JP2020513814A5 - - Google Patents

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Publication number
JP2020513814A5
JP2020513814A5 JP2019550861A JP2019550861A JP2020513814A5 JP 2020513814 A5 JP2020513814 A5 JP 2020513814A5 JP 2019550861 A JP2019550861 A JP 2019550861A JP 2019550861 A JP2019550861 A JP 2019550861A JP 2020513814 A5 JP2020513814 A5 JP 2020513814A5
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JP
Japan
Prior art keywords
viral vector
cas9
composition
antigenic escape
disease
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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.)
Pending
Application number
JP2019550861A
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English (en)
Japanese (ja)
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JP2020513814A (ja
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Publication date
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Priority claimed from PCT/US2018/022258 external-priority patent/WO2018170015A1/en
Publication of JP2020513814A publication Critical patent/JP2020513814A/ja
Publication of JP2020513814A5 publication Critical patent/JP2020513814A5/ja
Pending legal-status Critical Current

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JP2019550861A 2017-03-14 2018-03-13 Crispr cas9免疫ステルス(immune stealth)の操作 Pending JP2020513814A (ja)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201762471267P 2017-03-14 2017-03-14
US62/471,267 2017-03-14
US201862614875P 2018-01-08 2018-01-08
US62/614,875 2018-01-08
PCT/US2018/022258 WO2018170015A1 (en) 2017-03-14 2018-03-13 Engineering crispr cas9 immune stealth

Publications (2)

Publication Number Publication Date
JP2020513814A JP2020513814A (ja) 2020-05-21
JP2020513814A5 true JP2020513814A5 (https=) 2021-09-02

Family

ID=63522593

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2019550861A Pending JP2020513814A (ja) 2017-03-14 2018-03-13 Crispr cas9免疫ステルス(immune stealth)の操作

Country Status (7)

Country Link
US (1) US11332727B2 (https=)
EP (1) EP3595694A4 (https=)
JP (1) JP2020513814A (https=)
CN (1) CN110913881A (https=)
GB (1) GB2574561A (https=)
IL (1) IL268829A (https=)
WO (1) WO2018170015A1 (https=)

Families Citing this family (56)

* 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
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
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
AU2015330699B2 (en) 2014-10-10 2021-12-02 Editas Medicine, Inc. Compositions and methods for promoting homology directed repair
EP4464338A3 (en) 2014-11-07 2025-02-12 Editas Medicine, Inc. Systems for improving crispr/cas-mediated genome-editing
CA2986310A1 (en) 2015-05-11 2016-11-17 Editas Medicine, Inc. Optimized crispr/cas9 systems and methods for gene editing in stem cells
CN108026526B (zh) 2015-06-09 2023-05-12 爱迪塔斯医药公司 用于改善移植的crispr/cas相关方法和组合物
EP3353296B1 (en) 2015-09-24 2020-11-04 Editas Medicine, Inc. Use of exonucleases to improve crispr/cas-mediated genome editing
IL310721B2 (en) 2015-10-23 2025-11-01 Harvard College Nucleobase editors and uses thereof
US11597924B2 (en) 2016-03-25 2023-03-07 Editas Medicine, Inc. Genome editing systems comprising repair-modulating enzyme molecules and methods of their use
US11236313B2 (en) 2016-04-13 2022-02-01 Editas Medicine, Inc. Cas9 fusion molecules, gene editing systems, and methods of use thereof
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
EP3555297A1 (en) 2016-12-19 2019-10-23 Editas Medicine, Inc. Assessing nuclease cleavage
WO2018119359A1 (en) 2016-12-23 2018-06-28 President And Fellows Of Harvard College Editing of ccr5 receptor gene to protect against hiv infection
JP7229923B2 (ja) 2017-01-06 2023-02-28 エディタス・メディシン、インコーポレイテッド ヌクレアーゼ切断を評価する方法
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
US11499151B2 (en) 2017-04-28 2022-11-15 Editas Medicine, Inc. Methods and systems for analyzing guide RNA molecules
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
MX2019014640A (es) 2017-06-09 2020-10-05 Editas Medicine Inc Nucleasas de repeticiones palíndromicas agrupadas y regularmente interespaciadas de proteina asociada 9 (cas9) genomanipuladas.
US11866726B2 (en) 2017-07-14 2024-01-09 Editas Medicine, Inc. Systems and methods for targeted integration and genome editing and detection thereof using integrated priming sites
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
AU2019247748A1 (en) * 2018-04-03 2020-10-08 Ginkgo Bioworks, Inc. Antibody-evading virus vectors
US12157760B2 (en) 2018-05-23 2024-12-03 The Broad Institute, Inc. Base editors and uses thereof
EP3823633A4 (en) 2018-06-29 2023-05-03 Editas Medicine, Inc. SYNTHETIC LEAD MOLECULES, COMPOSITIONS AND METHODS RELATED THERETO
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
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
US12473543B2 (en) 2019-04-17 2025-11-18 The Broad Institute, Inc. Adenine base editors with reduced off-target effects
CN110499334A (zh) * 2019-08-08 2019-11-26 复旦大学 CRISPR/SlugCas9基因编辑系统及其应用
US12435330B2 (en) 2019-10-10 2025-10-07 The Broad Institute, Inc. Methods and compositions for prime editing RNA
CA3177869A1 (en) * 2020-05-05 2021-11-11 Duke University Cross-species compatible adeno-associated virus compositions and methods of use thereof
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
IL298134A (en) * 2020-05-12 2023-01-01 Lonza Houston Inc Methods and kits for the detection of adeno-associated viruses
CN113717960B (zh) * 2021-08-27 2023-07-18 电子科技大学 一种新Cas9蛋白、CRISPR-Cas9基因组定向编辑载体及基因组编辑方法
WO2024112479A1 (en) * 2022-11-22 2024-05-30 The Regents Of The University Of California Crispr-cas effector polypeptides, lipid nanoparticles, and methods of use thereof
WO2024211456A1 (en) * 2023-04-03 2024-10-10 The Broad Institute, Inc. Engineered type ii cas polynucleotides with reduced immunogenicity and uses thereof
CN116751764B (zh) * 2023-05-08 2024-01-30 珠海舒桐医疗科技有限公司 一种Cas9蛋白、II型CRISPR/Cas9基因编辑系统及应用
US20250001010A1 (en) 2023-06-30 2025-01-02 Christiana Care Gene Editing Institute, Inc. Nras gene knockout for treatment of cancer
WO2025190256A1 (en) * 2024-03-11 2025-09-18 Geneditbio Limited Type ii cas protein and uses thereof
US20250319206A1 (en) 2024-04-04 2025-10-16 Christina Care Gene Editing Institute, Inc. Crispr/cas gene editing of neh4 and/or neh5 domains in nrf2
WO2025226816A1 (en) 2024-04-23 2025-10-30 Christiana Care Gene Editing Institute, Inc. Methods of identifying and correlating crispr-induced exon skipping to phenotypic outcomes

Family Cites Families (11)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US6730507B1 (en) 1993-06-24 2004-05-04 Merck & Co., Inc. Use of helper-dependent adenoviral vectors of alternative serotypes permits repeat vector administration
CN100522242C (zh) * 2001-02-19 2009-08-05 默克专利有限公司 具有降低的免疫原性的人工蛋白质
CN1524090A (zh) * 2001-03-20 2004-08-25 Ĭ��ר�����޹�˾ 免疫原性减弱的经修饰胰岛素
BR0211091A (pt) * 2001-07-13 2004-06-15 Merck Patent Gmbh Métodos para redução de imunogenicidade de polipeptìdeos
AU2013266968B2 (en) * 2012-05-25 2017-06-29 Emmanuelle CHARPENTIER Methods and compositions for RNA-directed target DNA modification and for RNA-directed modulation of transcription
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
EP3003391B1 (en) * 2013-05-31 2021-09-22 The Regents of The University of California Adeno-associated virus variants and methods of use thereof
MX374532B (es) * 2013-06-17 2025-03-06 Broad Inst Inc Suministro, uso y aplicaciones terapéuticas de los sistemas y composiciones crispr-cas, para actuar sobre trastornos y enfermedades utilizando componentes víricos.
EP3666892A1 (en) * 2013-07-10 2020-06-17 President and Fellows of Harvard College Orthogonal cas9 proteins for rna-guided gene regulation and editing
WO2015153789A1 (en) * 2014-04-01 2015-10-08 Editas Medicine, Inc. Crispr/cas-related methods and compositions for treating herpes simplex virus type 1 (hsv-1)
SG11201701245QA (en) * 2014-08-27 2017-03-30 Caribou Biosciences Inc Methods for increasing cas9-mediated engineering efficiency

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