EA201890565A1 - Композиции и способы улучшения специфичности в геномной инженерии с применением рнк-направляемых эндонуклеаз - Google Patents

Композиции и способы улучшения специфичности в геномной инженерии с применением рнк-направляемых эндонуклеаз

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EA201890565A1
EA201890565A1 EA201890565A EA201890565A EA201890565A1 EA 201890565 A1 EA201890565 A1 EA 201890565A1 EA 201890565 A EA201890565 A EA 201890565A EA 201890565 A EA201890565 A EA 201890565A EA 201890565 A1 EA201890565 A1 EA 201890565A1
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methods
endonucleas
rna
compositions
directed
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EA201890565A
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English (en)
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Эрик Джозефс
Девран Коцак
Петр Маршалек
Чарльз А. Герсбах
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Дьюк Юниверсити
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Abstract

В данном документе раскрываются оптимизированные направляющие РНК (gRNA) и способы конструирования и применения указанных оптимизированных gRNA, которые характеризуются повышенной специфичностью связывания с мишенью и уменьшенным нецелевым связыванием.
EA201890565A 2015-08-25 2016-08-25 Композиции и способы улучшения специфичности в геномной инженерии с применением рнк-направляемых эндонуклеаз EA201890565A1 (ru)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201562209466P 2015-08-25 2015-08-25
PCT/US2016/048798 WO2017035416A2 (en) 2015-08-25 2016-08-25 Compositions and methods of improving specificity in genomic engineering using rna-guided endonucleases

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EA201890565A1 true EA201890565A1 (ru) 2019-04-30

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US (3) US11427817B2 (ru)
EP (4) EP4177346A3 (ru)
JP (3) JP6905755B2 (ru)
KR (2) KR20240132120A (ru)
CN (2) CN114634930A (ru)
AU (2) AU2016311454C1 (ru)
BR (1) BR112018003625A2 (ru)
CA (1) CA2996001A1 (ru)
EA (1) EA201890565A1 (ru)
ES (1) ES2929110T3 (ru)
IL (2) IL288662B2 (ru)
MX (1) MX2018002339A (ru)
WO (1) WO2017035416A2 (ru)

Families Citing this family (61)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AU2012333134B2 (en) 2011-07-22 2017-05-25 John Paul Guilinger Evaluation and improvement of nuclease cleavage specificity
EP3597741A1 (en) 2012-04-27 2020-01-22 Duke University Genetic correction of mutated genes
US20150044192A1 (en) 2013-08-09 2015-02-12 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
US9322037B2 (en) 2013-09-06 2016-04-26 President And Fellows Of Harvard College Cas9-FokI fusion proteins and uses thereof
US9737604B2 (en) 2013-09-06 2017-08-22 President And Fellows Of Harvard College Use of cationic lipids to deliver CAS9
US20150166982A1 (en) 2013-12-12 2015-06-18 President And Fellows Of Harvard College Methods for correcting pi3k point mutations
US11028388B2 (en) 2014-03-05 2021-06-08 Editas Medicine, Inc. CRISPR/Cas-related methods and compositions for treating Usher syndrome and retinitis pigmentosa
US11339437B2 (en) 2014-03-10 2022-05-24 Editas Medicine, Inc. Compositions and methods for treating CEP290-associated disease
US9938521B2 (en) 2014-03-10 2018-04-10 Editas Medicine, Inc. CRISPR/CAS-related methods and compositions for treating leber's congenital amaurosis 10 (LCA10)
US11141493B2 (en) 2014-03-10 2021-10-12 Editas Medicine, Inc. Compositions and methods for treating CEP290-associated disease
EP3122880B1 (en) 2014-03-26 2021-05-05 Editas Medicine, Inc. Crispr/cas-related methods and compositions for treating sickle cell disease
US10077453B2 (en) 2014-07-30 2018-09-18 President And Fellows Of Harvard College CAS9 proteins including ligand-dependent inteins
US10676726B2 (en) 2015-02-09 2020-06-09 Duke University Compositions and methods for epigenome editing
AU2016253150B2 (en) 2015-04-24 2022-04-21 Editas Medicine, Inc. Evaluation of Cas9 molecule/guide RNA molecule complexes
WO2017066497A2 (en) 2015-10-13 2017-04-20 Duke University Genome engineering with type i crispr systems in eukaryotic cells
AU2016342380B2 (en) 2015-10-23 2022-04-07 President And Fellows Of Harvard College Nucleobase editors and uses thereof
WO2017165862A1 (en) 2016-03-25 2017-09-28 Editas Medicine, Inc. Systems and methods for treating alpha 1-antitrypsin (a1at) deficiency
AU2017305404B2 (en) 2016-08-02 2023-11-30 Editas Medicine, Inc. Compositions and methods for treating CEP290 associated disease
AU2017306676B2 (en) 2016-08-03 2024-02-22 President And Fellows Of Harvard College Adenosine nucleobase editors and uses thereof
CN109804066A (zh) 2016-08-09 2019-05-24 哈佛大学的校长及成员们 可编程cas9-重组酶融合蛋白及其用途
US11542509B2 (en) 2016-08-24 2023-01-03 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
WO2018098480A1 (en) * 2016-11-28 2018-05-31 The Board Of Regents Of The University Of Texas System Prevention of muscular dystrophy by crispr/cpf1-mediated gene editing
US10745677B2 (en) 2016-12-23 2020-08-18 President And Fellows Of Harvard College Editing of CCR5 receptor gene to protect against HIV infection
US12110545B2 (en) 2017-01-06 2024-10-08 Editas Medicine, Inc. Methods of assessing nuclease cleavage
US11898179B2 (en) 2017-03-09 2024-02-13 President And Fellows Of Harvard College Suppression of pain by gene editing
WO2018165629A1 (en) 2017-03-10 2018-09-13 President And Fellows Of Harvard College Cytosine to guanine base editor
WO2018170184A1 (en) 2017-03-14 2018-09-20 Editas Medicine, Inc. Systems and methods for the treatment of hemoglobinopathies
SG11201908658TA (en) 2017-03-23 2019-10-30 Harvard College Nucleobase editors comprising nucleic acid programmable dna binding proteins
EP3622070A2 (en) 2017-05-10 2020-03-18 Editas Medicine, Inc. Crispr/rna-guided nuclease systems and methods
WO2018209320A1 (en) 2017-05-12 2018-11-15 President And Fellows Of Harvard College Aptazyme-embedded guide rnas for use with crispr-cas9 in genome editing and transcriptional activation
JP2020534795A (ja) 2017-07-28 2020-12-03 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 ファージによって支援される連続的進化（ｐａｃｅ）を用いて塩基編集因子を進化させるための方法および組成物
EP3676376A2 (en) 2017-08-30 2020-07-08 President and Fellows of Harvard College High efficiency base editors comprising gam
JP2020536501A (ja) * 2017-09-28 2020-12-17 ツールゲン インコーポレイテッドＴｏｏｌｇｅｎ Ｉｎｃｏｒｐｏｒａｔｅｄ 遺伝子発現調節のための人為的なゲノム操作
US11572574B2 (en) 2017-09-28 2023-02-07 Toolgen Incorporated Artificial genome manipulation for gene expression regulation
CA3082251A1 (en) 2017-10-16 2019-04-25 The Broad Institute, Inc. Uses of adenosine base editors
WO2019089761A1 (en) * 2017-10-31 2019-05-09 The Broad Institute, Inc. Crispr protein inhibitors
CN111699254A (zh) 2018-02-08 2020-09-22 齐默尔根公司 使用crispr在棒状杆菌属中进行基因组编辑
CN112020558A (zh) 2018-03-14 2020-12-01 爱迪塔斯医药公司 治疗血红蛋白病的系统和方法
US11380420B1 (en) * 2018-05-07 2022-07-05 X Development Llc Data structure, compilation service, and graphical user interface for rapid simulation generation
WO2019222545A1 (en) 2018-05-16 2019-11-21 Synthego Corporation Methods and systems for guide rna design and use
US10839937B1 (en) * 2018-07-19 2020-11-17 X Development Llc Whole cell circular delta viewer and navigator
WO2020149395A1 (ja) * 2019-01-18 2020-07-23 国立大学法人大阪大学 栄養障害型表皮水疱症治療薬
CN114729365A (zh) 2019-03-19 2022-07-08 布罗德研究所股份有限公司 用于编辑核苷酸序列的方法和组合物
JP2022526669A (ja) * 2019-04-12 2022-05-25 デューク ユニバーシティ ジストロフィン機能を修復するためのＣＲＩＳＰＲ／Ｃａｓをベースにした塩基編集組成物
WO2020236645A1 (en) * 2019-05-17 2020-11-26 Beth Israel Deaconess Medical Center, Inc. Compositions and methods for homology directed repair
AU2020342207A1 (en) * 2019-09-04 2022-04-21 Edigene (Guangzhou) Inc. Method for evaluating gene editing therapy based on off-target assessment
CN110777159B (zh) * 2019-12-03 2023-05-05 苏州缔因安生物科技有限公司 一种基于CRISPR-Cas9的核酸检测系统及其应用
CN111172164B (zh) * 2020-03-09 2022-11-15 中国药科大学 一种用于任意核酸编辑的组合物及方法
EP4146804A1 (en) 2020-05-08 2023-03-15 The Broad Institute Inc. Methods and compositions for simultaneous editing of both strands of a target double-stranded nucleotide sequence
CN111781343B (zh) * 2020-07-17 2023-03-21 北京林业大学 一种检测dna甲基化修饰影响转录因子与dna序列结合的方法
JP2023540285A (ja) * 2020-08-31 2023-09-22 エタロン ダイアグノスティックス 所定の表現型又は遺伝子型を有する非ヒト動物を産生又は特定するためのシステム及び方法
CN112233730B (zh) * 2020-10-16 2023-11-28 南京大学 一种区分PBDEs衍生物对烯酰-ACP还原酶活性效应模型的构建方法
CN112662674B (zh) * 2021-01-12 2023-04-11 广州瑞风生物科技有限公司 靶向编辑VEGFA基因外显子区域的gRNA及其应用
EP4433598A1 (en) * 2021-11-17 2024-09-25 Cornell University Modulation of the crispr roadblock
CN114231596B (zh) * 2021-12-12 2023-11-21 徐州医科大学 基于CRISPR/dcas9和磁纳米材料的基因检测方法及应用
WO2023225349A2 (en) * 2022-05-20 2023-11-23 Helex Inc. Tissue specific methods and compositions for gene editing
WO2023250511A2 (en) 2022-06-24 2023-12-28 Tune Therapeutics, Inc. Compositions, systems, and methods for reducing low-density lipoprotein through targeted gene repression
CN118291415A (zh) * 2024-03-14 2024-07-05 内蒙古大学 一种CRISPR::Cr-P300M巴豆酰化修饰编辑工具及其应用

Family Cites Families (120)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5139941A (en) 1985-10-31 1992-08-18 University Of Florida Research Foundation, Inc. AAV transduction vectors
US5436146A (en) 1989-09-07 1995-07-25 The Trustees Of Princeton University Helper-free stocks of recombinant adeno-associated virus vectors
GB9206016D0 (en) 1992-03-19 1992-04-29 Sandoz Ltd Improvements in or relating to organic compounds
US6268213B1 (en) 1992-06-03 2001-07-31 Richard Jude Samulski Adeno-associated virus vector and cis-acting regulatory and promoter elements capable of expressing at least one gene and method of using same for gene therapy
IL105914A0 (en) 1992-06-04 1993-10-20 Univ California Methods and compositions for in vivo gene therapy
US5869305A (en) 1992-12-04 1999-02-09 The University Of Pittsburgh Recombinant viral vector system
US5593972A (en) 1993-01-26 1997-01-14 The Wistar Institute Genetic immunization
ES2249760T3 (es) 1993-01-26 2006-04-01 The Trustees Of The University Of Pennsylvania (A Corporation Of Pennsylvania) Composiciones y procedimientos de administracion de materias geneticas.
US5658784A (en) 1994-04-14 1997-08-19 Dana-Farber Cancer Institute, Inc. Nucleic acid encoding transcription factor p300 and uses of p300
US6204059B1 (en) 1994-06-30 2001-03-20 University Of Pittsburgh AAV capsid vehicles for molecular transfer
US5962428A (en) 1995-03-30 1999-10-05 Apollon, Inc. Compositions and methods for delivery of genetic material
US6093570A (en) 1995-06-07 2000-07-25 The University Of North Carolina At Chapel Hill Helper virus-free AAV production
US5741683A (en) 1995-06-07 1998-04-21 The Research Foundation Of State University Of New York In vitro packaging of adeno-associated virus DNA
ATE270324T1 (de) 1997-04-14 2004-07-15 Cell Genesys Inc Methoden zur erhöhung der effizienz rekombinanter aav produkte
EP1080218A1 (en) 1998-05-27 2001-03-07 University of Florida Method of preparing recombinant adeno-associated virus compositions by using an iodixanol gradient
CA2348382C (en) 1998-11-10 2013-09-17 The University Of North Carolina At Chapel Hill Chimeric parvovirus vectors and methods of making and administering the same
ES2478635T3 (es) 1999-08-09 2014-07-22 Targeted Genetics Corporation Incremento de la expresión de una secuencia de nucleótidos heteróloga monocatenaria de vectores virales recombinantes diseñando la secuencia de modo que forma pares de bases intracatenarios
US20020006397A1 (en) 2000-04-28 2002-01-17 Roberts Bruce L. In VIVO loading of MHC
AU6814901A (en) 2000-06-01 2001-12-11 Univ North Carolina Methods and compounds for controlled release of recombinant parvovirus vectors
CA2406745C (en) 2001-11-13 2006-01-10 The Trustees Of The University Of Pennsylvania A method of detecting and/or identifying adeno-associated virus (aav) sequences and isolating novel sequences identified thereby
PT2359869T (pt) 2001-12-17 2019-04-16 Univ Pennsylvania Sequências do vírus adeno-associado (aav) do serotipo 8, vetores contendo as mesmas, e utilizações destas
WO2003104413A2 (en) 2002-06-05 2003-12-18 University Of Florida Production of pseudotyped recombinant aav virions
US20040175727A1 (en) 2002-11-04 2004-09-09 Advisys, Inc. Synthetic muscle promoters with activities exceeding naturally occurring regulatory sequences in cardiac cells
CA3072423A1 (en) 2003-09-30 2005-04-14 The Trustees Of The University Of Pennsylvania Adeno-associated virus (aav) clades, sequences, vectors containing same, and uses therefor
EP2206781B1 (en) 2004-06-28 2015-12-02 The University Of Western Australia Antisense oligonucleotides for inducing exon skipping and methods of use thereof
ES2580044T3 (es) 2005-04-07 2016-08-18 The Trustees Of The University Of Pennsylvania Método de incremento de la función de un vector de AAV
WO2006132118A1 (ja) 2005-06-09 2006-12-14 Matsushita Electric Industrial Co., Ltd. 振幅誤差補償装置及び直交度誤差補償装置
EP2007795B1 (en) 2006-03-30 2016-11-16 The Board Of Trustees Of The Leland Stanford Junior University Aav capsid proteins
EP2046806A4 (en) 2006-07-05 2009-10-21 Scripps Research Inst CHIMERIC RECOMBINASES WITH ZINC FINGERS OPTIMIZED FOR CATALYSIS BY DIRECT EVOLUTION
PT2203173E (pt) 2007-10-26 2016-03-15 Academisch Ziekenhuis Leiden Resumo
EP2620161A1 (en) 2008-05-13 2013-07-31 University of Washington Diblock copolymers and polynucleotide complexes thereof for delivery into cells
ES2560281T3 (es) 2008-10-17 2016-02-18 Joule Unlimited Technologies, Inc. Producción de etanol por microorganismos
EP2364330B1 (en) 2008-11-06 2015-03-25 University Of Washington Multiblock copolymers
KR101734955B1 (ko) 2008-11-07 2017-05-12 메사추세츠 인스티튜트 오브 테크놀로지 아미노알콜 리피도이드 및 그의 용도
EP2206723A1 (en) 2009-01-12 2010-07-14 Bonas, Ulla Modular DNA-binding domains
EP2281579A1 (en) 2009-08-05 2011-02-09 BioNTech AG Vaccine composition comprising 5'-Cap modified RNA
WO2011036640A2 (en) 2009-09-24 2011-03-31 Cellectis Meganuclease reagents of uses thereof for treating genetic diseases caused by frame shift/non sense mutations
WO2011100505A2 (en) 2010-02-11 2011-08-18 Recombinetics, Inc. Methods and materials for producing transgenic artiodactyls
US9315825B2 (en) 2010-03-29 2016-04-19 The Trustees Of The University Of Pennsylvania Pharmacologically induced transgene ablation system
JP5922095B2 (ja) 2010-03-29 2016-05-24 ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア 薬理学的に誘導される導入遺伝子アブレーション系
CA2799095A1 (en) 2010-05-12 2011-11-17 Cellectis Meganuclease variants cleaving a dna target sequence from the dystrophin gene and uses thereof
EP3156062A1 (en) 2010-05-17 2017-04-19 Sangamo BioSciences, Inc. Novel dna-binding proteins and uses thereof
IT1400425B1 (it) 2010-06-08 2013-05-31 Amsterdam Molecular Therapeutics Bv Modified snrnas for use in therapy.
WO2012153638A1 (ja) 2011-05-12 2012-11-15 日立建機株式会社 建設機械
BR112013031553A2 (pt) 2011-06-08 2020-11-10 Shire Human Genetic Therapies, Inc. composições, mrna que codifica para uma hgla e seu uso, uso de pelo menos uma molécula de mrna e um veículo de transferência e uso de um mrna que codifica para proteína exógena
WO2013009525A1 (en) 2011-07-08 2013-01-17 Cellectis S.A. Method for increasing the efficiency of double-strand break-induced mutagenssis
WO2013112053A1 (en) 2012-01-27 2013-08-01 Prosensa Technologies B.V. Rna modulating oligonucleotides with improved characteristics for the treatment of duchenne and becker muscular dystrophy
WO2013143555A1 (en) 2012-03-26 2013-10-03 Biontech Ag Rna formulation for immunotherapy
US20130274129A1 (en) 2012-04-04 2013-10-17 Geneart Ag Tal-effector assembly platform, customized services, kits and assays
US9738879B2 (en) 2012-04-27 2017-08-22 Duke University Genetic correction of mutated genes
EP3597741A1 (en) 2012-04-27 2020-01-22 Duke University Genetic correction of mutated genes
RS64622B1 (sr) 2012-05-25 2023-10-31 Univ California Metode i sastavi za modifikaciju ciljane dnk upravljenu pomoću rnk i za modulaciju transkripcije upravljanu rnk
EP3800254A1 (en) 2012-06-08 2021-04-07 Ethris GmbH Pulmonary delivery of messenger rna
EP3494997B1 (en) * 2012-07-25 2019-09-18 The Broad Institute, Inc. Inducible dna binding proteins and genome perturbation tools and applications thereof
BR112015008708A2 (pt) 2012-10-23 2017-09-26 Toolgen Inc composição para clivagem de dna alvo compreendendo um rna guia específico para o dna alvo e ácido nucleico que codifica proteína cas ou proteína cas e uso da mesma
EP2914728B1 (en) 2012-11-01 2020-07-08 Factor Bioscience Inc. Methods and products for expressing proteins in cells
WO2014081855A1 (en) 2012-11-20 2014-05-30 Universite De Montreal Methods and compositions for muscular dystrophies
ES2757808T3 (es) 2012-12-06 2020-04-30 Sigma Aldrich Co Llc Modificación y regulación del genoma basada en CRISPR
WO2014093694A1 (en) 2012-12-12 2014-06-19 The Broad Institute, Inc. Crispr-cas nickase systems, methods and compositions for sequence manipulation in eukaryotes
WO2014093701A1 (en) 2012-12-12 2014-06-19 The Broad Institute, Inc. Functional genomics using crispr-cas systems, compositions, methods, knock out libraries and applications thereof
PT2771468E (pt) * 2012-12-12 2015-06-02 Harvard College Engenharia de sistemas, métodos e composições-guia otimizadas para manipulação de sequências
PL2931898T3 (pl) 2012-12-12 2016-09-30 Le Cong Projektowanie i optymalizacja systemów, sposoby i kompozycje do manipulacji sekwencją z domenami funkcjonalnymi
EP3327127B1 (en) * 2012-12-12 2021-03-24 The Broad Institute, Inc. Delivery, engineering and optimization of systems, methods and compositions for sequence manipulation and therapeutic applications
BR122021008308B1 (pt) * 2012-12-12 2022-12-27 The Broad Institute, Inc. Sistemas de componente crispr-cas, métodos e composições para manipulação de sequência
EP3434776A1 (en) 2012-12-12 2019-01-30 The Broad Institute, Inc. Methods, models, systems, and apparatus for identifying target sequences for cas enzymes or crispr-cas systems for target sequences and conveying results thereof
US8697359B1 (en) 2012-12-12 2014-04-15 The Broad Institute, Inc. CRISPR-Cas systems and methods for altering expression of gene products
JP6663841B2 (ja) 2013-03-15 2020-03-13 ブリット，エドワード，ジェー． エネルギー変換デバイスならびにその作成および使用方法
EP2971041B1 (en) 2013-03-15 2018-11-28 The General Hospital Corporation Using rna-guided foki nucleases (rfns) to increase specificity for rna-guided genome editing
WO2014204578A1 (en) 2013-06-21 2014-12-24 The General Hospital Corporation Using rna-guided foki nucleases (rfns) to increase specificity for rna-guided genome editing
US10760064B2 (en) 2013-03-15 2020-09-01 The General Hospital Corporation RNA-guided targeting of genetic and epigenomic regulatory proteins to specific genomic loci
US9828582B2 (en) 2013-03-19 2017-11-28 Duke University Compositions and methods for the induction and tuning of gene expression
WO2014172470A2 (en) 2013-04-16 2014-10-23 Whitehead Institute For Biomedical Research Methods of mutating, modifying or modulating nucleic acid in a cell or nonhuman mammal
AU2014265331B2 (en) 2013-05-15 2019-12-05 Sangamo Therapeutics, Inc. Methods and compositions for treatment of a genetic condition
AU2014273490B2 (en) 2013-05-29 2019-05-09 Cellectis Methods for engineering T cells for immunotherapy by using RNA-guided Cas nuclease system
US9267135B2 (en) 2013-06-04 2016-02-23 President And Fellows Of Harvard College RNA-guided transcriptional regulation
CA2914519A1 (en) 2013-06-05 2014-12-11 Duke University Rna-guided gene editing and gene regulation
MX2015017311A (es) * 2013-06-17 2017-04-10 Broad Inst Inc Suministro, modificación y optimización de sistemas, métodos y composiciones para generar modelos y actuar sobre enfermedades y trastornos de células posmitóticas.
RU2716420C2 (ru) * 2013-06-17 2020-03-11 Те Брод Инститьют Инк. Доставка и применение систем crispr-cas, векторов и композиций для целенаправленного воздействия и терапии в печени
US10421957B2 (en) 2013-07-29 2019-09-24 Agilent Technologies, Inc. DNA assembly using an RNA-programmable nickase
AU2014296278C1 (en) 2013-07-30 2023-02-02 Genevant Sciences Gmbh Block copolymers and their conjugates or complexes with oligonucleotides
US9340800B2 (en) * 2013-09-06 2016-05-17 President And Fellows Of Harvard College Extended DNA-sensing GRNAS
US10822606B2 (en) * 2013-09-27 2020-11-03 The Regents Of The University Of California Optimized small guide RNAs and methods of use
CN111218447B (zh) 2013-11-07 2024-10-11 爱迪塔斯医药有限公司 使用统治型gRNA的CRISPR相关方法和组合物
SG10201804976YA (en) * 2013-12-12 2018-07-30 Broad Inst Inc Delivery, Use and Therapeutic Applications of the Crispr-Cas Systems and Compositions for Genome Editing
CN106536729A (zh) * 2013-12-12 2017-03-22 布罗德研究所有限公司 使用粒子递送组分靶向障碍和疾病的crispr‑cas系统和组合物的递送、用途和治疗应用
WO2015089486A2 (en) 2013-12-12 2015-06-18 The Broad Institute Inc. Systems, methods and compositions for sequence manipulation with optimized functional crispr-cas systems
US20150166982A1 (en) 2013-12-12 2015-06-18 President And Fellows Of Harvard College Methods for correcting pi3k point mutations
RU2016128077A (ru) 2013-12-12 2018-12-06 Те Брод Инститьют Инк. Доставка, применение и применения в терапии систем и композиций crispr-cas для лечения обусловленных hbv и вирусных заболеваний и нарушений
CN106232823A (zh) 2014-02-18 2016-12-14 杜克大学 灭活病毒复制的组合物及其制备和使用方法
US9932566B2 (en) 2014-08-07 2018-04-03 Agilent Technologies, Inc. CIS-blocked guide RNA
EP3985115A1 (en) 2014-12-12 2022-04-20 The Broad Institute, Inc. Protected guide rnas (pgrnas)
WO2016094880A1 (en) 2014-12-12 2016-06-16 The Broad Institute Inc. Delivery, use and therapeutic applications of crispr systems and compositions for genome editing as to hematopoietic stem cells (hscs)
US10190106B2 (en) 2014-12-22 2019-01-29 Univesity Of Massachusetts Cas9-DNA targeting unit chimeras
US20180002706A1 (en) 2014-12-30 2018-01-04 University Of South Florida Methods and compositions for cloning into large vectors
US10676726B2 (en) 2015-02-09 2020-06-09 Duke University Compositions and methods for epigenome editing
CA2978314A1 (en) 2015-03-03 2016-09-09 The General Hospital Corporation Engineered crispr-cas9 nucleases with altered pam specificity
EP3325018A4 (en) 2015-07-22 2019-04-24 Duke University HIGH EFFICIENCY SCREENING OF REGULATORY ELEMENT FUNCTION USING EPIGENOUS EDITING TECHNOLOGIES
WO2017066497A2 (en) 2015-10-13 2017-04-20 Duke University Genome engineering with type i crispr systems in eukaryotic cells
EA201891317A3 (ru) 2015-11-30 2019-04-30 Дьюк Юниверсити Терапевтические мишени для коррекции гена дистрофина человека с помощью редактирования генов и способы их применения
WO2017180915A2 (en) 2016-04-13 2017-10-19 Duke University Crispr/cas9-based repressors for silencing gene targets in vivo and methods of use
ES2957660T3 (es) 2016-05-05 2024-01-23 Univ Duke Composiciones relacionadas con crispr/cas para tratar la distrofia muscular de duchenne
US20190151476A1 (en) 2016-07-19 2019-05-23 Duke University Therapeutic applications of cpf1-based genome editing
US20190194633A1 (en) 2016-08-10 2019-06-27 Duke University Compositions, systems and methods for programming immune cell function through targeted gene regulation
EP3500675A4 (en) 2016-08-19 2020-01-29 Whitehead Institute for Biomedical Research METHOD FOR EDITING DNA METHYLATION
EP3579858A4 (en) 2017-02-07 2020-12-23 The Regents of The University of California GENE THERAPY AGAINST HAPLOINSUFFICIENCY
EP3740580A4 (en) 2018-01-19 2021-10-20 Duke University GENOME ENGINEERING WITH CRISPR-CAS SYSTEMS IN EUKARYONTS
JP2022504075A (ja) 2018-10-02 2022-01-13 サンガモ セラピューティクス， インコーポレイテッド 操作された遺伝子モジュレーター
JP2022526669A (ja) 2019-04-12 2022-05-25 デューク ユニバーシティ ジストロフィン機能を修復するためのＣＲＩＳＰＲ／Ｃａｓをベースにした塩基編集組成物
CN113785063A (zh) 2019-04-14 2021-12-10 杜克大学 用于治疗杜氏肌营养不良症的aav载体介导的大规模突变热点缺失
WO2020214613A1 (en) 2019-04-14 2020-10-22 Duke University Crispr/cas-based genome editing composition for restoring dystrophin function
US20220396790A1 (en) 2019-08-08 2022-12-15 Duke University High-throughput screening platform for engineering next-generation gene therapy vectors
US20220305141A1 (en) 2019-08-19 2022-09-29 Duke University Skeletal myoblast progenitor cell lineage specification by crispr/cas9-based transcriptional activators
KR20220047623A (ko) 2019-08-19 2022-04-18 듀크 유니버시티 세포 유형 운명 특정화의 조절인자를 확인하기 위한 조성물 및 방법
EP4038204A4 (en) 2019-10-02 2023-09-13 Duke University EPIGENETIC MODULATION OF GENOMIC TARGETS TO CONTROL EXPRESSION OF PWS-ASSOCIATED GENES
EP4069314A4 (en) 2019-12-03 2024-06-26 Duke University SYSTEMS AND METHODS FOR LIPID NANOPARTICLE DELIVERY OF GENE EDITING MACHINERY
EP4125349A4 (en) 2020-04-27 2024-07-10 Univ Duke GENE EDITING OF SATELLITE CELLS IN VIVO USING AAV VECTORS ENCODING MUSCLE-SPECIFIC PROMOTERS
JP2023515710A (ja) 2020-04-27 2023-04-13 デューク ユニバーシティ ＣＲＩＳＰＲ媒介式エクソン欠失用の最適なｇＲＮＡ対を発見するためのハイスループットスクリーニング法
WO2021222328A1 (en) 2020-04-27 2021-11-04 Duke University Targeted genomic integration to restore neurofibromin coding sequence in neurofibromatosis type 1 (nf1)
US20230257723A1 (en) 2020-04-27 2023-08-17 Duke University Crispr/cas9 therapies for correcting duchenne muscular dystrophy by targeted genomic integration
CA3182672A1 (en) 2020-05-08 2021-11-11 Duke University Chromatin remodelers to enhance targeted gene activation

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