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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- 102000039446 nucleic acids Human genes 0.000 claims description 55
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- 210000000130 stem cell Anatomy 0.000 claims description 24
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- 239000003153 chemical reaction reagent Substances 0.000 claims description 16
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- 108091032973 (ribonucleotides)n+m Proteins 0.000 claims description 2
- 108090000623 proteins and genes Proteins 0.000 claims description 2
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- 208000003923 Hereditary Corneal Dystrophies Diseases 0.000 claims 14
- 206010011005 corneal dystrophy Diseases 0.000 claims 14
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- 108091007000 SLC44A3 Proteins 0.000 claims 8
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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 (cg-RX-API-DMAC7.html) | 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 (cg-RX-API-DMAC7.html) |
| EP (2) | EP4036228A1 (cg-RX-API-DMAC7.html) |
| JP (2) | JP6929865B2 (cg-RX-API-DMAC7.html) |
| KR (1) | KR20180120670A (cg-RX-API-DMAC7.html) |
| CN (1) | CN108699542A (cg-RX-API-DMAC7.html) |
| ES (1) | ES2905558T3 (cg-RX-API-DMAC7.html) |
| WO (1) | WO2017083852A1 (cg-RX-API-DMAC7.html) |
Families Citing this family (24)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US9228207B2 (en) | 2013-09-06 | 2016-01-05 | President And Fellows Of Harvard College | Switchable gRNAs comprising aptamers |
| KR20230155013A (ko) * | 2016-08-20 | 2023-11-09 | 아벨리노 랩 유에스에이, 인크. | 단일 가이드 RNA, CRISPR/Cas9 시스템, 및 이의 사용방법 |
| US12390514B2 (en) | 2017-03-09 | 2025-08-19 | President And Fellows Of Harvard College | Cancer vaccine |
| JP2020510439A (ja) | 2017-03-10 | 2020-04-09 | 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 | シトシンからグアニンへの塩基編集因子 |
| WO2018226602A1 (en) * | 2017-06-05 | 2018-12-13 | Research Institute At Nationwide Children's Hospital | Enhanced modified viral capsid proteins |
| WO2018225807A1 (ja) * | 2017-06-07 | 2018-12-13 | 国立大学法人東京大学 | 顆粒状角膜変性症に対する遺伝子治療薬 |
| EP3658573A1 (en) | 2017-07-28 | 2020-06-03 | President and Fellows of Harvard College | Methods and compositions for evolving base editors using phage-assisted continuous evolution (pace) |
| WO2019060469A2 (en) * | 2017-09-19 | 2019-03-28 | Massachusetts Institute Of Technology | CAS9 STREPTOCOCCUS CANIS AS A GENOMIC ENGINEERING PLATFORM WITH NEW PAM SPECIFICITY |
| CN118581083A (zh) * | 2017-09-28 | 2024-09-03 | 株式会社图尔金 | 用于基因表达调节的人工基因组操纵 |
| US11572574B2 (en) | 2017-09-28 | 2023-02-07 | Toolgen Incorporated | Artificial genome manipulation for gene expression regulation |
| WO2019118935A1 (en) * | 2017-12-14 | 2019-06-20 | Casebia Therapeutics Limited Liability Partnership | Novel rna-programmable endonuclease systems and their use in genome editing and other applications |
| EP3724214A4 (en) | 2017-12-15 | 2021-09-01 | The Broad Institute Inc. | SYSTEMS AND METHODS FOR PREDICTING REPAIR RESULTS IN GENETIC ENGINEERING |
| WO2019210109A1 (en) | 2018-04-25 | 2019-10-31 | Children's Medical Center Corporation | Abcb5 ligands and substrates |
| US12281338B2 (en) | 2018-10-29 | 2025-04-22 | The Broad Institute, Inc. | Nucleobase editors comprising GeoCas9 and uses thereof |
| KR20210106527A (ko) * | 2018-12-20 | 2021-08-30 | 페킹 유니버시티 | 바코드화 가이드 rna 구축물을 사용한 고효율의 유전자 스크리닝을 위한 조성물 및 방법 |
| US12351837B2 (en) | 2019-01-23 | 2025-07-08 | The Broad Institute, Inc. | Supernegatively charged proteins and uses thereof |
| GB2601617B (en) * | 2019-03-19 | 2024-02-21 | Broad Inst Inc | Methods and compositions for editing nucleotide sequences |
| US20220184136A1 (en) * | 2019-03-28 | 2022-06-16 | Children's Medical Center Corporation | Highly functional manufactured abcb5+ mesenchymal stem cells |
| US12473543B2 (en) | 2019-04-17 | 2025-11-18 | 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 |
| EP4065702A4 (en) * | 2019-11-27 | 2024-03-20 | 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)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| 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 |
| 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 |
| 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 |
| ATE318327T1 (de) | 1996-06-04 | 2006-03-15 | Univ Utah Res Found | Fluoreszenz-donor-akzeptor paar |
| 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 |
| WO2000058509A2 (en) | 1999-03-29 | 2000-10-05 | 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 |
| EP1290220B1 (en) | 2000-04-13 | 2007-12-19 | Georgetown University | Genetic diagnosis of qt prolongations as adverse drug reactions |
| 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 |
| JP4229394B2 (ja) | 2003-08-06 | 2009-02-25 | 日本電信電話株式会社 | 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 |
| 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 |
| EP1766458A4 (en) | 2004-05-19 | 2010-05-05 | Vp Holding Llc | OPTICAL SENSOR WITH PLASMONIC LAYER STRUCTURE FOR EFFICIENT DETECTION OF CHEMICAL GROUPS BY MEANS OF SERS |
| 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 |
| 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 |
| KR20070076532A (ko) | 2006-01-18 | 2007-07-24 | 메디제네스(주) | 각막이영양증 진단용 ｄｎａ 칩 |
| 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 |
| SG11201500852WA (en) | 2012-08-29 | 2015-04-29 | Sangamo Biosciences Inc | Methods and compositions for treatment of a genetic condition |
| US20140310830A1 (en) | 2012-12-12 | 2014-10-16 | Feng Zhang | CRISPR-Cas Nickase Systems, Methods And Compositions For Sequence Manipulation in Eukaryotes |
| EP2931899A1 (en) | 2012-12-12 | 2015-10-21 | The Broad Institute, Inc. | Functional genomics using crispr-cas systems, compositions, methods, knock out libraries and applications thereof |
| EP2896697B1 (en) | 2012-12-12 | 2015-09-02 | The Broad Institute, Inc. | Engineering of systems, methods and optimized guide compositions for sequence manipulation |
| KR20150105635A (ko) | 2012-12-12 | 2015-09-17 | 더 브로드 인스티튜트, 인코퍼레이티드 | 서열 조작을 위한 crispr-cas 성분 시스템, 방법 및 조성물 |
| US20140186919A1 (en) | 2012-12-12 | 2014-07-03 | Feng Zhang | Engineering and optimization of improved systems, methods and enzyme compositions for sequence manipulation |
| PL2771468T3 (pl) | 2012-12-12 | 2015-07-31 | Broad Inst Inc | Inżynieria systemów, metod i zoptymalizowanych kompozycji kierujących dla manipulacji sekwencjami |
| IL239317B (en) | 2012-12-12 | 2022-07-01 | Broad Inst Inc | 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 |
| 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 |
| DK2964238T3 (da) * | 2013-02-19 | 2021-03-22 | Childrens Medical Center | Abcb5(+) stem cells for treating ocular disease |
| JP6414945B2 (ja) | 2013-03-11 | 2018-10-31 | Ｊｃｒファーマ株式会社 | ヒト角膜上皮シートの製造法 |
| AU2014235794A1 (en) | 2013-03-14 | 2015-10-22 | Caribou Biosciences, Inc. | Compositions and methods of nucleic acid-targeting nucleic acids |
| WO2014186585A2 (en) | 2013-05-15 | 2014-11-20 | Sangamo Biosciences, Inc. | Methods and compositions for treatment of a genetic condition |
| SG11201600059QA (en) | 2013-07-09 | 2016-02-26 | Harvard College | Multiplex rna-guided genome engineering |
| WO2015021426A1 (en) * | 2013-08-09 | 2015-02-12 | Sage Labs, Inc. | A crispr/cas system-based novel fusion protein and its application in genome editing |
| JP6672147B2 (ja) * | 2013-11-15 | 2020-03-25 | アベリノ ラボ ユーエスエー インコーポレイテッドＡｖｅｌｌｉｎｏ Ｌａｂ ＵＳＡ， Ｉｎｃ． | 眼疾患に関連したアレルのマルチプレックス検出方法 |
| CN118813621A (zh) * | 2013-12-12 | 2024-10-22 | 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 |
| CA2989331A1 (en) * | 2015-07-02 | 2017-01-05 | The Johns Hopkins University | Crispr/cas9-based treatments |
| US11473084B2 (en) | 2015-10-09 | 2022-10-18 | The Children's Hospital Of Philadelphia | Compositions and methods for treating Huntington's disease and related disorders |
| JP7231935B2 (ja) | 2016-08-03 | 2023-03-08 | 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 | アデノシン核酸塩基編集因子およびそれらの使用 |
| US20210222171A1 (en) | 2016-08-20 | 2021-07-22 | Avellino Lab Usa, Inc. | Crispr/cas9 systems, and methods of use thereof |
| KR20230155013A (ko) | 2016-08-20 | 2023-11-09 | 아벨리노 랩 유에스에이, 인크. | 단일 가이드 RNA, CRISPR/Cas9 시스템, 및 이의 사용방법 |
| 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 |
| WO2019165322A1 (en) | 2018-02-22 | 2019-08-29 | Moore Tara | 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 |
| 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 |
| CN110997940A (zh) | 2017-04-28 | 2020-04-10 | 阿维利诺美国实验室股份有限公司 | 用于检测与圆锥角膜相关的等位基因的方法 |
| 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 EP21204643.7A patent/EP4036228A1/en not_active Withdrawn
- 2016-11-14 ES ES16865232T patent/ES2905558T3/es active Active
- 2016-11-14 CN CN201680077123.2A patent/CN108699542A/zh active Pending
- 2016-11-14 JP JP2018545108A patent/JP6929865B2/ja not_active Expired - Fee Related
- 2016-11-14 KR KR1020187016752A patent/KR20180120670A/ko not_active Ceased
- 2016-11-14 EP EP16865232.9A patent/EP3374502B1/en 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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