ES2821149T3 - Eliminación del exón del gen de la distrofina mediante nucleasas modificadas genéticamente - Google Patents
Eliminación del exón del gen de la distrofina mediante nucleasas modificadas genéticamente Download PDFInfo
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- ES2821149T3 ES2821149T3 ES15761320T ES15761320T ES2821149T3 ES 2821149 T3 ES2821149 T3 ES 2821149T3 ES 15761320 T ES15761320 T ES 15761320T ES 15761320 T ES15761320 T ES 15761320T ES 2821149 T3 ES2821149 T3 ES 2821149T3
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| US201461951648P | 2014-03-12 | 2014-03-12 | |
| PCT/US2015/020205 WO2015138739A2 (en) | 2014-03-12 | 2015-03-12 | Dystrophin gene oxon deletion using engineered nucleases |
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| ES2821149T3 true ES2821149T3 (es) | 2021-04-23 |
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| ES20189872T Active ES3037550T3 (en) | 2014-03-12 | 2015-03-12 | Dystrophin gene exon deletion using engineered nucleases |
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| ES20189872T Active ES3037550T3 (en) | 2014-03-12 | 2015-03-12 | Dystrophin gene exon deletion using engineered nucleases |
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| WO2013066438A2 (en) | 2011-07-22 | 2013-05-10 | President And Fellows Of Harvard College | Evaluation and improvement of nuclease cleavage specificity |
| EP3456831B1 (en) | 2013-04-16 | 2021-07-14 | Regeneron Pharmaceuticals, Inc. | Targeted modification of rat genome |
| 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 |
| US9737604B2 (en) | 2013-09-06 | 2017-08-22 | President And Fellows Of Harvard College | Use of cationic lipids to deliver CAS9 |
| 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 |
| KR20230054509A (ko) | 2013-11-07 | 2023-04-24 | 에디타스 메디신, 인코포레이티드 | 지배적인 gRNA를 이용하는 CRISPR-관련 방법 및 조성물 |
| MX388127B (es) | 2013-12-11 | 2025-03-19 | Regeneron Pharma | Metodos y composiciones para la modificacion dirigida de un genoma. |
| 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 |
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| AU2016333886B2 (en) * | 2015-10-05 | 2020-10-08 | Precision Biosciences, Inc. | Engineered meganucleases with recognition sequences found in the human T cell receptor alpha constant region gene |
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| 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 |
| 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 |
| CA3044531A1 (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 |
| WO2018119359A1 (en) | 2016-12-23 | 2018-06-28 | President And Fellows Of Harvard College | Editing of ccr5 receptor gene to protect against hiv infection |
| JOP20190166A1 (ar) * | 2017-01-05 | 2019-07-02 | Univ Texas | استراتيجية مثلى من أجل تعديلات تخطي إكسون باستخدام crispr/cas9 مع متواليات توجيه ثلاثي |
| EP3592381A1 (en) | 2017-03-09 | 2020-01-15 | President and Fellows of Harvard College | Cancer vaccine |
| EP3592853A1 (en) | 2017-03-09 | 2020-01-15 | President and Fellows of Harvard College | Suppression of pain by gene editing |
| 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 |
| KR102758434B1 (ko) * | 2017-03-30 | 2025-01-21 | 고쿠리츠 다이가쿠 호진 교토 다이가쿠 | 게놈 편집에 의한 엑손 스키핑 유도 방법 |
| 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 |
| AU2018292526A1 (en) | 2017-06-30 | 2020-01-16 | Precision Biosciences, Inc. | Genetically-modified T cells comprising a modified intron in the T cell receptor alpha gene |
| 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 |
| WO2019200122A1 (en) | 2018-04-12 | 2019-10-17 | Precision Biosciences, Inc. | Optimized engineered nucleases having specificity for the human t cell receptor alpha constant region gene |
| WO2019226953A1 (en) | 2018-05-23 | 2019-11-28 | The Broad Institute, Inc. | Base editors and uses thereof |
| US12281338B2 (en) | 2018-10-29 | 2025-04-22 | The Broad Institute, Inc. | Nucleobase editors comprising GeoCas9 and uses thereof |
| WO2020106633A1 (en) * | 2018-11-19 | 2020-05-28 | Altius Institute For Biomedical Sciences | Compositions and methods for detection of cleavage of genomic dna by a nuclease |
| US12351837B2 (en) | 2019-01-23 | 2025-07-08 | The Broad Institute, Inc. | Supernegatively charged proteins and uses thereof |
| 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 |
| US20220195407A1 (en) | 2019-05-07 | 2022-06-23 | Precision Biosciences, Inc. | Optimization of engineered meganucleases for recognition sequences |
| US12435330B2 (en) | 2019-10-10 | 2025-10-07 | The Broad Institute, Inc. | Methods and compositions for prime editing RNA |
| CN115484975A (zh) * | 2020-04-06 | 2022-12-16 | 同源药物公司 | 用于ids基因转移的腺相关病毒组合物和其使用方法 |
| EP4143308A4 (en) * | 2020-04-27 | 2024-06-26 | The Trustees of The University of Pennsylvania | COMPOSITIONS AND METHODS FOR REDUCING NUCLEASE EXPRESSION AND OFF-TARGET ACTIVITY USING A PROMOTER WITH LOW TRANSCRIPTION ACTIVITY |
| 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 |
| WO2021231259A1 (en) | 2020-05-11 | 2021-11-18 | Precision Biosciences, Inc. | Self-limiting viral vectors encoding nucleases |
| EP4244342A1 (en) * | 2020-11-12 | 2023-09-20 | Precision BioSciences, Inc. | Engineered meganucleases having specificity for recognition sequences in the dystrophin gene |
| EP4444886A1 (en) * | 2021-12-08 | 2024-10-16 | Mammoth Biosciences, Inc. | Systems and uses thereof for the treatment of dmd-associated diseases |
| JP7691595B1 (ja) | 2024-02-26 | 2025-06-12 | 株式会社ニチレイフーズ | 欠失した改変ゲノムｄｎａを含む細胞の作製方法、該細胞を含む生物体の作製方法 |
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| EP1590468A2 (en) * | 2003-01-28 | 2005-11-02 | Cellectis | Use of meganucleases for inducing homologous recombination ex vivo and in toto in vertebrate somatic tissues and application thereof |
| ES2626025T3 (es) | 2005-10-18 | 2017-07-21 | Precision Biosciences | Meganucleasas diseñadas racionalmente con especificidad de secuencia y afinidad de unión a ADN alteradas |
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| WO2009101399A1 (en) * | 2008-02-12 | 2009-08-20 | Isis Innovation Limited | Treatment of muscular dystrophy using peptide nucleic acid ( pna) |
| DK2313498T3 (en) | 2008-07-14 | 2017-05-22 | Prec Biosciences Inc | RECOGNITION SEQUENCES FOR I-CREI-DERIVED MECHANUCLEASES AND APPLICATIONS THEREOF |
| 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 |
| SG169914A1 (en) * | 2009-09-29 | 2011-04-29 | Univ Singapore | A clinical method for genotyping large genes for mutations that potentially cause disease |
| US20130145487A1 (en) * | 2010-05-12 | 2013-06-06 | Cellectis | Meganuclease variants cleaving a dna target sequence from the dystrophin gene and uses thereof |
| JP2013544082A (ja) * | 2010-10-27 | 2013-12-12 | セレクティス | 二本鎖破断−誘導変異誘発の効率を増大させる方法 |
| DK3489366T3 (da) | 2011-06-01 | 2020-02-24 | Prec Biosciences Inc | Fremgangsmåder og produkter til produktion af manipulerede mammale cellelinier med forstærkede transgener |
| EP2766483B1 (en) * | 2011-10-10 | 2022-03-23 | The Hospital For Sick Children | Methods and compositions for screening and treating developmental disorders |
| US8885683B2 (en) * | 2011-12-21 | 2014-11-11 | Canon Kabushiki Kaisha | Process for forming microstructure of nitride semiconductor, surface emitting laser using two-dimensional photonic crystal and production process thereof |
| WO2013163628A2 (en) * | 2012-04-27 | 2013-10-31 | Duke University | Genetic correction of mutated genes |
| EP2684892A1 (en) * | 2012-07-13 | 2014-01-15 | Association Française contre les Myopathies | Compositions and methods for duchenne muscular dystrophy gene therapy |
| BR122019025681B1 (pt) * | 2012-11-01 | 2023-04-18 | Factor Bioscience Inc | Método para inserir uma sequência de ácido nucleico em uma localização segura de um genoma de uma célula |
| US8697359B1 (en) * | 2012-12-12 | 2014-04-15 | The Broad Institute, Inc. | CRISPR-Cas systems and methods for altering expression of gene products |
| JP7085716B2 (ja) * | 2013-06-05 | 2022-06-17 | デューク ユニバーシティ | Ｒｎａガイド遺伝子編集及び遺伝子調節 |
| CA2878645C (en) | 2014-01-22 | 2017-02-21 | Alfa Wassermann, Inc. | Centrifugation systems with non-contact seal assemblies |
| ES3040945T3 (en) * | 2015-10-28 | 2025-11-06 | Vertex Pharma | Materials and methods for treatment of duchenne muscular dystrophy |
| EP4244342A1 (en) * | 2020-11-12 | 2023-09-20 | Precision BioSciences, Inc. | Engineered meganucleases having specificity for recognition sequences in the dystrophin gene |
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| AU2020260387A1 (en) | 2020-11-19 |
| JP2019214609A (ja) | 2019-12-19 |
| EP3116533A4 (en) | 2018-04-18 |
| WO2015138739A2 (en) | 2015-09-17 |
| JP2021088563A (ja) | 2021-06-10 |
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| JP2023002555A (ja) | 2023-01-10 |
| EP3116533A2 (en) | 2017-01-18 |
| ES3037550T3 (en) | 2025-10-03 |
| WO2015138739A8 (en) | 2015-11-19 |
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