CN108136047B - 提高原代细胞中基于核酸内切酶的基因编辑 - Google Patents

提高原代细胞中基于核酸内切酶的基因编辑 Download PDF

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CN108136047B
CN108136047B CN201680039989.4A CN201680039989A CN108136047B CN 108136047 B CN108136047 B CN 108136047B CN 201680039989 A CN201680039989 A CN 201680039989A CN 108136047 B CN108136047 B CN 108136047B
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CN108136047A (zh
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安德鲁·沙尔博格
戴维德·罗林斯
迈克尔·C·延森
卡米拉·格威亚兹达
亚历山德拉·格里尔
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CN201680039989.4A 2015-05-13 2016-05-12 提高原代细胞中基于核酸内切酶的基因编辑 Active CN108136047B (zh)

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US201562161104P 2015-05-13 2015-05-13
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PCT/US2016/032153 WO2016183345A1 (en) 2015-05-13 2016-05-12 Enhancing endonuclease based gene editing in primary cells

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Families Citing this family (78)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP2734621B1 (en) 2011-07-22 2019-09-04 President and Fellows of Harvard College Evaluation and improvement of nuclease cleavage specificity
US9458450B2 (en) 2012-03-15 2016-10-04 Flodesign Sonics, Inc. Acoustophoretic separation technology using multi-dimensional standing waves
US10704021B2 (en) 2012-03-15 2020-07-07 Flodesign Sonics, Inc. Acoustic perfusion devices
US10967298B2 (en) 2012-03-15 2021-04-06 Flodesign Sonics, Inc. Driver and control for variable impedence load
US9950282B2 (en) 2012-03-15 2018-04-24 Flodesign Sonics, Inc. Electronic configuration and control for acoustic standing wave generation
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
US9340800B2 (en) 2013-09-06 2016-05-17 President And Fellows Of Harvard College Extended DNA-sensing GRNAS
US9526784B2 (en) 2013-09-06 2016-12-27 President And Fellows Of Harvard College Delivery system for functional nucleases
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
US9725710B2 (en) 2014-01-08 2017-08-08 Flodesign Sonics, Inc. Acoustophoresis device with dual acoustophoretic chamber
CA2956224A1 (en) 2014-07-30 2016-02-11 President And Fellows Of Harvard College Cas9 proteins including ligand-dependent inteins
US11377651B2 (en) 2016-10-19 2022-07-05 Flodesign Sonics, Inc. Cell therapy processes utilizing acoustophoresis
US11708572B2 (en) 2015-04-29 2023-07-25 Flodesign Sonics, Inc. Acoustic cell separation techniques and processes
US11021699B2 (en) 2015-04-29 2021-06-01 FioDesign Sonics, Inc. Separation using angled acoustic waves
US11474085B2 (en) 2015-07-28 2022-10-18 Flodesign Sonics, Inc. Expanded bed affinity selection
US11459540B2 (en) 2015-07-28 2022-10-04 Flodesign Sonics, Inc. Expanded bed affinity selection
IL295616A (en) 2015-07-31 2022-10-01 Us Health Adapted cells and treatment methods
EP4434589A3 (en) 2015-10-23 2025-05-14 President and Fellows of Harvard College Evolved cas9 proteins for gene editing
WO2017184674A1 (en) * 2016-04-19 2017-10-26 President And Fellows Of Harvard College Methods for increasing efficiency of nuclease-mediated gene editing in stem cells
US11214789B2 (en) 2016-05-03 2022-01-04 Flodesign Sonics, Inc. Concentration and washing of particles with acoustics
US11085035B2 (en) 2016-05-03 2021-08-10 Flodesign Sonics, Inc. Therapeutic cell washing, concentration, and separation utilizing acoustophoresis
JP7231935B2 (ja) 2016-08-03 2023-03-08 プレジデント アンド フェローズ オブ ハーバード カレッジ アデノシン核酸塩基編集因子およびそれらの使用
US11661590B2 (en) 2016-08-09 2023-05-30 President And Fellows Of Harvard College Programmable CAS9-recombinase fusion proteins and uses thereof
WO2018039438A1 (en) 2016-08-24 2018-03-01 President And Fellows Of Harvard College Incorporation of unnatural amino acids into proteins using base editing
AU2017342543B2 (en) 2016-10-14 2024-06-27 President And Fellows Of Harvard College AAV delivery of nucleobase editors
EP4338799A3 (en) 2016-10-18 2024-06-05 Regents of the University of Minnesota Tumor infiltrating lymphocytes and methods of therapy
KR20190127655A (ko) 2016-10-19 2019-11-13 프로디자인 소닉스, 인크. 음향학에 의한 친화성 세포 추출
GB2607227B (en) * 2016-10-27 2023-05-10 Intima Bioscience Inc Viral methods of T cell therapy
WO2018119359A1 (en) 2016-12-23 2018-06-28 President And Fellows Of Harvard College Editing of ccr5 receptor gene to protect against hiv infection
CN107746845B (zh) * 2016-12-28 2021-03-26 北京微旋基因技术有限公司 特异性靶向LAG-3基因的sgRNA和特异性敲除LAG-3基因的方法
CN107760680B (zh) * 2016-12-28 2020-09-22 北京微旋基因技术有限公司 特异性靶向TIM-3基因的sgRNA和特异性敲除TIM-3基因的方法
CN110662556A (zh) 2017-03-09 2020-01-07 哈佛大学的校长及成员们 癌症疫苗
US11898179B2 (en) 2017-03-09 2024-02-13 President And Fellows Of Harvard College Suppression of pain by gene editing
JP2020510439A (ja) 2017-03-10 2020-04-09 プレジデント アンド フェローズ オブ ハーバード カレッジ シトシンからグアニンへの塩基編集因子
WO2018176009A1 (en) 2017-03-23 2018-09-27 President And Fellows Of Harvard College Nucleobase editors comprising nucleic acid programmable dna binding proteins
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
CN108866100A (zh) * 2017-05-16 2018-11-23 中国科学院上海生命科学研究院 一种高效率的基因编辑方法
CA3067914A1 (en) 2017-06-30 2019-01-03 Cellectis Cellular immunotherapy for repetitive administration
EP3645021A4 (en) 2017-06-30 2021-04-21 Intima Bioscience, Inc. ADENO-ASSOCIATED VIRAL VECTORS FOR GENE THERAPY
US11732274B2 (en) 2017-07-28 2023-08-22 President And Fellows Of Harvard College Methods and compositions for evolving base editors using phage-assisted continuous evolution (PACE)
WO2019139645A2 (en) 2017-08-30 2019-07-18 President And Fellows Of Harvard College High efficiency base editors comprising gam
KR20200121782A (ko) 2017-10-16 2020-10-26 더 브로드 인스티튜트, 인코퍼레이티드 아데노신 염기 편집제의 용도
DK3701028T3 (da) * 2017-10-24 2024-11-11 Editas Medicine Inc Systemer og fremgangsmåder til behandling af hyper-igm-syndrom
JP2021507561A (ja) 2017-12-14 2021-02-22 フロデザイン ソニックス, インク.Flodesign Sonics, Inc. 音響トランスデューサドライバ及びコントローラ
US12406749B2 (en) 2017-12-15 2025-09-02 The Broad Institute, Inc. Systems and methods for predicting repair outcomes in genetic engineering
AU2019236204B2 (en) 2018-03-15 2026-02-26 KSQ Therapeutics, Inc. Gene-regulating compositions and methods for improved immunotherapy
JP7469806B2 (ja) * 2018-03-29 2024-04-17 学校法人自治医科大学 ゲノム編集方法、組成物、細胞、細胞製剤、及び細胞製剤の製造方法
CN112312931A (zh) * 2018-04-27 2021-02-02 西雅图儿童医院(Dba西雅图儿童研究所) X连锁高IgM综合征的治疗性基因组编辑
CN112654710A (zh) 2018-05-16 2021-04-13 辛瑟高公司 用于指导rna设计和使用的方法和系统
US12157760B2 (en) 2018-05-23 2024-12-03 The Broad Institute, Inc. Base editors and uses thereof
WO2019232069A1 (en) * 2018-05-30 2019-12-05 Emerson Collective Investments, Llc Cell therapy
WO2020002380A1 (en) * 2018-06-25 2020-01-02 Ospedale San Raffaele S.R.L Gene therapy
US12522807B2 (en) 2018-07-09 2026-01-13 The Broad Institute, Inc. RNA programmable epigenetic RNA modifiers and uses thereof
WO2020092453A1 (en) 2018-10-29 2020-05-07 The Broad Institute, Inc. Nucleobase editors comprising geocas9 and uses thereof
CN109528653B (zh) * 2018-11-22 2020-07-31 北京大学 具有基因编辑功能的膜性囊泡及其制备方法、药物组合物和用途
CN109735516B (zh) * 2019-01-22 2021-09-03 自然资源部第一海洋研究所 受核苷酸片段引导具有特异核酸内切酶活性的piwi蛋白
WO2020154500A1 (en) 2019-01-23 2020-07-30 The Broad Institute, Inc. Supernegatively charged proteins and uses thereof
MX2021011426A (es) 2019-03-19 2022-03-11 Broad Inst Inc Metodos y composiciones para editar secuencias de nucleótidos.
CN111812066B (zh) * 2019-04-10 2023-04-28 华东理工大学 基于CRISPR/Cas12a系统的生物传感器、试剂盒及其在小分子检测中的用途
WO2020214842A1 (en) 2019-04-17 2020-10-22 The Broad Institute, Inc. Adenine base editors with reduced off-target effects
US12435330B2 (en) 2019-10-10 2025-10-07 The Broad Institute, Inc. Methods and compositions for prime editing RNA
WO2021226558A1 (en) 2020-05-08 2021-11-11 The Broad Institute, Inc. Methods and compositions for simultaneous editing of both strands of a target double-stranded nucleotide sequence
CN111909961B (zh) * 2020-08-21 2022-04-19 华侨大学 一种CRISPR/Cas介导的ATL细胞基因编辑载体及其应用
US20240336904A1 (en) * 2020-12-21 2024-10-10 2Seventy Bio, Inc. Compositions and methods for site-directed mutagenesis
JP2024506016A (ja) * 2021-02-08 2024-02-08 インテリア セラピューティクス,インコーポレイテッド 免疫療法のためのt細胞免疫グロブリン及びムチンドメイン3(tim3)組成物及び方法
CN117042793A (zh) * 2021-02-08 2023-11-10 因特利亚治疗公司 用于免疫疗法的淋巴细胞活化基因3(lag3)组合物和方法
BR112023018948A2 (pt) * 2021-03-19 2023-10-17 Metagenomi Inc Edição multiplex com enzimas cas
CN113481184A (zh) * 2021-08-06 2021-10-08 北京大学 融合蛋白以及其使用方法
EP4392060A1 (en) * 2021-08-24 2024-07-03 Intellia Therapeutics, Inc. Programmed cell death protein 1 (pd1) compositions and methods for cell-based therapy
CA3241474A1 (en) * 2021-12-03 2023-06-08 The General Hospital Corporation Crispr-associated transposases and methods of use thereof
WO2023122036A1 (en) * 2021-12-20 2023-06-29 Sobol Robert E Methods and compositions for improved molecular therapies of multigenic diseases
WO2024064633A2 (en) * 2022-09-19 2024-03-28 Emendobio Inc. Biallelic knockout of pdcd1
WO2024102954A1 (en) 2022-11-10 2024-05-16 Massachusetts Institute Of Technology Activation induced clipping system (aics)
WO2024107119A1 (en) * 2022-11-17 2024-05-23 Agency For Science, Technology And Research T3 vectors for recombinant protein production in mammalian cells
CN117987436B (zh) * 2024-04-03 2024-06-25 南京鸿明生物科技有限公司 一种双链靶dna序列的制备方法
WO2025235862A1 (en) 2024-05-10 2025-11-13 Inndura Therapeutics Inc. A modified immune cell receptor comprising a target-binding domain and the extracellular domain of cd16a

Family Cites Families (18)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP1320582A2 (en) * 2000-08-03 2003-06-25 Onyx Pharmaceuticals Adenovirus e1b-55k single amino acid mutants and methods of use
US7838219B2 (en) 2001-02-02 2010-11-23 Novici Biotech Llc Method of increasing complementarity in a heteroduplex
WO2004067753A2 (en) 2003-01-28 2004-08-12 Cellectis Use of meganucleases for inducing homologous recombination ex vivo and in toto in vertebrate somatic tissues and application thereof.
EP1591521A1 (en) 2004-04-30 2005-11-02 Cellectis I-Dmo I derivatives with enhanced activity at 37 degrees C and use thereof
WO2008153421A1 (en) * 2007-06-15 2008-12-18 Otago Innovation Limited Anticancer therapies
EP2412806B1 (en) 2010-07-28 2014-01-08 Institut Pasteur Use of terminal deoxynucleotidyl transferase for mutagenic DNA repair to generate variability, at a determined position in DNA
EP2633040B1 (en) 2010-10-27 2019-07-10 Cellectis Method for increasing the efficiency of double-strand break-induced mutagenesis
US9044492B2 (en) 2011-02-04 2015-06-02 Cellectis Sa Method for modulating the efficiency of double-strand break-induced mutagenesis
WO2013009525A1 (en) 2011-07-08 2013-01-17 Cellectis S.A. Method for increasing the efficiency of double-strand break-induced mutagenssis
US20130129668A1 (en) * 2011-09-01 2013-05-23 The Regents Of The University Of California Diagnosis and treatment of arthritis using epigenetics
WO2014093655A2 (en) * 2012-12-12 2014-06-19 The Broad Institute, Inc. Engineering and optimization of systems, methods and compositions for sequence manipulation with functional domains
US9234213B2 (en) * 2013-03-15 2016-01-12 System Biosciences, Llc Compositions and methods directed to CRISPR/Cas genomic engineering systems
WO2014165349A1 (en) 2013-04-04 2014-10-09 Trustees Of Dartmouth College Compositions and methods for in vivo excision of hiv-1 proviral dna
EP3038661B1 (en) * 2013-08-29 2023-12-27 Temple University Of The Commonwealth System Of Higher Education Methods and compositions for rna-guided treatment of hiv infection
WO2015048577A2 (en) * 2013-09-27 2015-04-02 Editas Medicine, Inc. Crispr-related methods and compositions
CN105899665B (zh) * 2013-10-17 2019-10-22 桑格摩生物科学股份有限公司 用于核酸酶介导的基因组工程改造的递送方法和组合物
US9943612B2 (en) * 2014-10-09 2018-04-17 Seattle Children's Hospital Long poly(A) plasmids and methods for introduction of long poly(A) sequences into the plasmid
US10584321B2 (en) * 2015-02-13 2020-03-10 University Of Massachusetts Compositions and methods for transient delivery of nucleases

Non-Patent Citations (2)

* Cited by examiner, † Cited by third party
Title
GenBank: ADV03652.1;Weaver,E.A.等;《genbank》;20110112;第1-2页 *
The Nuclear Export Signal within the E4orf6 Protein of Adenovirus Type 5 Supports Virus Replication and Cytoplasmic Accumulation of Viral mRNA;SILKE WEIGEL等;《American Society for Microbiology》;20001231;第74卷(第2期);第764-772页 *

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