JP2018534114A5 - - Google Patents

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JP2018534114A5
JP2018534114A5 JP2018545108A JP2018545108A JP2018534114A5 JP 2018534114 A5 JP2018534114 A5 JP 2018534114A5 JP 2018545108 A JP2018545108 A JP 2018545108A JP 2018545108 A JP2018545108 A JP 2018545108A JP 2018534114 A5 JP2018534114 A5 JP 2018534114A5
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nucleic acid
pharmaceutical composition
composition according
stem cells
target nucleic
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JP2018545108A
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JP2018534114A (ja
JP6929865B2 (ja
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Priority claimed from PCT/US2016/061893 external-priority patent/WO2017083852A1/en
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Priority to JP2021131116A priority Critical patent/JP7239649B2/ja
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JP2018545108A 2015-11-13 2016-11-14 角膜ジストロフィーの治療方法 Expired - Fee Related JP6929865B2 (ja)

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JP2021131116A JP7239649B2 (ja) 2015-11-13 2021-08-11 角膜ジストロフィーの治療方法

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201562255310P 2015-11-13 2015-11-13
US62/255,310 2015-11-13
PCT/US2016/061893 WO2017083852A1 (en) 2015-11-13 2016-11-14 Methods for the treatment of corneal dystrophies

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JP2018534114A JP2018534114A (ja) 2018-11-22
JP2018534114A5 true JP2018534114A5 (enExample) 2019-12-19
JP6929865B2 JP6929865B2 (ja) 2021-09-01

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JP2021131116A Active JP7239649B2 (ja) 2015-11-13 2021-08-11 角膜ジストロフィーの治療方法

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US (1) US11987809B2 (enExample)
EP (2) EP4036228A1 (enExample)
JP (2) JP6929865B2 (enExample)
KR (1) KR20180120670A (enExample)
CN (1) CN108699542A (enExample)
ES (1) ES2905558T3 (enExample)
WO (1) WO2017083852A1 (enExample)

Families Citing this family (26)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US9340799B2 (en) 2013-09-06 2016-05-17 President And Fellows Of Harvard College MRNA-sensing switchable gRNAs
EP3500677A4 (en) * 2016-08-20 2020-04-01 Avellino Lab USA, Inc. Single guide rna, crispr/cas9 systems, and methods of use thereof
US11898179B2 (en) 2017-03-09 2024-02-13 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
WO2018165629A1 (en) 2017-03-10 2018-09-13 President And Fellows Of Harvard College Cytosine to guanine base editor
AU2018280116B2 (en) * 2017-06-05 2025-03-27 Research Institute At Nationwide Children's Hospital Enhanced modified viral capsid proteins
CN111065736A (zh) * 2017-06-07 2020-04-24 国立大学法人东京大学 针对颗粒状角膜变性症的基因治疗药物
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
WO2019060469A2 (en) * 2017-09-19 2019-03-28 Massachusetts Institute Of Technology CAS9 STREPTOCOCCUS CANIS AS A GENOMIC ENGINEERING PLATFORM WITH NEW PAM SPECIFICITY
AU2018339164B2 (en) * 2017-09-28 2022-06-16 Toolgen Incorporated Artificial genome manipulation for gene expression regulation
US11572574B2 (en) 2017-09-28 2023-02-07 Toolgen Incorporated Artificial genome manipulation for gene expression regulation
CN111801417B (zh) * 2017-12-14 2024-10-29 克里斯珀医疗股份公司 新的rna-可编程的内切核酸酶系统及其在基因组编辑和其他应用中的用途
WO2019118949A1 (en) 2017-12-15 2019-06-20 The Broad Institute, Inc. Systems and methods for predicting repair outcomes in genetic engineering
EP3784249A4 (en) 2018-04-25 2022-03-02 Children's Medical Center Corporation ABCB5 LIGANDS AND SUBSTRATES
EP3820495A4 (en) 2018-07-09 2022-07-20 The Broad Institute Inc. RNA-PROGRAMMABLE EPIGENETIC RNA MODIFIERS AND THEIR USES
WO2020092453A1 (en) 2018-10-29 2020-05-07 The Broad Institute, Inc. Nucleobase editors comprising geocas9 and uses thereof
WO2020125762A1 (en) * 2018-12-20 2020-06-25 Peking University Compositions and methods for highly efficient genetic screening using barcoded guide rna constructs
US12351837B2 (en) 2019-01-23 2025-07-08 The Broad Institute, Inc. Supernegatively charged proteins and uses thereof
EP3942043A2 (en) 2019-03-19 2022-01-26 The Broad Institute, Inc. Methods and compositions for editing nucleotide sequences
BR112021019349A2 (pt) * 2019-03-28 2021-12-07 Childrens Medical Center Células-tronco preparadas altamente funcionais
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
US20230036273A1 (en) * 2019-11-27 2023-02-02 The General Hospital Corporation System and method for activating gene expression
RU2730975C1 (ru) * 2020-02-18 2020-08-26 Ирина Викторовна Булыгина Способ лечения эндотелиально-эпителиальной дистрофии роговицы
WO2023004189A1 (en) * 2021-07-23 2023-01-26 Avellino Lab Usa, Inc. Method for detecting alleles associated with keratoconus
CN116286823A (zh) * 2023-04-04 2023-06-23 尧唐(上海)生物科技有限公司 靶向TGFBI基因的sgRNA、载体及其应用

Family Cites Families (80)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5049386A (en) 1985-01-07 1991-09-17 Syntex (U.S.A.) Inc. N-ω,(ω-1)-dialkyloxy)- and N-(ω,(ω-1)-dialkenyloxy)Alk-1-YL-N,N,N-tetrasubstituted ammonium lipids and uses therefor
US4897355A (en) 1985-01-07 1990-01-30 Syntex (U.S.A.) Inc. N[ω,(ω-1)-dialkyloxy]- and N-[ω,(ω-1)-dialkenyloxy]-alk-1-yl-N,N,N-tetrasubstituted ammonium lipids and uses therefor
US4946787A (en) 1985-01-07 1990-08-07 Syntex (U.S.A.) Inc. N-(ω,(ω-1)-dialkyloxy)- and N-(ω,(ω-1)-dialkenyloxy)-alk-1-yl-N,N,N-tetrasubstituted ammonium lipids and uses therefor
US5210015A (en) 1990-08-06 1993-05-11 Hoffman-La Roche Inc. Homogeneous assay system using the nuclease activity of a nucleic acid polymerase
US5925517A (en) 1993-11-12 1999-07-20 The Public Health Research Institute Of The City Of New York, Inc. Detectably labeled dual conformation oligonucleotide probes, assays and kits
ATE428801T1 (de) 1996-06-04 2009-05-15 Univ Utah Res Found Überwachung der hybridisierung während pcr
US6117635A (en) 1996-07-16 2000-09-12 Intergen Company Nucleic acid amplification oligonucleotides with molecular energy transfer labels and methods based thereon
US6018713A (en) 1997-04-09 2000-01-25 Coli; Robert D. Integrated system and method for ordering and cumulative results reporting of medical tests
JP3380744B2 (ja) 1998-05-19 2003-02-24 株式会社日立製作所 センサおよびこれを利用した測定装置
GB9812768D0 (en) 1998-06-13 1998-08-12 Zeneca Ltd Methods
US6171112B1 (en) 1998-09-18 2001-01-09 Wyngate, Inc. Methods and apparatus for authenticating informed consent
AU4135600A (en) 1999-03-29 2000-10-16 Genset Prostate cancer associated human fibronectin gene and biallelic markers
US6270970B1 (en) 1999-05-14 2001-08-07 Promega Corporation Mixed-bed solid phase and its use in the isolation of nucleic acids
DE60131970T2 (de) 2000-04-13 2008-12-11 Georgetown University Genetische diagnose zur feststellung von qt-verlängerungen als unerwünschte reaktion auf arzneimittel
US8438042B2 (en) 2002-04-25 2013-05-07 National Biomedical Research Foundation Instruments and methods for obtaining informed consent to genetic tests
WO2003014703A2 (en) 2001-08-09 2003-02-20 Curagen Corporation Nucleic acids, polypeptides, single nucleotide polymorphisms and methods of use thereof
AU2002352069A1 (en) 2001-11-28 2003-06-10 Graffinity Pharmaceuticals Ag Surface plasmon resonance (spr) sensor surface support
US6943417B2 (en) 2003-05-01 2005-09-13 Clemson University DNA-based memory device and method of reading and writing same
US20050019757A1 (en) 2003-06-12 2005-01-27 Stolarchuk Danylo J. Contaminant detection apparatus
WO2005015198A1 (ja) 2003-08-06 2005-02-17 Nippon Telegraph And Telephone Corporation 多孔質材料を用いた分子の検出方法ならびに該多孔質材料及び該多孔質材料の製造方法
WO2005040756A2 (en) 2003-10-22 2005-05-06 The Regents Of The University Of California Methods for preparing and functionalizing nanoparticles
US20080113344A1 (en) 2003-10-28 2008-05-15 Ralph Wirtz Methods and Compositions for the Response Prediction of Malignant Neoplasia to Treatment
EP1541528A1 (en) 2003-12-08 2005-06-15 Institut Jozef Stefan Quasi-one-dimensional polymers based on the metal-chalcogen-halogen system
US20060057604A1 (en) 2004-03-15 2006-03-16 Thinkfar Nanotechnology Corporation Method for electrically detecting oligo-nucleotides with nano-particles
MXPA06013440A (es) 2004-05-19 2007-06-12 Vp Holding Llc Sensor optico con estructura estratificada de plasmon para deteccion mejorada de grupos quimicos por dispersion raman intensificada por superficie.
US7713849B2 (en) 2004-08-20 2010-05-11 Illuminex Corporation Metallic nanowire arrays and methods for making and using same
US7332329B2 (en) 2004-09-24 2008-02-19 Wisconsin Alumni Research Foundation Versatile substrate for SPR detection
US20080267946A1 (en) 2005-03-08 2008-10-30 Medigenes Co., Ltd Pharmaceutical Composition for Treating Avellino Cornea Dystrophy Comprising an Antibody Against Tgf-Beta
JP2006250668A (ja) 2005-03-10 2006-09-21 Tatsuro Endo 非標識バイオチップ
EP1715326A1 (en) 2005-04-22 2006-10-25 Universität Heidelberg Sensor chip with connected non-metallic particles comprising a metallic coating
WO2007002567A2 (en) 2005-06-23 2007-01-04 Nanosphere, Inc. Selective isolation and concentration of nucleic acids from complex samples
KR20070076532A (ko) 2006-01-18 2007-07-24 메디제네스(주) 각막이영양증 진단용 dna 칩
CN101374850A (zh) 2006-01-18 2009-02-25 株式会社美迪基尼斯 用于角膜营养不良诊断的dna芯片
US7790383B2 (en) 2006-03-21 2010-09-07 Washington State University Research Foundation Genetic polymorphisms in the corticotropin-releasing hormone (CRH) gene as markers for improving beef marbling score and/or subcutaneous fat depth
EP1932922A1 (de) 2006-12-13 2008-06-18 Desitin Arzneimittel GmbH Schnelltest zum Nachweis von DNA-Sequenzen
US20080176320A1 (en) 2007-01-16 2008-07-24 Applera Corporation Selective Lysis of Sperm Cells
US7898658B2 (en) 2007-01-23 2011-03-01 The Regents Of The University Of California Platform for chemical and biological sensing by surface-enhanced Raman spectroscopy
KR100891096B1 (ko) 2007-02-13 2009-03-31 삼성전자주식회사 올리고머 프로브 어레이 및 이의 제조 방법
JP5222599B2 (ja) 2007-07-20 2013-06-26 株式会社日立ハイテクノロジーズ 核酸分析デバイス及びそれを用いた核酸分析装置
CN101144812B (zh) 2007-10-17 2012-02-22 中国科学院光电技术研究所 一种局域表面等离子体生化传感器的制作方法
US20120231537A1 (en) 2008-04-30 2012-09-13 Gradalis, Inc. Highly Pure Plasmid DNA Preparations
KR101251538B1 (ko) 2009-04-17 2013-04-08 (주)아벨리노 아벨리노 각막이상증 진단용 프라이머
US8865402B2 (en) 2009-08-26 2014-10-21 Clemson University Research Foundation Nanostructured substrates for surface enhanced raman spectroscopy (SERS) and detection of biological and chemical analytes by electrical double layer (EDL) capacitance
KR101125212B1 (ko) 2010-10-01 2012-03-21 (주)아벨리노 아벨리노 각막이상증 진단용 시스템
KR20130027281A (ko) 2011-09-07 2013-03-15 (주)에스엘에스 원추각막 검사 방법 및 키트
US9895451B2 (en) 2011-12-02 2018-02-20 Yale University Formulations for targeted release of agents to low pH tissue environments or cellular compartments and methods of use thereof
DK2890780T3 (da) 2012-08-29 2020-09-21 Sangamo Therapeutics Inc Fremgangsmåder og sammensætninger til behandling af en genetisk tilstand
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
CN114634950A (zh) 2012-12-12 2022-06-17 布罗德研究所有限公司 用于序列操纵的crispr-cas组分系统、方法以及组合物
ES2576126T3 (es) 2012-12-12 2016-07-05 The Broad Institute, Inc. Modificación por tecnología genética y optimización de sistemas, métodos y composiciones enzimáticas mejorados para la manipulación de secuencias
US8697359B1 (en) 2012-12-12 2014-04-15 The Broad Institute, Inc. CRISPR-Cas systems and methods for altering expression of gene products
IL239317B (en) 2012-12-12 2022-07-01 Broad Inst Inc Providing, engineering and optimizing systems, methods and compositions for sequence manipulation and therapeutic applications
US20140310830A1 (en) 2012-12-12 2014-10-16 Feng Zhang CRISPR-Cas Nickase Systems, Methods And Compositions For Sequence Manipulation in Eukaryotes
EP2896697B1 (en) 2012-12-12 2015-09-02 The Broad Institute, Inc. Engineering of systems, methods and optimized guide compositions for sequence manipulation
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
JP2016504026A (ja) 2012-12-12 2016-02-12 ザ・ブロード・インスティテュート・インコーポレイテッド 配列操作のための系、方法および最適化ガイド組成物のエンジニアリング
WO2014130518A1 (en) * 2013-02-19 2014-08-28 Children's Medical Center Corporation Abcb5(+) stem cells for treating ocular disease
CN105308175B (zh) * 2013-03-11 2018-09-21 日本化学研究株式会社 人角膜上皮片的制造法
SG11201507378UA (en) 2013-03-14 2015-10-29 Caribou Biosciences Inc Compositions and methods of nucleic acid-targeting nucleic acids
EP3730615A3 (en) 2013-05-15 2020-12-09 Sangamo Therapeutics, Inc. Methods and compositions for treatment of a genetic condition
KR20260047646A (ko) 2013-07-09 2026-04-08 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 멀티플렉스 rna-가이드된 게놈 조작
US20150044772A1 (en) * 2013-08-09 2015-02-12 Sage Labs, Inc. Crispr/cas system-based novel fusion protein and its applications in genome editing
AU2014348279B2 (en) * 2013-11-15 2021-02-18 Avellino Lab Usa, Inc. Methods for multiplex detection of alleles associated with ophthalmic conditions
CN111206032B (zh) * 2013-12-12 2024-07-19 布罗德研究所有限公司 用于基因组编辑的crispr-cas系统和组合物的递送、用途和治疗应用
US20150225801A1 (en) * 2014-02-11 2015-08-13 California Institute Of Technology Recording and mapping lineage information and molecular events in individual cells
KR20170020527A (ko) * 2014-06-27 2017-02-22 더 리젠츠 오브 더 유니버시티 오브 캘리포니아 배양된 포유동물 윤부 줄기세포, 이를 생성하는 방법, 및 이의 용도
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
GB2531454A (en) 2016-01-10 2016-04-20 Snipr Technologies Ltd Recombinogenic nucleic acid strands in situ
WO2017004616A1 (en) * 2015-07-02 2017-01-05 The Johns Hopkins University Crispr/cas9-based treatments
CA3004713A1 (en) 2015-10-09 2017-04-13 The Children's Hospital Of Philadelphia Compositions and methods for treating huntington's disease and related disorders
KR20250103795A (ko) 2016-08-03 2025-07-07 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 아데노신 핵염기 편집제 및 그의 용도
US20220056440A1 (en) 2016-08-20 2022-02-24 Tara Moore Crispr gene editing for autosomal dominant diseases
EP3500677A4 (en) 2016-08-20 2020-04-01 Avellino Lab USA, Inc. Single guide rna, crispr/cas9 systems, and methods of use thereof
WO2019165322A1 (en) 2018-02-22 2019-08-29 Moore Tara Crispr/cas9 systems, and methods of use thereof
US20210222171A1 (en) 2016-08-20 2021-07-22 Avellino Lab Usa, Inc. Crispr/cas9 systems, and methods of use thereof
WO2020046861A1 (en) 2018-08-27 2020-03-05 Avellino Lab Usa, Inc. Crispr/cas9 systems, and methods of use thereof
CN106282383B (zh) 2016-10-08 2019-05-31 山东省眼科研究所 Krt12基因在检测圆锥角膜中的应用
CN110382695A (zh) 2016-11-28 2019-10-25 得克萨斯州大学系统董事会 通过crispr/cpf1介导的基因编辑来预防肌营养不良
CA3061620A1 (en) 2017-04-28 2018-11-01 Avellino Lab Usa, Inc. Methods for detecting alleles associated with keratoconus
EP3676378A4 (en) 2017-08-31 2021-06-09 The New York Genome Center PROCEDURES AND COMPOSITIONS USING CRIMP-CPF1 AND PAIRED GUIDE-CRISPR-RNA FOR PROGRAMMABLE GENOMIC DELETIONS

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