JP2019518454A5 - - Google Patents

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JP2019518454A5
JP2019518454A5 JP2018560977A JP2018560977A JP2019518454A5 JP 2019518454 A5 JP2019518454 A5 JP 2019518454A5 JP 2018560977 A JP2018560977 A JP 2018560977A JP 2018560977 A JP2018560977 A JP 2018560977A JP 2019518454 A5 JP2019518454 A5 JP 2019518454A5
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human
mouse
guide rna
human animal
antigen
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JP2019518454A (ja
JP7324583B2 (ja
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JP2018560977A 2016-05-20 2017-05-19 複数のガイドｒｎａを使用して免疫寛容を破綻させるための方法 Active JP7324583B2 (ja)

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JP2023060156A JP2023078479A (ja) 2016-05-20 2023-04-03 複数のガイドｒｎａを使用して免疫寛容を破綻させるための方法
JP2025127388A JP2025159002A (ja) 2016-05-20 2025-07-30 複数のガイドｒｎａを使用して免疫寛容を破綻させるための方法

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US201662339472P 2016-05-20 2016-05-20
US62/339,472 2016-05-20
US201662368604P 2016-07-29 2016-07-29
US62/368,604 2016-07-29
PCT/US2017/033648 WO2017201476A1 (en) 2016-05-20 2017-05-19 Methods for breaking immunological tolerance using multiple guide rnas

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JP2019518454A JP2019518454A (ja) 2019-07-04
JP2019518454A5 true JP2019518454A5 (enExample) 2020-06-18
JP7324583B2 JP7324583B2 (ja) 2023-08-10

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JP2023060156A Pending JP2023078479A (ja) 2016-05-20 2023-04-03 複数のガイドｒｎａを使用して免疫寛容を破綻させるための方法
JP2025127388A Pending JP2025159002A (ja) 2016-05-20 2025-07-30 複数のガイドｒｎａを使用して免疫寛容を破綻させるための方法

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JP2025127388A Pending JP2025159002A (ja) 2016-05-20 2025-07-30 複数のガイドｒｎａを使用して免疫寛容を破綻させるための方法

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US (3) US20170332610A1 (enExample)
EP (2) EP4368637A3 (enExample)
JP (3) JP7324583B2 (enExample)
KR (3) KR102356542B1 (enExample)
CN (2) CN109475109B (enExample)
AU (2) AU2017268458B2 (enExample)
BR (1) BR112018073750A2 (enExample)
CA (1) CA3022997C (enExample)
ES (1) ES2977547T3 (enExample)
IL (2) IL262888B1 (enExample)
MX (1) MX2018014172A (enExample)
NZ (1) NZ747857A (enExample)
RU (1) RU2745563C2 (enExample)
SG (2) SG10202102885UA (enExample)
WO (1) WO2017201476A1 (enExample)

Families Citing this family (50)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2013066438A2 (en) 2011-07-22 2013-05-10 President And Fellows Of Harvard College Evaluation and improvement of nuclease cleavage specificity
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
US9340799B2 (en) 2013-09-06 2016-05-17 President And Fellows Of Harvard College MRNA-sensing switchable gRNAs
US9388430B2 (en) 2013-09-06 2016-07-12 President And Fellows Of Harvard College Cas9-recombinase 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
US11053481B2 (en) 2013-12-12 2021-07-06 President And Fellows Of Harvard College Fusions of Cas9 domains and nucleic acid-editing domains
AU2015298571B2 (en) 2014-07-30 2020-09-03 President And Fellows Of Harvard College Cas9 proteins including ligand-dependent inteins
CA3176380A1 (en) 2014-11-21 2016-05-26 Regeneron Pharmaceuticals, Inc. Methods and compositions for targeted genetic modification using paired guide rnas
US12043852B2 (en) 2015-10-23 2024-07-23 President And Fellows Of Harvard College Evolved Cas9 proteins for gene editing
WO2017201476A1 (en) 2016-05-20 2017-11-23 Regeneron Pharmaceuticals, Inc. Methods for breaking immunological tolerance using multiple guide rnas
KR20250103795A (ko) 2016-08-03 2025-07-07 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 아데노신 핵염기 편집제 및 그의 용도
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
JP6958917B2 (ja) * 2016-08-10 2021-11-02 国立大学法人 東京医科歯科大学 遺伝子ノックイン細胞の作製方法
US11542509B2 (en) 2016-08-24 2023-01-03 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
WO2018119359A1 (en) 2016-12-23 2018-06-28 President And Fellows Of Harvard College Editing of ccr5 receptor gene to protect against hiv infection
EP3592853A1 (en) 2017-03-09 2020-01-15 President and Fellows of Harvard College Suppression of pain by gene editing
EP3592381A1 (en) 2017-03-09 2020-01-15 President and Fellows of Harvard College Cancer vaccine
KR20190127797A (ko) 2017-03-10 2019-11-13 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 시토신에서 구아닌으로의 염기 편집제
CA3057192A1 (en) 2017-03-23 2018-09-27 President And Fellows Of Harvard College Nucleobase editors comprising nucleic acid programmable dna binding proteins
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
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
WO2019139645A2 (en) 2017-08-30 2019-07-18 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
EP3724214A4 (en) 2017-12-15 2021-09-01 The Broad Institute Inc. SYSTEMS AND METHODS FOR PREDICTING REPAIR RESULTS IN GENETIC ENGINEERING
US20210002668A1 (en) * 2018-03-07 2021-01-07 Icahn School Of Medicine At Mount Sinai Sphingolipid-metabolizing proteins enhance the efficiency of gene editing in cells
SMT202500038T1 (it) 2018-03-24 2025-03-12 Regeneron Pharma Topi o ratti geneticamente modificati per la generazione di anticorpi terapeutici contro complessi peptide-mhc, metodi di produzione e loro utilizzo
KR20250121150A (ko) 2018-03-26 2025-08-11 리제너론 파마슈티칼스 인코포레이티드 치료제를 시험하기 위한 인간화된 설치류
EP3781683A4 (en) * 2018-04-18 2022-02-16 Ligandal, Inc. Methods and compositions for genome editing
KR20210045360A (ko) 2018-05-16 2021-04-26 신테고 코포레이션 가이드 rna 설계 및 사용을 위한 방법 및 시스템
WO2019226953A1 (en) 2018-05-23 2019-11-28 The Broad Institute, Inc. Base editors and uses thereof
SG11202012785VA (en) * 2018-06-26 2021-01-28 Broad Inst Inc Crispr double nickase based amplification compositions, systems, and methods
US20210244827A1 (en) 2018-06-29 2021-08-12 Icahn School Of Medicine At Mount Sinai Anc80 encoding sphingolipid-metabolizing proteins
US12281338B2 (en) 2018-10-29 2025-04-22 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
AU2020226445B2 (en) 2019-02-18 2025-05-29 Biocytogen Pharmaceuticals (Beijing) Co., Ltd. Genetically modified non-human animals with humanized immunoglobulin locus
DE112020001306T5 (de) 2019-03-19 2022-01-27 Massachusetts Institute Of Technology Verfahren und zusammensetzungen zur editierung von nukleotidsequenzen
US12473543B2 (en) 2019-04-17 2025-11-18 The Broad Institute, Inc. Adenine base editors with reduced off-target effects
EP3997222A4 (en) * 2019-07-12 2023-08-09 Duke University 3' UTR CRISPR DCAS 13 ENGINEERING SYSTEM AND METHODS OF USE
DK3785536T4 (da) * 2019-08-28 2025-10-27 Trianni Inc Adam6-knockin-mus
US12435330B2 (en) 2019-10-10 2025-10-07 The Broad Institute, Inc. Methods and compositions for prime editing RNA
WO2021113297A1 (en) 2019-12-02 2021-06-10 Regeneron Pharmaceuticals, Inc. Peptide-mhc ii protein constructs and uses thereof
KR20210095781A (ko) 2020-01-24 2021-08-03 주식회사 에이프릴바이오 항원결합 단편 및 생리활성 이펙터 모이어티로 구성된 융합 컨스트럭트를 포함하는 다중결합항체 및 이를 포함하는 약학조성물
JP2023525304A (ja) 2020-05-08 2023-06-15 ザ ブロード インスティテュート，インコーポレーテッド 標的二本鎖ヌクレオチド配列の両鎖同時編集のための方法および組成物
AU2021283564B2 (en) 2020-06-02 2025-05-29 Biocytogen Pharmaceuticals (Beijing) Co., Ltd. Genetically modified non-human animals with common light chain immunoglobulin locus
JP7419168B2 (ja) * 2020-06-10 2024-01-22 株式会社東芝 改変型ｐｉｇｇｙＢａｃトランスポゼースのポリペプチド、それをコードするポリヌクレオチド、導入キャリア、キット、細胞のゲノムに目的配列を組込む方法及び細胞製造方法
EP4130263A1 (en) * 2021-08-04 2023-02-08 Eberhard Karls Universität Tübingen Medizinische Fakultät Compound and method for a specific targeting of the hax1 gene
CN118251491A (zh) * 2021-10-28 2024-06-25 瑞泽恩制药公司 用于敲除C5的CRISPR/Cas相关方法及组合物
US20250059570A1 (en) * 2021-12-24 2025-02-20 Osaka University Method for producing genome-edited cells utilizing homologous recombination

Family Cites Families (159)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5523226A (en) * 1993-05-14 1996-06-04 Biotechnology Research And Development Corp. Transgenic swine compositions and methods
EP0721346B1 (en) 1993-09-02 1998-06-10 Trustees of Dartmouth College Methods for inducing antigen-specific t cell tolerance
US7119248B1 (en) * 1994-04-12 2006-10-10 Miltenyi Biotec Gmbh Antibodies against epitopes with homology to self antigens, methods of preparation and applications thereof
WO1999005266A2 (en) 1997-07-26 1999-02-04 Wisconsin Alumni Research Foundation Trans-species nuclear transfer
ATE387909T1 (de) 2000-05-31 2008-03-15 Novartis Vaccines & Diagnostic Verfahren und zusammensetzungen zur behandlung von neoplastischen erkrankungen mittels chemotherapie und strahlungssensibilisatoren
US20050144655A1 (en) 2000-10-31 2005-06-30 Economides Aris N. Methods of modifying eukaryotic cells
US7105348B2 (en) 2000-10-31 2006-09-12 Regeneron Pharmaceuticals, Inc. Methods of modifying eukaryotic cells
US6586251B2 (en) 2000-10-31 2003-07-01 Regeneron Pharmaceuticals, Inc. Methods of modifying eukaryotic cells
US6596541B2 (en) 2000-10-31 2003-07-22 Regeneron Pharmaceuticals, Inc. Methods of modifying eukaryotic cells
AUPR451401A0 (en) 2001-04-20 2001-05-24 Monash University A method of nuclear transfer
US7612250B2 (en) 2002-07-29 2009-11-03 Trustees Of Tufts College Nuclear transfer embryo formation method
CA2547077C (en) 2003-12-01 2015-11-03 Kudos Pharmaceuticals Limited Dna damage repair inhibitors for treatment of cancer
EP1802193B1 (en) 2004-10-19 2014-04-30 Regeneron Pharmaceuticals, Inc. Method for generating a mouse homozygous for a genetic modification
JP2006187215A (ja) * 2004-12-28 2006-07-20 Univ Of Tsukuba ベクターによる遺伝子改変型樹状細胞を用いた抗体作製方法
CA2652976C (en) 2006-06-02 2015-08-11 Regeneron Pharmaceuticals, Inc. High affinity antibodies to human il-6 receptor
CN101117633B (zh) 2006-08-03 2011-07-20 上海交通大学附属儿童医院 一种细胞核移植方法
WO2008070367A2 (en) * 2006-11-01 2008-06-12 Facet Biotech Corporation Mammalian cell-based immunoglobulin display libraries
KR20100120226A (ko) 2008-04-11 2010-11-12 유티씨 파워 코포레이션 매니폴드 섬프를 갖는 바이폴라 플레이트 및 연료 전지
MX368932B (es) 2009-06-26 2019-10-22 Regeneron Pharma Anticuerpos biespecificos facilmente aislados con formato de inmunoglobulina original.
EP3028565B1 (en) 2009-07-08 2017-09-27 Kymab Limited Animal models and therapeutic molecules
RU2425880C2 (ru) * 2009-07-30 2011-08-10 Учреждение Российской академии наук Институт общей генетики им. Н.И. Вавилова РАН Способ получения трансгенных мышей
US8518392B2 (en) 2009-08-14 2013-08-27 Regeneron Pharmaceuticals, Inc. Promoter-regulated differentiation-dependent self-deleting cassette
TR201903376T4 (tr) 2009-10-29 2019-04-22 Regeneron Pharma Çok fonksiyonlu alleller.
US8754287B2 (en) 2009-12-10 2014-06-17 Regeneron Pharmaceuticals, Inc. Mice that make heavy chain antibodies
EP3336102A3 (en) 2009-12-21 2018-08-01 Regeneron Pharmaceuticals, Inc. Humanized fc gamma r mice
JP2013517774A (ja) 2010-01-22 2013-05-20 ダウ アグロサイエンシィズ エルエルシー 遺伝子改変生物における導入遺伝子の切除
US9796788B2 (en) 2010-02-08 2017-10-24 Regeneron Pharmaceuticals, Inc. Mice expressing a limited immunoglobulin light chain repertoire
US20130185821A1 (en) 2010-02-08 2013-07-18 Regeneron Pharmaceuticals, Inc. Common Light Chain Mouse
SMT201900372T1 (it) 2010-02-08 2019-09-09 Regeneron Pharma Topo con catena leggera comune
US20120021409A1 (en) 2010-02-08 2012-01-26 Regeneron Pharmaceuticals, Inc. Common Light Chain Mouse
US20130045492A1 (en) 2010-02-08 2013-02-21 Regeneron Pharmaceuticals, Inc. Methods For Making Fully Human Bispecific Antibodies Using A Common Light Chain
CA2798988C (en) 2010-05-17 2020-03-10 Sangamo Biosciences, Inc. Tal-effector (tale) dna-binding polypeptides and uses thereof
SI2480676T1 (sl) 2010-06-22 2016-10-28 Regeneron Pharmaceuticals, Inc. Hibridna mišja lahka veriga
CA2805442C (en) 2010-07-21 2020-05-12 Sangamo Biosciences, Inc. Methods and compositions for modification of an hla locus
RU2612903C2 (ru) 2010-08-02 2017-03-13 Ридженерон Фармасьютикалз, Инк. Мыши, у которых вырабатываются связывающие белки, содержащие vl-домены
EA201370161A1 (ru) 2011-01-18 2013-12-30 Амген Инк. НОКАУТНЫЕ ПО Na1.7 МЫШИ И ИХ ПРИМЕНЕНИЕ
DK2813573T1 (da) 2011-02-25 2015-01-12 Regeneron Pharma ADAM6 mus
CN106432506A (zh) 2011-05-24 2017-02-22 泽恩格尼亚股份有限公司 多价和单价多特异性复合物及其用途
HUE068728T2 (hu) 2011-08-05 2025-01-28 Regeneron Pharma Humanizált univerzális könnyûláncú egerek
EP2757875B2 (en) 2011-09-19 2023-03-22 Kymab Limited Manipulation of immunoglobulin gene diversity and multi-antibody therapeutics
PL3216871T3 (pl) 2011-10-17 2022-03-21 Regeneron Pharmaceuticals, Inc. Ograniczony łańcuch ciężki immonoglobuliny pochodzącej od myszy
MX378871B (es) 2011-12-20 2025-03-10 Regeneron Pharma Ratones con cadena ligera humanizada.
PL2809150T3 (pl) 2012-02-01 2020-04-30 Regeneron Pharmaceuticals, Inc. Humanizowane myszy wykazujące ekspresję łańcuchów ciężkich zawierających domeny VL
US20140013456A1 (en) 2012-03-16 2014-01-09 Regeneron Pharmaceuticals, Inc. Histidine Engineered Light Chain Antibodies and Genetically Modified Non-Human Animals for Generating the Same
KR102213535B1 (ko) 2012-03-16 2021-02-08 리제너론 파마슈티칼스 인코포레이티드 pH-민감성 면역글로불린 서열을 발현하는 비-사람 동물
CA2865644A1 (en) 2012-03-16 2013-09-19 Regeneron Pharmaceuticals, Inc. Mice that produce antigen-binding proteins with ph-dependent binding characteristics
EP3348140B1 (en) 2012-03-16 2020-12-30 Regeneron Pharmaceuticals, Inc. Histidine engineered light chain antibodies and genetically modified rodents for generating the same
WO2013141680A1 (en) 2012-03-20 2013-09-26 Vilnius University RNA-DIRECTED DNA CLEAVAGE BY THE Cas9-crRNA COMPLEX
US9637739B2 (en) 2012-03-20 2017-05-02 Vilnius University RNA-directed DNA cleavage by the Cas9-crRNA complex
US10251377B2 (en) * 2012-03-28 2019-04-09 Kymab Limited Transgenic non-human vertebrate for the expression of class-switched, fully human, antibodies
BR112014027813A2 (pt) 2012-05-07 2017-08-08 Dow Agrosciences Llc métodos e composições para integração de transgenes direcionada mediada por nuclease
FI3597749T3 (fi) 2012-05-25 2023-10-09 Univ California Menetelmiä ja koostumuksia rna-ohjattua kohde-dna-modifikaatiota varten ja rna-ohjattua transkription modulaatiota varten
EP2858487B1 (en) * 2012-06-12 2019-10-23 Regeneron Pharmaceuticals, Inc. Humanized non-human animals with restricted immunoglobulin heavy chain loci
EP2877213B1 (en) 2012-07-25 2020-12-02 The Broad Institute, Inc. Inducible dna binding proteins and genome perturbation tools and applications thereof
WO2014033644A2 (en) 2012-08-28 2014-03-06 Novartis Ag Methods of nuclease-based genetic engineering
KR101656236B1 (ko) 2012-10-23 2016-09-12 주식회사 툴젠 표적 DNA에 특이적인 가이드 RNA 및 Cas 단백질을 암호화하는 핵산 또는 Cas 단백질을 포함하는, 표적 DNA를 절단하기 위한 조성물 및 이의 용도
PT3363902T (pt) 2012-12-06 2019-12-19 Sigma Aldrich Co Llc Modificação e regulação de genoma baseadas em crispr
EP2932421A1 (en) 2012-12-12 2015-10-21 The Broad Institute, Inc. Methods, systems, and apparatus for identifying target sequences for cas enzymes or crispr-cas systems for target sequences and conveying results thereof
KR20150105956A (ko) 2012-12-12 2015-09-18 더 브로드 인스티튜트, 인코퍼레이티드 서열 조작 및 치료적 적용을 위한 시스템, 방법 및 조성물의 전달, 유전자 조작 및 최적화
EP2931898B1 (en) 2012-12-12 2016-03-09 The Broad Institute, Inc. Engineering and optimization of systems, methods and compositions for sequence manipulation with functional domains
US8697359B1 (en) 2012-12-12 2014-04-15 The Broad Institute, Inc. CRISPR-Cas systems and methods for altering expression of gene products
EP4282970A3 (en) 2012-12-17 2024-01-17 President and Fellows of Harvard College Rna-guided human genome engineering
JP6475172B2 (ja) * 2013-02-20 2019-02-27 リジェネロン・ファーマシューティカルズ・インコーポレイテッドＲｅｇｅｎｅｒｏｎ Ｐｈａｒｍａｃｅｕｔｉｃａｌｓ， Ｉｎｃ． ラットの遺伝子組換え
RS57582B1 (sr) 2013-02-20 2018-11-30 Regeneron Pharma Nehumane životinje sa modifikovanim sekvencama imunoglobulinskog teškog lanca
JP2016507244A (ja) 2013-02-27 2016-03-10 ヘルムホルツ・ツェントルム・ミュンヒェン・ドイチェス・フォルシュンクスツェントルム・フューア・ゲズントハイト・ウント・ウムベルト（ゲーエムベーハー）Ｈｅｌｍｈｏｌｔｚ Ｚｅｎｔｒｕｍ ＭｕｅｎｃｈｅｎＤｅｕｔｓｃｈｅｓ Ｆｏｒｓｃｈｕｎｇｓｚｅｎｔｒｕｍ ｆｕｅｒ Ｇｅｓｕｎｄｈｅｉｔ ｕｎｄ Ｕｍｗｅｌｔ （ＧｍｂＨ） Ｃａｓ９ヌクレアーゼによる卵母細胞における遺伝子編集
US10612043B2 (en) 2013-03-09 2020-04-07 Agilent Technologies, Inc. Methods of in vivo engineering of large sequences using multiple CRISPR/cas selections of recombineering events
AU2014235794A1 (en) 2013-03-14 2015-10-22 Caribou Biosciences, Inc. Compositions and methods of nucleic acid-targeting nucleic acids
US20140273230A1 (en) 2013-03-15 2014-09-18 Sigma-Aldrich Co., Llc Crispr-based genome modification and regulation
KR102874079B1 (ko) 2013-03-15 2025-10-22 더 제너럴 하스피탈 코포레이션 Rna-안내 게놈 편집을 위해 특이성을 증가시키기 위한 절단된 안내 rna(tru-grnas)의 이용
US9788534B2 (en) 2013-03-18 2017-10-17 Kymab Limited Animal models and therapeutic molecules
EP2981617B1 (en) 2013-04-04 2023-07-05 President and Fellows of Harvard College Therapeutic uses of genome editing with crispr/cas systems
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
EP3456831B1 (en) 2013-04-16 2021-07-14 Regeneron Pharmaceuticals, Inc. Targeted modification of rat genome
EP2994531B1 (en) 2013-05-10 2018-03-28 Sangamo Therapeutics, Inc. Delivery methods and compositions for nuclease-mediated genome engineering
AU2014266833B2 (en) * 2013-05-13 2020-07-02 Cellectis Methods for engineering highly active T cell for immunotherapy
EP3004349B1 (en) 2013-05-29 2018-03-28 Cellectis S.A. A method for producing precise dna cleavage using cas9 nickase activity
CN105492611A (zh) 2013-06-17 2016-04-13 布罗德研究所有限公司 用于序列操纵的优化的crispr-cas双切口酶系统、方法以及组合物
CN107995927B (zh) 2013-06-17 2021-07-30 布罗德研究所有限公司 用于肝靶向和治疗的crispr-cas系统、载体和组合物的递送与用途
CA2915795C (en) 2013-06-17 2021-07-13 The Broad Institute, Inc. Delivery, use and therapeutic applications of the crispr-cas systems and compositions for targeting disorders and diseases using viral components
EP3011035B1 (en) 2013-06-17 2020-05-13 The Broad Institute, Inc. Assay for quantitative evaluation of target site cleavage by one or more crispr-cas guide sequences
EP3674411A1 (en) 2013-06-17 2020-07-01 The Broad Institute, Inc. Delivery, engineering and optimization of tandem guide systems, methods and compositions for sequence manipulation
JP6738729B2 (ja) 2013-06-17 2020-08-12 ザ・ブロード・インスティテュート・インコーポレイテッド 分裂終了細胞の疾患および障害をターゲティングおよびモデリングするための系、方法および組成物の送達、エンジニアリングおよび最適化
US10011850B2 (en) 2013-06-21 2018-07-03 The General Hospital Corporation Using RNA-guided FokI Nucleases (RFNs) to increase specificity for RNA-Guided Genome Editing
EP3019595A4 (en) 2013-07-09 2016-11-30 THERAPEUTIC USES OF A GENERIC CHANGE WITH CRISPR / CAS SYSTEMS
WO2015010114A1 (en) 2013-07-19 2015-01-22 Larix Bioscience, Llc Methods and compositions for producing double allele knock outs
US11060083B2 (en) 2013-07-19 2021-07-13 Larix Bioscience Llc Methods and compositions for producing double allele knock outs
AU2014312123A1 (en) 2013-08-29 2016-03-17 Temple University Of The Commonwealth System Of Higher Education Methods and compositions for RNA-guided treatment of HIV infection
EP3988649B1 (en) 2013-09-18 2024-11-27 Kymab Limited Methods, cells and organisms
US20160237455A1 (en) 2013-09-27 2016-08-18 Editas Medicine, Inc. Crispr-related methods and compositions
US10822606B2 (en) 2013-09-27 2020-11-03 The Regents Of The University Of California Optimized small guide RNAs and methods of use
US9957526B2 (en) 2013-10-17 2018-05-01 Sangamo Therapeutics, Inc. Delivery methods and compositions for nuclease-mediated genome engineering
US20160264995A1 (en) 2013-11-06 2016-09-15 Hiroshima University Vector for Nucleic Acid Insertion
KR20230054509A (ko) 2013-11-07 2023-04-24 에디타스 메디신, 인코포레이티드 지배적인 gRNA를 이용하는 CRISPR-관련 방법 및 조성물
US10787684B2 (en) 2013-11-19 2020-09-29 President And Fellows Of Harvard College Large gene excision and insertion
CA2928635C (en) 2013-11-28 2022-06-21 Horizon Genomics Gmbh Somatic haploid human cell line
MX388127B (es) 2013-12-11 2025-03-19 Regeneron Pharma Metodos y composiciones para la modificacion dirigida de un genoma.
EP3540051B1 (en) 2013-12-12 2022-08-17 The Broad Institute, Inc. Delivery, use and therapeutic applications of the crispr-cas systems and compositions for hsv and viral diseases and disorders.
EP3080259B1 (en) 2013-12-12 2023-02-01 The Broad Institute, Inc. Engineering of systems, methods and optimized guide compositions with new architectures for sequence manipulation
BR112016013201B1 (pt) 2013-12-12 2023-01-31 The Broad Institute, Inc. Uso de uma composição compreendendo um sistema crispr-cas no tratamento de uma doença genética ocular
SG10201804973TA (en) 2013-12-12 2018-07-30 Broad Inst Inc Compositions and Methods of Use of Crispr-Cas Systems in Nucleotide Repeat Disorders
US10233456B2 (en) 2014-01-30 2019-03-19 The Board Of Trustees Of The University Of Arkansas Method, vectors, cells, seeds and kits for stacking genes into a single genomic site
US20150291969A1 (en) 2014-01-30 2015-10-15 Chromatin, Inc. Compositions for reduced lignin content in sorghum and improving cell wall digestibility, and methods of making the same
JP6606088B2 (ja) 2014-02-24 2019-11-13 サンガモ セラピューティクス， インコーポレイテッド ヌクレアーゼ媒介性標的化組み込みのための方法および組成物
WO2015134812A1 (en) 2014-03-05 2015-09-11 Editas Medicine, Inc. Crispr/cas-related methods and compositions for treating usher syndrome and retinitis pigmentosa
EP3553176A1 (en) 2014-03-10 2019-10-16 Editas Medicine, Inc. Crispr/cas-related methods and compositions for treating leber's congenital amaurosis 10 (lca10)
ES2821149T3 (es) 2014-03-12 2021-04-23 Prec Biosciences Inc Eliminación del exón del gen de la distrofina mediante nucleasas modificadas genéticamente
CA2942407C (en) 2014-03-14 2023-09-26 Cibus Us Llc Methods and compositions for increasing efficiency of targeted gene modification using oligonucleotide-mediated gene repair
RU2016141307A (ru) * 2014-03-21 2018-04-24 Регенерон Фармасьютикалз, Инк. Отличные от человека животные, которые вырабатывают однодоменные связывающие белки
US10349639B2 (en) 2014-03-26 2019-07-16 University Of Maryland, College Park Targeted genome editing in zygotes of domestic large animals
GB201406968D0 (en) 2014-04-17 2014-06-04 Green Biologics Ltd Deletion mutants
WO2015173436A1 (en) 2014-05-16 2015-11-19 Vrije Universiteit Brussel Genetic correction of myotonic dystrophy type 1
US20160060655A1 (en) 2014-05-30 2016-03-03 The Board Of Trustees Of The Leland Stanford Junior University Compositions and methods to treat latent viral infections
BR112016028564A2 (pt) * 2014-06-06 2018-01-30 Regeneron Pharma método para modificar um locus-alvo em uma célula.
SG10202002486QA (en) 2014-06-16 2020-04-29 Univ Johns Hopkins Compositions and methods for the expression of crispr guide rnas using the h1 promoter
US20150376586A1 (en) 2014-06-25 2015-12-31 Caribou Biosciences, Inc. RNA Modification to Engineer Cas9 Activity
SG10201911411YA (en) 2014-06-26 2020-02-27 Regeneron Pharma Methods and compositions for targeted genetic modifications and methods of use
MX2017000646A (es) 2014-07-15 2017-04-27 Juno Therapeutics Inc Celulas geneticamente modificadas para terapia celular adoptiva.
EP3193944B1 (en) 2014-07-17 2021-04-07 University of Pittsburgh - Of the Commonwealth System of Higher Education Methods of treating cells containing fusion genes
US9970030B2 (en) 2014-08-27 2018-05-15 Caribou Biosciences, Inc. Methods for increasing CAS9-mediated engineering efficiency
US10570418B2 (en) 2014-09-02 2020-02-25 The Regents Of The University Of California Methods and compositions for RNA-directed target DNA modification
WO2016049024A2 (en) 2014-09-24 2016-03-31 The Broad Institute Inc. Delivery, use and therapeutic applications of the crispr-cas systems and compositions for modeling competition of multiple cancer mutations in vivo
WO2016049163A2 (en) 2014-09-24 2016-03-31 The Broad Institute Inc. Use and production of chd8+/- transgenic animals with behavioral phenotypes characteristic of autism spectrum disorder
WO2016049258A2 (en) 2014-09-25 2016-03-31 The Broad Institute Inc. Functional screening with optimized functional crispr-cas systems
WO2016061073A1 (en) 2014-10-14 2016-04-21 Memorial Sloan-Kettering Cancer Center Composition and method for in vivo engineering of chromosomal rearrangements
EP3207139B1 (en) 2014-10-17 2025-05-07 The Penn State Research Foundation Methods and compositions for multiplex rna guided genome editing and other rna technologies
WO2016073955A2 (en) 2014-11-06 2016-05-12 President And Fellows Of Harvard College Cells lacking b2m surface expression and methods for allogeneic administration of such cells
AU2015342749B2 (en) 2014-11-07 2022-01-27 Editas Medicine, Inc. Methods for improving CRISPR/Cas-mediated genome-editing
CA3176380A1 (en) 2014-11-21 2016-05-26 Regeneron Pharmaceuticals, Inc. Methods and compositions for targeted genetic modification using paired guide rnas
US10883111B2 (en) 2014-11-27 2021-01-05 Danziger Innovations Ltd. Nucleic acid constructs for genome editing
WO2016089866A1 (en) 2014-12-01 2016-06-09 President And Fellows Of Harvard College Rna-guided systems for in vivo gene editing
WO2016094874A1 (en) 2014-12-12 2016-06-16 The Broad Institute Inc. Escorted and functionalized guides for crispr-cas systems
WO2016094872A1 (en) 2014-12-12 2016-06-16 The Broad Institute Inc. Dead guides for crispr transcription factors
WO2016106236A1 (en) 2014-12-23 2016-06-30 The Broad Institute Inc. Rna-targeting system
EP3239298A4 (en) 2014-12-26 2018-06-13 Riken Gene knockout method
US20180155708A1 (en) 2015-01-08 2018-06-07 President And Fellows Of Harvard College Split Cas9 Proteins
WO2016112351A1 (en) 2015-01-09 2016-07-14 Bio-Rad Laboratories, Inc. Detection of genome editing
WO2016130697A1 (en) 2015-02-11 2016-08-18 Memorial Sloan Kettering Cancer Center Methods and kits for generating vectors that co-express multiple target molecules
US12129471B2 (en) 2015-02-23 2024-10-29 Vertex Pharmaceuticals Incorporated Materials and methods for treatment of human genetic diseases including hemoglobinopathies
AU2016225178B2 (en) 2015-02-23 2022-05-05 Crispr Therapeutics Ag Materials and methods for treatment of hemoglobinopathies
GB201504223D0 (en) 2015-03-12 2015-04-29 Genome Res Ltd Biallelic genetic modification
US11111314B2 (en) 2015-03-19 2021-09-07 Regeneron Pharmaceuticals, Inc. Non-human animals that select for light chain variable regions that bind antigen
EP3274453B1 (en) 2015-03-26 2021-01-27 Editas Medicine, Inc. Crispr/cas-mediated gene conversion
BR112017020750A2 (pt) 2015-03-27 2018-06-26 Harvard College células t modificadas e métodos de produção e utilização das mesmas
EP3748004A1 (en) 2015-04-01 2020-12-09 Editas Medicine, Inc. Crispr/cas-related methods and compositions for treating duchenne muscular dystrophy and becker muscular dystrophy
US10155938B2 (en) 2015-04-14 2018-12-18 City Of Hope Coexpression of CAS9 and TREX2 for targeted mutagenesis
US20180346879A1 (en) 2015-04-22 2018-12-06 Genea Ip Holdings Pty Limited Generation of muscle-lineage clels from stem cells
EP3289081B1 (en) 2015-04-27 2019-03-27 Genethon Compositions and methods for the treatment of nucleotide repeat expansion disorders
EP4008780A1 (en) 2015-04-30 2022-06-08 The Trustees of Columbia University in the City of New York Gene therapy for autosomal dominant diseases
CN107849547B (zh) 2015-05-16 2022-04-19 建新公司 深内含子突变的基因编辑
US9790490B2 (en) 2015-06-18 2017-10-17 The Broad Institute Inc. CRISPR enzymes and systems
IL297017A (en) 2015-10-08 2022-12-01 Harvard College Multiplexed genome editing
KR20180069832A (ko) 2015-10-20 2018-06-25 파이어니어 하이 부렛드 인터내쇼날 인코포레이팃드 유도 cas 시스템을 통한 비기능성 유전자 산물에 대한 기능 회복 및 이용 방법
CA3004497A1 (en) 2015-11-06 2017-05-11 The Jackson Laboratory Large genomic dna knock-in and uses thereof
EP3416479A1 (en) 2016-02-16 2018-12-26 Regeneron Pharmaceuticals, Inc. Non-human animals having a mutant kynureninase gene
EP3433363A1 (en) 2016-03-25 2019-01-30 Editas Medicine, Inc. Genome editing systems comprising repair-modulating enzyme molecules and methods of their use
WO2017173004A1 (en) 2016-03-30 2017-10-05 Mikuni Takayasu A method for in vivo precise genome editing
US11773410B2 (en) 2016-04-13 2023-10-03 University Of Massachusetts Repairing compound heterozygous recessive mutations by allele exchange
WO2017201476A1 (en) 2016-05-20 2017-11-23 Regeneron Pharmaceuticals, Inc. Methods for breaking immunological tolerance using multiple guide rnas
US20200029538A1 (en) 2016-11-28 2020-01-30 Osaka University Genome editing method

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