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
JP2018531023A5 (enExample
JP2018531023A (ja
JP2018531023A6 (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
Original Assignee
Centre National de la Recherche Scientifique
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 filed Critical Centre National de la Recherche Scientifique
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/JP7879292B2/ja
Active legal-status Critical Current
Anticipated expiration legal-status Critical

Links

Images

Classifications

    • 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
    • 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
    • C12N15/10—Processes for the isolation, preparation or purification of DNA or RNA
    • C12N15/102—Mutagenizing nucleic acids
    • 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
    • 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
    • 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
    • C12N15/90—Stable introduction of foreign DNA into chromosome
    • 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
    • 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
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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
    • 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]
    • 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
    • C12N2740/10023—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
    • C12N2740/10041—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

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Biomedical Technology (AREA)
  • Molecular Biology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • 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 JP7879292B2 (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 (enExample) 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 Active JP7879292B2 (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 Active JP7879292B2 (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 (enExample)
JP (3) JP7059179B2 (enExample)
WO (1) WO2017068077A1 (enExample)

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 (95)

* 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
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
US9526784B2 (en) 2013-09-06 2016-12-27 President And Fellows Of Harvard College Delivery system for functional nucleases
US9340799B2 (en) 2013-09-06 2016-05-17 President And Fellows Of Harvard College MRNA-sensing switchable gRNAs
US9322037B2 (en) 2013-09-06 2016-04-26 President And Fellows Of Harvard College Cas9-FokI 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
AU2015298571B2 (en) 2014-07-30 2020-09-03 President And Fellows Of Harvard College Cas9 proteins including ligand-dependent inteins
US11980663B2 (en) 2015-07-08 2024-05-14 American Gene Technologies International Inc. HIV pre-immunization and immunotherapy
WO2017070632A2 (en) 2015-10-23 2017-04-27 President And Fellows Of Harvard College Nucleobase editors and uses thereof
IL297238B2 (en) 2016-01-15 2024-08-01 American Gene Tech Int Inc Methods and preparations for gamma-delta T-cell activation
US10137144B2 (en) 2016-01-15 2018-11-27 American Gene Technologies International Inc. Methods and compositions for the activation of gamma-delta T-cells
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
WO2017213697A1 (en) 2016-06-08 2017-12-14 American Gene Technologies International Inc. Non-integrating viral delivery system and methods related thereto
CA3028982A1 (en) 2016-07-08 2018-01-11 American Gene Technologies International Inc. Hiv pre-immunization and immunotherapy
WO2018017882A1 (en) 2016-07-21 2018-01-25 American Gene Technologies International Inc. Viral vectors for treating parkinson's disease
WO2018027078A1 (en) 2016-08-03 2018-02-08 President And Fellows Of Harard College Adenosine nucleobase editors and uses thereof
AU2017308889B2 (en) 2016-08-09 2023-11-09 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
KR20240007715A (ko) 2016-10-14 2024-01-16 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
KR20190123328A (ko) 2017-03-09 2019-10-31 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 암 백신
WO2018165504A1 (en) 2017-03-09 2018-09-13 President And Fellows Of Harvard College Suppression of pain by gene editing
CN110914310A (zh) 2017-03-10 2020-03-24 哈佛大学的校长及成员们 胞嘧啶至鸟嘌呤碱基编辑器
IL306092A (en) 2017-03-23 2023-11-01 Harvard College Nucleic base editors that include 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
WO2018195360A1 (en) 2017-04-21 2018-10-25 Seattle Children's Hospital (Dba Seattle Childern'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
US12559769B2 (en) 2017-06-16 2026-02-24 American Gene Technologies International Inc. Methods and compositions for the activation of tumor cytotoxicity via human gamma-delta t-cells
EP3658573A1 (en) 2017-07-28 2020-06-03 President and Fellows of Harvard College Methods and compositions for evolving base editors using phage-assisted continuous evolution (pace)
EP3676376B1 (en) 2017-08-30 2025-01-15 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
US12406749B2 (en) 2017-12-15 2025-09-02 The Broad Institute, Inc. Systems and methods for predicting repair outcomes in genetic engineering
KR102446690B1 (ko) * 2017-12-22 2022-09-23 에이에스엠엘 네델란즈 비.브이. 광학 수차를 포함하는 패터닝 공정 개선
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
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
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
WO2020022802A1 (ko) * 2018-07-25 2020-01-30 주식회사 툴젠 인위적인 유전자 조작을 통한 자가면역질환 치료
EP3852813A4 (en) 2018-09-18 2022-06-22 VNV Newco Inc. ARC-BASED CAPSIDS AND THEIR USES
US20210403907A1 (en) 2018-09-18 2021-12-30 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
EP3876952A4 (en) 2018-11-05 2022-08-24 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
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
US20220194990A1 (en) * 2019-01-31 2022-06-23 Board Of Regents Of The University Of Nebraska Virus-like particles and methods of use thereof
WO2020191241A1 (en) 2019-03-19 2020-09-24 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
US20220259617A1 (en) 2019-06-13 2022-08-18 The General Hospital Corporation Engineered human-endogenous virus-like particles and methods of use thereof for delivery to cells
EP4009972A4 (en) 2019-08-09 2023-03-08 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
US12551560B2 (en) 2019-09-09 2026-02-17 Scribe Therapeutics Inc. Compositions and methods for use in immunotherapy
EP4028522A1 (en) 2019-09-09 2022-07-20 Scribe Therapeutics Inc. Compositions and methods for the targeting of sod1
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
US20240083973A1 (en) 2019-10-09 2024-03-14 Ecole Normale Supérieure de Lyon 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
WO2021113763A1 (en) * 2019-12-06 2021-06-10 Scribe Therapeutics Inc. Compositions and methods for the targeting of rhodopsin
WO2021113772A1 (en) * 2019-12-06 2021-06-10 Scribe Therapeutics Inc. Particle delivery systems
EP4069846A1 (en) * 2019-12-07 2022-10-12 Scribe Therapeutics Inc. Compositions and methods for the targeting of htt
CA3177481A1 (en) 2020-05-08 2021-11-11 David R. Liu Methods and compositions for simultaneous editing of both strands of a target double-stranded nucleotide sequence
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
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
IL308836A (en) 2021-05-28 2024-01-01 Sana Biotechnology Inc Lipid Particles Containing Baboon Truncated Endogenous Retrovirus (BAEV) Envelope Glycoprotein and Related Methods and Uses
WO2022261150A2 (en) * 2021-06-09 2022-12-15 Scribe Therapeutics Inc. Particle delivery systems
IL311610A (en) 2021-09-21 2024-05-01 Scribe Therapeutics Inc Engineered CASX suppressor systems
WO2023077107A1 (en) 2021-10-29 2023-05-04 Sana Biotechnology, Inc. Methods and reagents for amplifying viral vector nucleic acid products
US20250223584A2 (en) * 2021-12-03 2025-07-10 The Broad Institute, Inc. Compositions and methods for efficient in vivo delivery
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
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
WO2023114949A1 (en) 2021-12-16 2023-06-22 Sana Biotechnology, Inc. Methods and systems of particle production
EP4448775A1 (en) 2021-12-17 2024-10-23 Sana Biotechnology, Inc. Modified paramyxoviridae attachment glycoproteins
US20250059239A1 (en) 2021-12-17 2025-02-20 Sana Biotechnology, Inc. Modified paramyxoviridae fusion glycoproteins
US20250082777A1 (en) 2022-01-10 2025-03-13 Sana Biotechnology, Inc. Methods of ex vivo dosing and administration of lipid particles or viral vectors and related systems and uses
WO2023150518A1 (en) 2022-02-01 2023-08-10 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
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
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
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
AU2025208175A1 (en) 2024-01-12 2026-06-25 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
WO2026107131A2 (en) 2024-11-14 2026-05-21 Orbital Therapeutics, Inc. Engineered viral particles for targeted delivery

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
WO2010062757A1 (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
EP2841572B1 (en) 2012-04-27 2019-06-19 Duke University Genetic correction of mutated genes
KR102955556B1 (ko) 2012-12-12 2026-04-22 더 브로드 인스티튜트, 인코퍼레이티드 서열 조작을 위한 crispr-cas 성분 시스템, 방법 및 조성물
PL3019619T3 (pl) * 2013-07-11 2022-01-10 Modernatx, Inc. Kompozycje zawierające syntetyczne polinkleotydy kodujące białka powiązane z crispr i syntetyczne sgrna oraz sposoby ich stosowania
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
ES2843552T3 (es) 2013-12-12 2021-07-19 Dow Agrosciences Llc Control selectivo de malas hierbas con halauxifen
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
WO2015184262A1 (en) * 2014-05-30 2015-12-03 The Board Of Trustees Of The Leland Stanford Junior University Compositions and methods of delivering treatments for latent viral infections
EA201790551A1 (ru) * 2014-09-07 2017-07-31 Селекта Байосайенсиз, Инк. Способы и композиции для ослабления иммунных ответов к пропускающему экзон вирусному трансферному вектору
DK3597740T3 (da) * 2014-11-06 2022-06-20 Dupont Us Holding Llc Peptidmedieret indføring af rna-styret endonuklease i celler
CA2990883A1 (en) 2015-06-30 2017-01-05 Ethris Gmbh Atp-binding cassette family coding polyribonucleotides and formulations thereof
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
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
WO2017068077A1 (en) 2017-04-27
US12202860B2 (en) 2025-01-21
US20240132547A1 (en) 2024-04-25
US10968253B2 (en) 2021-04-06
JP2025063185A (ja) 2025-04-15
US20190055288A1 (en) 2019-02-21
US20240228546A9 (en) 2024-07-11
US12049480B2 (en) 2024-07-30
US20240409588A1 (en) 2024-12-12
US20240109940A1 (en) 2024-04-04
US20250376492A1 (en) 2025-12-11
JP7621930B2 (ja) 2025-01-27
US20210284697A1 (en) 2021-09-16
US11649264B2 (en) 2023-05-16
JP7879292B2 (ja) 2026-06-23
JP2022046680A (ja) 2022-03-23
US12365707B2 (en) 2025-07-22
JP2018531023A (ja) 2018-10-25

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
JP2024544012A (ja) 核酸プログラマブル融合タンパク質を送達するためのセルフアセンブルウイルス様粒子ならびにその作製方法および使用方法
US20240279685A1 (en) Methods and compositions for producing viral fusosomes
EP3230451A1 (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
US20260098062A1 (en) Lipid particles comprising variant paramyxovirus attachment glycoproteins and uses thereof
US20250144235A1 (en) Methods of repeat dosing and administration of lipid particles or viral vectors and related systems and uses
EP4636083A2 (en) Methods and products for genetic engineering
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