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
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Priority Applications (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| 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 |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2021131116A Division JP7239649B2 (ja) | 2015-11-13 | 2021-08-11 | 角膜ジストロフィーの治療方法 |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2018534114A JP2018534114A (ja) | 2018-11-22 |
| JP2018534114A5 true JP2018534114A5 (enExample) | 2019-12-19 |
| JP6929865B2 JP6929865B2 (ja) | 2021-09-01 |
Family
ID=58696178
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2018545108A Expired - Fee Related JP6929865B2 (ja) | 2015-11-13 | 2016-11-14 | 角膜ジストロフィーの治療方法 |
| JP2021131116A Active JP7239649B2 (ja) | 2015-11-13 | 2021-08-11 | 角膜ジストロフィーの治療方法 |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2021131116A Active JP7239649B2 (ja) | 2015-11-13 | 2021-08-11 | 角膜ジストロフィーの治療方法 |
Country Status (7)
| Country | Link |
|---|---|
| US (1) | US11987809B2 (enExample) |
| EP (2) | EP3374502B1 (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 (24)
| 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 |
| KR102594051B1 (ko) * | 2016-08-20 | 2023-10-26 | 아벨리노 랩 유에스에이, 인크. | 단일 가이드 RNA, CRISPR/Cas9 시스템, 및 이의 사용방법 |
| EP3592381A1 (en) | 2017-03-09 | 2020-01-15 | President and Fellows of Harvard College | Cancer vaccine |
| CN110914310A (zh) | 2017-03-10 | 2020-03-24 | 哈佛大学的校长及成员们 | 胞嘧啶至鸟嘌呤碱基编辑器 |
| CA3065946A1 (en) * | 2017-06-05 | 2018-12-13 | 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 |
| 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) |
| US20190218532A1 (en) * | 2017-09-19 | 2019-07-18 | Massachusetts Institute Of Technology | Streptococcus Canis Cas9 as a Genome Engineering Platform with Novel PAM Specificity |
| SG11202002130WA (en) * | 2017-09-28 | 2020-04-29 | Toolgen Inc | Artificial genome manipulation for gene expression regulation |
| US11572574B2 (en) | 2017-09-28 | 2023-02-07 | Toolgen Incorporated | Artificial genome manipulation for gene expression regulation |
| EP3724332A1 (en) * | 2017-12-14 | 2020-10-21 | CRISPR Therapeutics AG | Novel rna-programmable endonuclease systems and their use in genome editing and other applications |
| 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 |
| 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 |
| DE112020001342T5 (de) * | 2019-03-19 | 2022-01-13 | President and Fellows of Harvard College | Verfahren und Zusammensetzungen zum Editing von Nukleotidsequenzen |
| EP3946389A4 (en) * | 2019-03-28 | 2023-05-10 | Children's Medical Center Corporation | HIGH FUNCTIONAL ENGINEERED ABCB5+ MESENCHYMAL STEM CELLS |
| WO2020214842A1 (en) | 2019-04-17 | 2020-10-22 | The Broad Institute, Inc. | Adenine base editors with reduced off-target effects |
| WO2021072328A1 (en) | 2019-10-10 | 2021-04-15 | The Broad Institute, Inc. | Methods and compositions for prime editing rna |
| JP2023503618A (ja) * | 2019-11-27 | 2023-01-31 | ザ ジェネラル ホスピタル コーポレイション | 遺伝子発現を活性化するためのシステムおよび方法 |
| 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)
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| 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 |
| 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 |
| 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 |
| 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 |
| WO1997046714A1 (en) | 1996-06-04 | 1997-12-11 | University Of Utah Research Foundation | Monitoring hybridization during 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 |
| ATE381626T1 (de) | 2000-04-13 | 2008-01-15 | Univ Georgetown | 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 |
| CA2451919A1 (en) | 2001-08-09 | 2003-02-20 | Curagen Corporation | Nucleic acids, polypeptides, single nucleotide polymorphisms and methods of use thereof |
| WO2003046526A1 (de) | 2001-11-28 | 2003-06-05 | Graffinity Pharmaceuticals Ag | Spr-sensorflächenträger |
| 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 |
| CA2544129A1 (en) | 2003-10-22 | 2005-05-06 | The Regents Of The University Of California | Methods for preparing and functionalizing nanoparticles |
| EP1682675A2 (en) | 2003-10-28 | 2006-07-26 | Bayer HealthCare AG | 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 |
| NZ551786A (en) | 2004-05-19 | 2011-03-31 | Vp Holding Llc | Optical sensor with layered plasmon structure for enhanced detection of chemical groups by surface enhanced raman scattering |
| 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 |
| US20070154903A1 (en) | 2005-06-23 | 2007-07-05 | 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 |
| WO2007109702A2 (en) | 2006-03-21 | 2007-09-27 | 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 |
| EP2115123A4 (en) | 2007-01-16 | 2010-07-07 | Applied Biosystems Llc | SELECTIVE LYSE 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 |
| RS60838B1 (sr) | 2012-08-29 | 2020-10-30 | Sangamo Therapeutics Inc | Postupci i kompozicije za tretman genskog stanja |
| US20140310830A1 (en) | 2012-12-12 | 2014-10-16 | Feng Zhang | CRISPR-Cas Nickase Systems, Methods And Compositions For Sequence Manipulation in Eukaryotes |
| EP4234696A3 (en) | 2012-12-12 | 2023-09-06 | The Broad Institute Inc. | Crispr-cas component systems, methods and compositions for sequence manipulation |
| IL307735A (en) | 2012-12-12 | 2023-12-01 | Broad Inst Inc | Systems engineering, methods and optimal guiding components for sequence manipulation |
| DK2921557T3 (en) | 2012-12-12 | 2016-11-07 | Broad Inst Inc | Design of systems, methods and optimized sequence manipulation guide compositions |
| 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 |
| CN105209621B (zh) | 2012-12-12 | 2021-05-25 | 布罗德研究所有限公司 | 对用于序列操纵的改进的系统、方法和酶组合物进行的工程化和优化 |
| PL2931898T3 (pl) | 2012-12-12 | 2016-09-30 | Le Cong | Projektowanie i optymalizacja systemów, sposoby i kompozycje do manipulacji sekwencją z domenami funkcjonalnymi |
| AU2013359199C1 (en) | 2012-12-12 | 2021-06-17 | Massachusetts Institute Of Technology | Delivery, engineering and optimization of systems, methods and compositions for sequence manipulation and therapeutic applications |
| US8697359B1 (en) | 2012-12-12 | 2014-04-15 | The Broad Institute, Inc. | CRISPR-Cas systems and methods for altering expression of gene products |
| EP2964238B1 (en) * | 2013-02-19 | 2021-01-13 | Children's Medical Center Corporation | Abcb5(+) stem cells for treating ocular disease |
| EP2975117B1 (en) | 2013-03-11 | 2018-01-10 | JCR Pharmaceuticals Co., Ltd. | Method for producing human corneal epithelium sheet |
| ES2901396T3 (es) | 2013-03-14 | 2022-03-22 | Caribou Biosciences Inc | Composiciones y métodos de ácidos nucleicos dirigidos a ácido nucleico |
| WO2014186585A2 (en) | 2013-05-15 | 2014-11-20 | Sangamo Biosciences, Inc. | Methods and compositions for treatment of a genetic condition |
| IL303973A (en) | 2013-07-09 | 2023-08-01 | Harvard College | Multiplexed RNA-guided genome engineering |
| US20150044772A1 (en) * | 2013-08-09 | 2015-02-12 | Sage Labs, Inc. | Crispr/cas system-based novel fusion protein and its applications in genome editing |
| KR20230007558A (ko) * | 2013-11-15 | 2023-01-12 | 아벨리노 랩 유에스에이, 인크. | 안과 질환과 관련된 대립유전자의 멀티플렉스 검출 방법 |
| KR20250068794A (ko) * | 2013-12-12 | 2025-05-16 | 더 브로드 인스티튜트, 인코퍼레이티드 | 게놈 편집을 위한 crispr-cas 시스템 및 조성물의 전달, 용도 및 치료적 응용 |
| 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 |
| EA201890203A1 (ru) | 2015-07-02 | 2018-07-31 | Дзе Джонс Хопкинс Юниверсити | Лечение на основе crispr/cas9 |
| AU2016335572B2 (en) | 2015-10-09 | 2022-12-08 | The Children's Hospital Of Philadelphia | Compositions and methods for treating Huntington's disease and related disorders |
| WO2018027078A1 (en) | 2016-08-03 | 2018-02-08 | President And Fellows Of Harard College | Adenosine nucleobase editors and uses thereof |
| WO2019165322A1 (en) | 2018-02-22 | 2019-08-29 | Moore Tara | Crispr/cas9 systems, and methods of use thereof |
| US20220056440A1 (en) | 2016-08-20 | 2022-02-24 | Tara Moore | Crispr gene editing for autosomal dominant diseases |
| WO2020046861A1 (en) | 2018-08-27 | 2020-03-05 | Avellino Lab Usa, Inc. | 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 |
| KR102594051B1 (ko) | 2016-08-20 | 2023-10-26 | 아벨리노 랩 유에스에이, 인크. | 단일 가이드 RNA, CRISPR/Cas9 시스템, 및 이의 사용방법 |
| 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 |
| 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 |
| EP3628056A4 (en) | 2017-04-28 | 2021-03-03 | Avellino Lab USA, Inc. | METHODS OF DETECTION OF ALLELS ASSOCIATED WITH A KERATOCON |
| 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 |
-
2016
- 2016-11-14 WO PCT/US2016/061893 patent/WO2017083852A1/en not_active Ceased
- 2016-11-14 EP EP16865232.9A patent/EP3374502B1/en active Active
- 2016-11-14 CN CN201680077123.2A patent/CN108699542A/zh active Pending
- 2016-11-14 EP EP21204643.7A patent/EP4036228A1/en not_active Withdrawn
- 2016-11-14 KR KR1020187016752A patent/KR20180120670A/ko not_active Ceased
- 2016-11-14 JP JP2018545108A patent/JP6929865B2/ja not_active Expired - Fee Related
- 2016-11-14 ES ES16865232T patent/ES2905558T3/es active Active
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2018
- 2018-05-11 US US15/977,565 patent/US11987809B2/en active Active
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2021
- 2021-08-11 JP JP2021131116A patent/JP7239649B2/ja active Active
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