Methods and compositions for editing nucleotide sequences
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
US20220280644A1 (en) 2019-08-09 2022-09-08 University Of Utah Research Foundation "TRPswitch" - A STEP FUNCTION CHEMO-OPTOGENETIC LIGAND
CA3152525A1 (en) 2019-09-03 2021-03-11 Sana Biotechnology, Inc. Cd24-associated particles and related methods and uses thereof
WO2021050593A1 (en) * 2019-09-09 2021-03-18 Scribe Therapeutics Inc. Compositions and methods for the targeting of sod1
WO2021050601A1 (en) * 2019-09-09 2021-03-18 Scribe Therapeutics Inc. Compositions and methods for use in immunotherapy
AU2020348872A1 (en) * 2019-09-20 2022-04-14 Massachusetts Institute Of Technology Compositions and methods for delivering cargo to a target cell
JP2022552270A (ja) 2019-10-09 2022-12-15 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 変異cxcr4を発現するように改変された又は部分的に欠失されたt細胞及びその使用
US12435330B2 (en) 2019-10-10 2025-10-07 The Broad Institute, Inc. Methods and compositions for prime editing RNA
EP4069716A1 (en) * 2019-12-06 2022-10-12 Scribe Therapeutics Inc. Particle delivery systems
WO2021113763A1 (en) * 2019-12-06 2021-06-10 Scribe Therapeutics Inc. Compositions and methods for the targeting of rhodopsin
EP4069846A1 (en) * 2019-12-07 2022-10-12 Scribe Therapeutics Inc. Compositions and methods for the targeting of htt
IL297761A (en) 2020-05-08 2022-12-01 Broad Inst Inc Methods and compositions for simultaneously editing two helices of a designated double-helix nucleotide sequence
CA3189601A1 (en) * 2020-07-24 2022-01-27 The General Hospital Corporation Enhanced virus-like particles and methods of use thereof for delivery to cells
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
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
WO2022261149A2 (en) 2021-06-09 2022-12-15 Scribe Therapeutics Inc. Particle delivery systems
GB2625500B (en) 2021-09-21 2026-04-01 Scribe Therapeutics Inc Engineered CasX repressor systems
US20250230511A1 (en) 2021-10-29 2025-07-17 Sana Biotechnology, Inc. Methods and reagents for amplifying viral vector nucleic acid products
EP4441074A2 (en) * 2021-12-03 2024-10-09 The Broad Institute, Inc. Compositions and methods for efficient in vivo delivery
WO2023102538A1 (en) * 2021-12-03 2023-06-08 The Broad Institute, Inc. Self-assembling virus-like particles for delivery of prime editors and methods of making and using same
EP4441073A2 (en) * 2021-12-03 2024-10-09 The Broad Institute, Inc. Self-assembling virus-like particles for delivery of nucleic acid programmable fusion proteins and methods of making and using same
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
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
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
WO2024026377A1 (en) 2022-07-27 2024-02-01 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
EP4698666A1 (en) 2023-04-18 2026-02-25 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
EP4716750A1 (en) 2023-05-23 2026-04-01 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
AU2221600A (en) * 1998-12-31 2000-07-31 Chiron Corporation 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
JP5683476B2 (ja) * 2008-11-03 2015-03-11 タケダ ヴァクシーンズ, インコーポレイテッド 感染因子を含まないエンベロープウイルスベースvlpを単離するための改良法
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
EP2840140B2 (en) 2012-12-12 2023-02-22 The Broad Institute, Inc. Crispr-Cas based method for mutation of prokaryotic cells
CA2917348A1 (en) * 2013-07-11 2015-01-15 Moderna Therapeutics, Inc. Compositions comprising synthetic polynucleotides encoding crispr related proteins and synthetic sgrnas and methods of use
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
JP7103750B2 (ja) * 2013-12-12 2022-07-20 ザ・ブロード・インスティテュート・インコーポレイテッド ゲノム編集のためのCRISPR-Cas系及び組成物の送達、使用及び治療適用
MX2016007655A (es) 2013-12-12 2016-10-04 Dow Agrosciences Llc Control selectivo de malezas con halauxifen.
WO2015184259A1 (en) * 2014-05-30 2015-12-03 The Board Of Trustees Of The Leland Stanford Junior University Compositions and methods to treat latent viral infections
JP7242180B2 (ja) * 2014-09-07 2023-03-20 セレクタ バイオサイエンシーズ インコーポレーテッド 抗ウイルス導入ベクター免疫応答を減弱化するための方法および組成物
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
GB2560250A (en) 2015-06-30 2018-09-05 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
US10316295B2 (en) * 2015-12-17 2019-06-11 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

Also Published As

Publication number Publication date
US20240132547A1 (en) 2024-04-25
JP2022046680A (ja) 2022-03-23
US20190055288A1 (en) 2019-02-21
JP7621930B2 (ja) 2025-01-27
US12049480B2 (en) 2024-07-30
US12365707B2 (en) 2025-07-22
US11649264B2 (en) 2023-05-16
US20210284697A1 (en) 2021-09-16
US12202860B2 (en) 2025-01-21
US20250376492A1 (en) 2025-12-11
JP2018531023A (ja) 2018-10-25
US20240109940A1 (en) 2024-04-04
US10968253B2 (en) 2021-04-06
US20240409588A1 (en) 2024-12-12
JP2025063185A (ja) 2025-04-15
US20240228546A9 (en) 2024-07-11
WO2017068077A1 (en) 2017-04-27

Similar Documents

Publication Publication Date Title
JP7621930B2 (ja) 遺伝子操作のための方法及び製品
EP3365437B1 (en) Methods and products for genetic engineering
JP2018531023A6 (ja) 遺伝子操作のための方法及び製品
US20210353543A1 (en) Targeted lipid particles and compositions and uses thereof
WO2016094867A1 (en) Protected guide rnas (pgrnas)
WO2016049163A2 (en) Use and production of chd8+/- transgenic animals with behavioral phenotypes characteristic of autism spectrum disorder
US11492614B2 (en) Stem loop RNA mediated transport of mitochondria genome editing molecules (endonucleases) into the mitochondria
US20240279685A1 (en) Methods and compositions for producing viral fusosomes
US20260098062A1 (en) Lipid particles comprising variant paramyxovirus attachment glycoproteins and uses thereof
EP4636083A2 (en) Methods and products for genetic engineering
US20250144235A1 (en) Methods of repeat dosing and administration of lipid particles or viral vectors and related systems and uses
WO2025262311A1 (en) Methods and products for prime editing genetic engineering
WO2025262314A1 (en) Methods and products for prime editing genetic engineering

Legal Events

Date Code Title Description
A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20191018

A621 Written request for application examination

Free format text: JAPANESE INTERMEDIATE CODE: A621

Effective date: 20191018

A131 Notification of reasons for refusal

Free format text: JAPANESE INTERMEDIATE CODE: A131

Effective date: 20201110

A601 Written request for extension of time

Free format text: JAPANESE INTERMEDIATE CODE: A601

Effective date: 20210209

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20210412

A02 Decision of refusal

Free format text: JAPANESE INTERMEDIATE CODE: A02

Effective date: 20210831

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20211227

C60 Trial request (containing other claim documents, opposition documents)

Free format text: JAPANESE INTERMEDIATE CODE: C60

Effective date: 20211227

C11 Written invitation by the commissioner to file amendments

Free format text: JAPANESE INTERMEDIATE CODE: C11

Effective date: 20220118

A911 Transfer to examiner for re-examination before appeal (zenchi)

Free format text: JAPANESE INTERMEDIATE CODE: A911

Effective date: 20220221

C21 Notice of transfer of a case for reconsideration by examiners before appeal proceedings

Free format text: JAPANESE INTERMEDIATE CODE: C21

Effective date: 20220222

TRDD Decision of grant or rejection written
A01 Written decision to grant a patent or to grant a registration (utility model)

Free format text: JAPANESE INTERMEDIATE CODE: A01

Effective date: 20220329

A61 First payment of annual fees (during grant procedure)

Free format text: JAPANESE INTERMEDIATE CODE: A61

Effective date: 20220413

R150 Certificate of patent or registration of utility model

Ref document number: 7059179

Country of ref document: JP

Free format text: JAPANESE INTERMEDIATE CODE: R150

R250 Receipt of annual fees

Free format text: JAPANESE INTERMEDIATE CODE: R250