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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Applications Claiming Priority (2)
| Application Number | Priority Date | Filing Date | Title |
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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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| EP (3) | EP3116533B1 (https=) |
| JP (5) | JP2017512767A (https=) |
| AU (3) | AU2015229299A1 (https=) |
| CA (1) | CA2942268A1 (https=) |
| DK (1) | DK3116533T3 (https=) |
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| JP6261500B2 (ja) | 2011-07-22 | 2018-01-17 | 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 | ヌクレアーゼ切断特異性の評価および改善 |
| SI2986729T1 (sl) | 2013-04-16 | 2019-02-28 | Regeneron Pharmaceuticals, Inc. | Ciljana sprememba genoma podgane |
| 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 |
| US9526784B2 (en) | 2013-09-06 | 2016-12-27 | President And Fellows Of Harvard College | Delivery system for functional nucleases |
| US9388430B2 (en) | 2013-09-06 | 2016-07-12 | President And Fellows Of Harvard College | Cas9-recombinase fusion proteins and uses thereof |
| AU2014346559B2 (en) | 2013-11-07 | 2020-07-09 | Editas Medicine,Inc. | CRISPR-related methods and compositions with governing gRNAs |
| US9546384B2 (en) | 2013-12-11 | 2017-01-17 | Regeneron Pharmaceuticals, Inc. | Methods and compositions for the targeted modification of a mouse genome |
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| 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 |
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| 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 |
| CA3068465A1 (en) | 2017-06-30 | 2019-01-03 | Precision Biosciences, Inc. | Genetically-modified t cells comprising a modified intron in the t cell receptor alpha gene |
| WO2019023680A1 (en) | 2017-07-28 | 2019-01-31 | President And Fellows Of Harvard College | METHODS AND COMPOSITIONS FOR EVOLUTION OF BASIC EDITORS USING PHAGE-ASSISTED CONTINUOUS EVOLUTION (PACE) |
| EP3676376B1 (en) | 2017-08-30 | 2025-01-15 | President and Fellows of Harvard College | High efficiency base editors comprising gam |
| KR20250107288A (ko) | 2017-10-16 | 2025-07-11 | 더 브로드 인스티튜트, 인코퍼레이티드 | 아데노신 염기 편집제의 용도 |
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| US12467077B2 (en) * | 2018-11-19 | 2025-11-11 | Altius Institute For Biomedical Sciences | Compositions and methods for DNA modification detection |
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| JP7657726B2 (ja) | 2019-03-19 | 2025-04-07 | ザ ブロード インスティテュート，インコーポレーテッド | 編集ヌクレオチド配列を編集するための方法および組成物 |
| US12473543B2 (en) | 2019-04-17 | 2025-11-18 | The Broad Institute, Inc. | Adenine base editors with reduced off-target effects |
| CA3137975A1 (en) | 2019-05-07 | 2020-11-12 | 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 |
| WO2021207077A1 (en) * | 2020-04-06 | 2021-10-14 | Homology Medicines, Inc. | Adeno-associated virus compositions for ids gene transfer and methods of use thereof |
| WO2021222238A1 (en) * | 2020-04-27 | 2021-11-04 | The Trustees Of The University Of Pennsylvania | Compositions and methods for reducing nuclease expression and off-target activity using a promoter with low transcriptional activity |
| DE112021002672T5 (de) | 2020-05-08 | 2023-04-13 | President And Fellows Of Harvard College | Vefahren und zusammensetzungen zum gleichzeitigen editieren beider stränge einer doppelsträngigen nukleotid-zielsequenz |
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| CA3172481A1 (en) * | 2020-11-12 | 2022-05-19 | 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 |
| WO2025182957A1 (ja) * | 2024-02-26 | 2025-09-04 | 株式会社ニチレイフーズ | 改変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、及び遺伝子産物の生産方法 |
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| US20060153826A1 (en) * | 2003-01-28 | 2006-07-13 | Sylvain Arnould | Use of meganucleases for inducing homologous recombination ex vivo and in toto in vertebrate somatic tissues and application thereof |
| CA2891996A1 (en) | 2005-10-18 | 2007-04-26 | Duke University | Rationally-designed meganucleases with altered sequence specificity and dna-binding affinity |
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| DK3211075T3 (en) | 2008-07-14 | 2019-01-21 | 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 |
| EP2633040B1 (en) * | 2010-10-27 | 2019-07-10 | Cellectis | Method for increasing the efficiency of double-strand break-induced mutagenesis |
| DK2714936T3 (en) | 2011-06-01 | 2019-03-25 | Prec Biosciences Inc | METHODS AND PRODUCTS FOR PRODUCING MANIPULATED MAMMAL CELL LINES WITH AMPLIFIED TRANSGENES |
| CA2851388C (en) * | 2011-10-10 | 2023-11-21 | 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 |
| EP3597741A1 (en) * | 2012-04-27 | 2020-01-22 | 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 |
| WO2014071219A1 (en) * | 2012-11-01 | 2014-05-08 | Factor Bioscience Inc. | Methods and products for expressing proteins in cells |
| US8697359B1 (en) * | 2012-12-12 | 2014-04-15 | The Broad Institute, Inc. | CRISPR-Cas systems and methods for altering expression of gene products |
| EP3004370B1 (en) * | 2013-06-05 | 2024-08-21 | Duke University | Rna-guided gene editing and gene regulation |
| CA2878645C (en) | 2014-01-22 | 2017-02-21 | Alfa Wassermann, Inc. | Centrifugation systems with non-contact seal assemblies |
| JP2019507579A (ja) * | 2015-10-28 | 2019-03-22 | クリスパー セラピューティクス アーゲー | デュシェンヌ型筋ジストロフィーの処置のための材料および方法 |
| CA3172481A1 (en) * | 2020-11-12 | 2022-05-19 | Precision Biosciences, Inc. | Engineered meganucleases having specificity for recognition sequences in the dystrophin gene |
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2015
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- 2015-03-12 AU AU2015229299A patent/AU2015229299A1/en not_active Abandoned
- 2015-03-12 WO PCT/US2015/020205 patent/WO2015138739A2/en not_active Ceased
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- 2015-03-12 EP EP15761320.9A patent/EP3116533B1/en active Active
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- 2015-03-12 EP EP25191909.8A patent/EP4659765A3/en active Pending
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2019
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2020
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2021
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2022
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2023
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2025
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| EP3116533A2 (en) | 2017-01-18 |
| JP6832995B2 (ja) | 2021-02-24 |
| US20240156919A1 (en) | 2024-05-16 |
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| US20250152679A1 (en) | 2025-05-15 |
| US20170106055A1 (en) | 2017-04-20 |
| EP3116533A4 (en) | 2018-04-18 |
| EP3116533B1 (en) | 2020-08-12 |
| CA2942268A1 (en) | 2015-09-17 |
| EP3858376C0 (en) | 2025-07-30 |
| EP3858376B1 (en) | 2025-07-30 |
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| US20210145940A1 (en) | 2021-05-20 |
| WO2015138739A3 (en) | 2015-10-22 |
| JP2021088563A (ja) | 2021-06-10 |
| US20190269762A1 (en) | 2019-09-05 |
| AU2024202080A1 (en) | 2024-04-18 |
| JP2017512767A (ja) | 2017-05-25 |
| DK3116533T3 (da) | 2020-08-24 |
| US20190365870A1 (en) | 2019-12-05 |
| JP2025013784A (ja) | 2025-01-28 |
| EP3858376A1 (en) | 2021-08-04 |
| AU2020260387A1 (en) | 2020-11-19 |
| ES3037550T3 (en) | 2025-10-03 |
| WO2015138739A8 (en) | 2015-11-19 |
| WO2015138739A2 (en) | 2015-09-17 |
| AU2015229299A1 (en) | 2016-09-22 |
| EP4659765A3 (en) | 2026-02-18 |
| JP2023002555A (ja) | 2023-01-10 |
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