CN109996880A - 基于模块化aav递送系统的crispr-cas基因组工程 - Google Patents

基于模块化aav递送系统的crispr-cas基因组工程 Download PDF

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CN109996880A
CN109996880A CN201780064482.9A CN201780064482A CN109996880A CN 109996880 A CN109996880 A CN 109996880A CN 201780064482 A CN201780064482 A CN 201780064482A CN 109996880 A CN109996880 A CN 109996880A
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grna
aav
capsid
composition
cas9
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普拉尚特·马利
德拉瓦·卡特埃卡
阿纳·莫雷诺·科拉多
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University of California San Diego UCSD
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CN201780064482.9A 2016-08-18 2017-08-18 基于模块化aav递送系统的crispr-cas基因组工程 Pending CN109996880A (zh)

Applications Claiming Priority (7)

Application Number Priority Date Filing Date Title
US201662376855P 2016-08-18 2016-08-18
US62/376,855 2016-08-18
US201662415858P 2016-11-01 2016-11-01
US62/415,858 2016-11-01
US201762481589P 2017-04-04 2017-04-04
US62/481,589 2017-04-04
PCT/US2017/047687 WO2018035503A1 (en) 2016-08-18 2017-08-18 Crispr-cas genome engineering via a modular aav delivery system

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CN109996880A true CN109996880A (zh) 2019-07-09

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CN201780064482.9A Pending CN109996880A (zh) 2016-08-18 2017-08-18 基于模块化aav递送系统的crispr-cas基因组工程

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US (1) US20200340012A1 (https=)
EP (1) EP3500667A4 (https=)
JP (3) JP2019524162A (https=)
KR (1) KR20190065251A (https=)
CN (1) CN109996880A (https=)
AU (1) AU2017313917B2 (https=)
CA (1) CA3034089A1 (https=)
IL (2) IL315358A (https=)
WO (1) WO2018035503A1 (https=)

Cited By (2)

* Cited by examiner, † Cited by third party
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CN110760544A (zh) * 2019-11-12 2020-02-07 保定诺未科技有限公司 拆分型SpCas9慢病毒载体及其在干细胞基因编辑中的应用
CN114107347A (zh) * 2021-11-24 2022-03-01 中国人民解放军空军军医大学 一种基于具有按需抗炎功能、装载炎症响应型mRNA的工程化外泌体及其构建方法和应用

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* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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
US9526784B2 (en) 2013-09-06 2016-12-27 President And Fellows Of Harvard College Delivery system for functional nucleases
US9228207B2 (en) 2013-09-06 2016-01-05 President And Fellows Of Harvard College Switchable gRNAs comprising aptamers
US9322037B2 (en) 2013-09-06 2016-04-26 President And Fellows Of Harvard College Cas9-FokI fusion proteins and uses thereof
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
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JP7305534B2 (ja) * 2016-07-06 2023-07-10 バーテックス ファーマシューティカルズ インコーポレイテッド 疼痛関連障害を処置するための物質及び方法
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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
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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
LT3645553T (lt) * 2017-06-27 2023-04-25 Regeneron Pharmaceuticals, Inc. Modifikuoto tropizmo rekombinantinės viruso dalelės ir jų panaudojimas genetinės medžiagos tiksliniam įvedimui į žmogaus ląsteles
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
CN109929839B (zh) * 2017-12-18 2021-02-12 华东师范大学 拆分型单碱基基因编辑系统及其应用
AU2019227910B2 (en) * 2018-02-28 2025-07-17 The Regents Of The University Of California A modular system for gene and protein delivery based on AAV
EP4169576A1 (en) * 2018-03-23 2023-04-26 University of Massachusetts Gene therapeutics for treating bone disorders
US12157760B2 (en) 2018-05-23 2024-12-03 The Broad Institute, Inc. Base editors and uses thereof
EP3810776A1 (en) * 2018-06-22 2021-04-28 F. Hoffmann-La Roche AG Oligonucleotides for modulating scn9a expression
US12522807B2 (en) 2018-07-09 2026-01-13 The Broad Institute, Inc. RNA programmable epigenetic RNA modifiers and uses thereof
AU2019317066A1 (en) * 2018-08-07 2021-02-18 Modalis Therapeutics Corporation Novel transcription activator
US20210355475A1 (en) * 2018-08-10 2021-11-18 Cornell University Optimized base editors enable efficient editing in cells, organoids and mice
KR20210058816A (ko) 2018-08-18 2021-05-24 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 원 위치에 유전자 편집
EP3847254A4 (en) 2018-09-07 2022-08-10 Beam Therapeutics Inc. COMPOSITIONS AND METHODS FOR DELIVERING A NUCLEOBASE EDITING SYSTEM
WO2020092453A1 (en) 2018-10-29 2020-05-07 The Broad Institute, Inc. Nucleobase editors comprising geocas9 and uses thereof
US12351837B2 (en) 2019-01-23 2025-07-08 The Broad Institute, Inc. Supernegatively charged proteins and uses thereof
WO2020191233A1 (en) 2019-03-19 2020-09-24 The Broad Institute, Inc. Methods and compositions for editing nucleotide sequences
US20220202957A1 (en) * 2019-04-09 2022-06-30 The Regents Of The University Of California Long-lasting analgesia via targeted in vivo epigenetic repression
US12473543B2 (en) 2019-04-17 2025-11-18 The Broad Institute, Inc. Adenine base editors with reduced off-target effects
EP3736330A1 (en) * 2019-05-08 2020-11-11 European Molecular Biology Laboratory Modified adeno-associated virus (aav) particles for gene therapy
WO2021064162A1 (en) * 2019-10-02 2021-04-08 Helmholtz Zentrum München - Deutsches Forschungszentrum für Gesundheit und Umwelt (GmbH) Treatment of diseases caused by frame shift mutations
US12435330B2 (en) 2019-10-10 2025-10-07 The Broad Institute, Inc. Methods and compositions for prime editing RNA
KR20220094216A (ko) 2019-11-08 2022-07-05 코브 테라퓨틱스 변형된 아데노-관련 바이러스 벡터 및 이의 중추신경계로의 전달
WO2021209574A1 (en) * 2020-04-15 2021-10-21 Fondazione Telethon Constructs comprising inteins
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
CN111500635B (zh) * 2020-07-02 2020-10-09 北京大学第三医院(北京大学第三临床医学院) 一种包含携带核酸分子的载体的试剂盒
CN116802279A (zh) 2020-11-06 2023-09-22 科亚韦治疗公司 内酰胺修饰的腺相关病毒载体
AU2021378075B2 (en) 2020-11-11 2025-03-27 Borea Therapeutics S.R.L. Modified viral particles for gene therapy
JP2024505570A (ja) * 2021-02-01 2024-02-06 ナヴェガ セラピューティクス, インコーポレイテッド 神経学的疾患及び疼痛を治療するためのエピジェネティック遺伝子調節
WO2024129990A2 (en) * 2022-12-14 2024-06-20 The Board Of Trustees Of The Leland Stanford Junior University Chemically modified aavs
CN116530470B (zh) * 2022-12-26 2025-09-23 华中科技大学同济医学院附属同济医院 一种自身免疫性肝炎小鼠模型的创建方法及应用
CN116676334B (zh) * 2023-02-08 2025-05-27 深圳大学 抑郁症动物模型的构建方法
CN121175075A (zh) * 2023-04-17 2025-12-19 桑加莫治疗公司 用于阻遏nav1.7表达的锌指蛋白转录因子

Citations (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2015062516A1 (zh) * 2013-10-30 2015-05-07 北京大学 定点突变和定点修饰的腺相关病毒、其制备方法及应用
CN105164264A (zh) * 2012-12-12 2015-12-16 布罗德研究所有限公司 用于序列操纵和治疗应用的系统、方法和组合物的递送、工程化和优化
CN105247044A (zh) * 2013-05-31 2016-01-13 加利福尼亚大学董事会 腺相关病毒变体及其使用方法
WO2016020399A1 (en) * 2014-08-04 2016-02-11 Centre Hospitalier Universitaire Vaudois (Chuv) GENOME EDITING FOR THE TREATMENT OF HUNTINGTON's DISEASE
CN105408486A (zh) * 2013-05-21 2016-03-16 佛罗里达大学研究基金会有限公司 衣壳修饰的raav3载体组合物以及在人肝癌基因治疗中的用途
WO2016112242A1 (en) * 2015-01-08 2016-07-14 President And Fellows Of Harvard College Split cas9 proteins
WO2017075335A1 (en) * 2015-10-28 2017-05-04 Voyager Therapeutics, Inc. Regulatable expression using adeno-associated virus (aav)
US20170233703A1 (en) * 2015-05-21 2017-08-17 Tsinghua University Genetic indicator and control system and method utilizing split Cas9/CRISPR domains for transcriptional control in eukaryotic cell lines

Family Cites Families (53)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5792462A (en) 1995-05-23 1998-08-11 University Of North Carolina At Chapel Hill Alphavirus RNA replicon systems
US5811407A (en) 1997-02-19 1998-09-22 The University Of North Carolina At Chapel Hill System for the in vivo delivery and expression of heterologous genes in the bone marrow
US6982082B1 (en) 1997-08-27 2006-01-03 President And Fellows Of Harvard College Gene therapy by cell specific targeting
US6759237B1 (en) 1998-11-05 2004-07-06 The Trustees Of The University Of Pennsylvania Adeno-associated virus serotype 1 nucleic acid sequences, vectors and host cells containing same
AU780231B2 (en) 1998-11-10 2005-03-10 University Of North Carolina At Chapel Hill, The Virus vectors and methods of making and administering the same
US7201898B2 (en) 2000-06-01 2007-04-10 The University Of North Carolina At Chapel Hill Methods and compounds for controlled release of recombinant parvovirus vectors
ATE318923T1 (de) 2000-06-01 2006-03-15 Univ North Carolina Doppelsträngige parvovirus-vektoren
US6531235B2 (en) 2001-06-06 2003-03-11 Max-Planck-Institute für Mikrostrukturphysik Non-c-axis oriented bismuth-layered perovskite ferroelectric structure epitaxially grown on buffered silicon
MX359371B (es) 2001-11-13 2018-09-25 Univ Pennsylvania Un metodo para detectar y/o identificar secuencias del virus adeno-asociado y aislamiento de secuencias novedosas identificadas de ese modo.
AU2002365366B2 (en) 2001-11-21 2007-05-10 The Trustees Of The University Of Pennsylvania Simian adenovirus nucleic acid and amino acid sequences, vectors containing same, and methods of use
PT1453547T (pt) 2001-12-17 2016-12-28 Univ Pennsylvania Sequências do vírus adeno-associado (aav) do serotipo 8, vetores contendo as mesmas, e utilizações destas
EP1456383B1 (en) * 2001-12-21 2014-03-12 Medigene AG A library of modified structural genes or capsid modified particles useful for the identification of viral clones with desired cell tropism
AU2003223766A1 (en) 2002-04-30 2003-11-17 University Of North Carolina At Chapel Hill Secretion signal vectors
US7301898B1 (en) 2002-07-29 2007-11-27 Brocade Communications Systems, Inc. Credit sharing for fibre channel links with multiple virtual channels
EP2345731B1 (en) 2003-09-30 2015-10-21 The Trustees of the University of Pennsylvania Adeno-associated virus (AAV) clades, sequences, vectors containing same, and uses thereof
JP5136766B2 (ja) 2004-12-15 2013-02-06 ユニバーシティ オブ ノース カロライナ アット チャペル ヒル キメラベクター
ES2525067T3 (es) 2005-04-07 2014-12-17 The Trustees Of The University Of Pennsylvania Método de incremento de la función de un vector de AAV
US7867484B2 (en) 2006-01-27 2011-01-11 University Of North Carolina At Chapel Hill Heparin and heparan sulfate binding chimeric vectors
EP2012822B1 (en) 2006-04-28 2010-01-20 The Trustees of the University of Pennsylvania Modified adenovirus hexon protein and uses thereof
EP2018421B1 (en) 2006-04-28 2012-12-19 The Trustees of the University of Pennsylvania Scalable production method for aav
US9725485B2 (en) * 2012-05-15 2017-08-08 University Of Florida Research Foundation, Inc. AAV vectors with high transduction efficiency and uses thereof for gene therapy
CN101883858B (zh) 2007-11-28 2015-07-22 宾夕法尼亚大学托管会 猿猴亚家族E腺病毒SAdV-39、-25.2、-26、-30、-37和-38及其应用
AU2008331905B2 (en) 2007-11-28 2014-09-18 The Trustees Of The University Of Pennsylvania Simian subfamily B adenovirus SAdV-28 and uses thereof
WO2009137006A2 (en) 2008-04-30 2009-11-12 The University Of North Carolina At Chapel Hill Directed evolution and in vivo panning of virus vectors
WO2010051367A1 (en) 2008-10-31 2010-05-06 The Trustees Of The University Of Pennsylvania Simian adenoviruses sadv-43, -45,-48,-49, and -50 and uses thereof
WO2010093784A2 (en) 2009-02-11 2010-08-19 The University Of North Carolina At Chapel Hill Modified virus vectors and methods of making and using the same
CA2762203A1 (en) 2009-05-29 2010-12-02 Soumitra Roy Simian adenovirus 41 and uses thereof
JP5618557B2 (ja) 2010-01-29 2014-11-05 富士フイルム株式会社 感活性光線性または感放射線性樹脂組成物、及び該組成物を用いたパターン形成方法
US9315825B2 (en) 2010-03-29 2016-04-19 The Trustees Of The University Of Pennsylvania Pharmacologically induced transgene ablation system
AU2011332025B2 (en) 2010-11-23 2015-06-25 The Trustees Of The University Of Pennsylvania Subfamily E simian adenoviruses A1321, A1325, A1295, A1309 and A1322 and uses thereof
WO2012109570A1 (en) 2011-02-10 2012-08-16 The University Of North Carolina At Chapel Hill Viral vectors with modified transduction profiles and methods of making and using the same
SG11201407343XA (en) 2012-05-18 2014-12-30 Univ Pennsylvania Subfamily e simian adenoviruses a1302, a1320, a1331 and a1337 and uses thereof
CA2886161A1 (en) 2012-09-29 2014-04-03 The Trustees Of The University Of Pennsylvania Veterinary composition and methods for non-surgical neutering and castration
US20140271550A1 (en) 2013-03-14 2014-09-18 The Trustees Of The University Of Pennsylvania Constructs and Methods for Delivering Molecules via Viral Vectors with Blunted Innate Immune Responses
AU2014235096B2 (en) 2013-03-15 2020-03-19 The Trustees Of The University Of Pennsylvania Compositions and methods for treating MPSI
WO2015012924A2 (en) 2013-04-29 2015-01-29 The Trustees Of The University Of Pennsylvania Tissue preferential codon modified expression cassettes, vectors containing same, and use thereof
CN115120746A (zh) 2013-05-15 2022-09-30 明尼苏达大学董事会 腺相关病毒介导的基因向中枢神经系统转移
WO2014197748A2 (en) * 2013-06-05 2014-12-11 Duke University Rna-guided gene editing and gene regulation
WO2015040075A1 (en) * 2013-09-18 2015-03-26 Genome Research Limited Genomic screening methods using rna-guided endonucleases
WO2015048577A2 (en) * 2013-09-27 2015-04-02 Editas Medicine, Inc. Crispr-related methods and compositions
EP3470089A1 (en) * 2013-12-12 2019-04-17 The Broad Institute Inc. Delivery, use and therapeutic applications of the crispr-cas systems and compositions for targeting disorders and diseases using particle delivery components
EP3115457B1 (en) * 2014-03-05 2019-10-02 National University Corporation Kobe University Genomic sequence modification method for specifically converting nucleic acid bases of targeted dna sequence, and molecular complex for use in same
SMT202000531T1 (it) 2014-03-09 2020-11-10 Univ Pennsylvania Composizioni utili in trattamento di deficit di ornitina transcarbamilasi(otc)
WO2015164723A1 (en) 2014-04-25 2015-10-29 The Trustees Of The University Of Pennsylvania Methods and compositions for treating metastatic breast cancer and other cancers in the brain
LT3137497T (lt) * 2014-05-02 2021-07-26 Genzyme Corporation Aav vektoriai, skirti tinklainės ir cns genų terapijai
ES2833230T3 (es) 2014-05-13 2021-06-14 Univ Pennsylvania Composiciones que comprenden AVV que expresan construcciones y usos de doble anticuerpo del mismo
EP3212165B1 (en) * 2014-10-30 2024-02-28 President and Fellows of Harvard College Delivery of negatively charged proteins using cationic lipids
PT3216867T (pt) * 2014-11-04 2020-07-16 Univ Kobe Nat Univ Corp Método para modificar a sequência de genoma para introduzir mutação específica a sequência de adn alvo por reação de remoção de bases, e complexo molecular nele utilizado
WO2016112921A1 (en) * 2015-01-15 2016-07-21 University Of Copenhagen Virus-like particle with efficient epitope display
WO2016205749A1 (en) * 2015-06-18 2016-12-22 The Broad Institute Inc. Novel crispr enzymes and systems
US10954513B2 (en) * 2015-06-18 2021-03-23 University Of Utah Research Foundation RNA-guided transcriptional regulation and methods of using the same for the treatment of back pain
CN116814590A (zh) 2015-10-22 2023-09-29 布罗德研究所有限公司 Vi-b型crispr酶和系统
CN109843914B (zh) * 2016-07-06 2024-03-15 沃泰克斯药物股份有限公司 用于治疗疼痛相关病症的材料和方法

Patent Citations (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN105164264A (zh) * 2012-12-12 2015-12-16 布罗德研究所有限公司 用于序列操纵和治疗应用的系统、方法和组合物的递送、工程化和优化
CN105408486A (zh) * 2013-05-21 2016-03-16 佛罗里达大学研究基金会有限公司 衣壳修饰的raav3载体组合物以及在人肝癌基因治疗中的用途
CN105247044A (zh) * 2013-05-31 2016-01-13 加利福尼亚大学董事会 腺相关病毒变体及其使用方法
WO2015062516A1 (zh) * 2013-10-30 2015-05-07 北京大学 定点突变和定点修饰的腺相关病毒、其制备方法及应用
WO2016020399A1 (en) * 2014-08-04 2016-02-11 Centre Hospitalier Universitaire Vaudois (Chuv) GENOME EDITING FOR THE TREATMENT OF HUNTINGTON's DISEASE
WO2016112242A1 (en) * 2015-01-08 2016-07-14 President And Fellows Of Harvard College Split cas9 proteins
US20170233703A1 (en) * 2015-05-21 2017-08-17 Tsinghua University Genetic indicator and control system and method utilizing split Cas9/CRISPR domains for transcriptional control in eukaryotic cell lines
WO2017075335A1 (en) * 2015-10-28 2017-05-04 Voyager Therapeutics, Inc. Regulatable expression using adeno-associated virus (aav)

Non-Patent Citations (3)

* Cited by examiner, † Cited by third party
Title
LINLIN SUN等: "The CRISPR/Cas9 system for gene editing and its potential application in pain research", 《TRANSL PERIOPER PAIN MED》, 3 August 2016 (2016-08-03), pages 22 - 23 *
LINLIN SUN等: "The CRISPR/Cas9 system for gene editing and its potential application in pain research", 《TRANSL PERIOPER PAIN MED》, vol. 1, no. 3, pages 22 - 33, XP055603094, DOI: 10.31480/2330-4871/040 *
LUKE A. GILBERT等: ""CRISPR-mediated modular RNA-guided regulation of transcription in eukaryotes", 《CELL》, vol. 154, no. 2, pages 442 - 451 *

Cited By (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN110760544A (zh) * 2019-11-12 2020-02-07 保定诺未科技有限公司 拆分型SpCas9慢病毒载体及其在干细胞基因编辑中的应用
CN114107347A (zh) * 2021-11-24 2022-03-01 中国人民解放军空军军医大学 一种基于具有按需抗炎功能、装载炎症响应型mRNA的工程化外泌体及其构建方法和应用

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