JP2017512767A5 - - Google Patents
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- Publication number
- JP2017512767A5 JP2017512767A5 JP2016556801A JP2016556801A JP2017512767A5 JP 2017512767 A5 JP2017512767 A5 JP 2017512767A5 JP 2016556801 A JP2016556801 A JP 2016556801A JP 2016556801 A JP2016556801 A JP 2016556801A JP 2017512767 A5 JP2017512767 A5 JP 2017512767A5
- Authority
- JP
- Japan
- Prior art keywords
- nuclease
- medicament according
- nucleases
- seq
- exon
- Prior art date
- Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
- Pending
Links
- 101710163270 Nuclease Proteins 0.000 claims 22
- 239000003814 drug Substances 0.000 claims 14
- 238000000034 method Methods 0.000 claims 5
- 108020004414 DNA Proteins 0.000 claims 3
- 108010069091 Dystrophin Proteins 0.000 claims 3
- 108091032973 (ribonucleotides)n+m Proteins 0.000 claims 2
- 108091033409 CRISPR Proteins 0.000 claims 2
- 238000010354 CRISPR gene editing Methods 0.000 claims 2
- 241000702421 Dependoparvovirus Species 0.000 claims 2
- 238000010459 TALEN Methods 0.000 claims 2
- 108010043645 Transcription Activator-Like Effector Nucleases Proteins 0.000 claims 2
- 108090000623 proteins and genes Proteins 0.000 claims 2
- 206010013801 Duchenne Muscular Dystrophy Diseases 0.000 claims 1
- 102000001039 Dystrophin Human genes 0.000 claims 1
- 108091028043 Nucleic acid sequence Proteins 0.000 claims 1
- 238000011144 upstream manufacturing Methods 0.000 claims 1
- 108700026220 vif Genes Proteins 0.000 claims 1
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201461951648P | 2014-03-12 | 2014-03-12 | |
| US61/951,648 | 2014-03-12 | ||
| PCT/US2015/020205 WO2015138739A2 (en) | 2014-03-12 | 2015-03-12 | Dystrophin gene oxon deletion using engineered nucleases |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2019152614A Division JP6832995B2 (ja) | 2014-03-12 | 2019-08-23 | 改変ヌクレアーゼを用いたジストロフィン遺伝子エクソンの欠失 |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2017512767A JP2017512767A (ja) | 2017-05-25 |
| JP2017512767A5 true JP2017512767A5 (enExample) | 2018-04-26 |
Family
ID=54072591
Family Applications (5)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2016556801A Pending JP2017512767A (ja) | 2014-03-12 | 2015-03-12 | 改変ヌクレアーゼを用いたジストロフィン遺伝子エクソンの欠失 |
| JP2019152614A Active JP6832995B2 (ja) | 2014-03-12 | 2019-08-23 | 改変ヌクレアーゼを用いたジストロフィン遺伝子エクソンの欠失 |
| JP2021014851A Pending JP2021088563A (ja) | 2014-03-12 | 2021-02-02 | 改変ヌクレアーゼを用いたジストロフィン遺伝子エクソンの欠失 |
| JP2022154637A Pending JP2023002555A (ja) | 2014-03-12 | 2022-09-28 | 改変ヌクレアーゼを用いたジストロフィン遺伝子エクソンの欠失 |
| JP2024168117A Pending JP2025013784A (ja) | 2014-03-12 | 2024-09-27 | 改変ヌクレアーゼを用いたジストロフィン遺伝子エクソンの欠失 |
Family Applications After (4)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2019152614A Active JP6832995B2 (ja) | 2014-03-12 | 2019-08-23 | 改変ヌクレアーゼを用いたジストロフィン遺伝子エクソンの欠失 |
| JP2021014851A Pending JP2021088563A (ja) | 2014-03-12 | 2021-02-02 | 改変ヌクレアーゼを用いたジストロフィン遺伝子エクソンの欠失 |
| JP2022154637A Pending JP2023002555A (ja) | 2014-03-12 | 2022-09-28 | 改変ヌクレアーゼを用いたジストロフィン遺伝子エクソンの欠失 |
| JP2024168117A Pending JP2025013784A (ja) | 2014-03-12 | 2024-09-27 | 改変ヌクレアーゼを用いたジストロフィン遺伝子エクソンの欠失 |
Country Status (8)
| Country | Link |
|---|---|
| US (6) | US20170106055A1 (enExample) |
| EP (3) | EP4659765A2 (enExample) |
| JP (5) | JP2017512767A (enExample) |
| AU (3) | AU2015229299A1 (enExample) |
| CA (1) | CA2942268A1 (enExample) |
| DK (1) | DK3116533T3 (enExample) |
| ES (2) | ES3037550T3 (enExample) |
| WO (1) | WO2015138739A2 (enExample) |
Families Citing this family (55)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CA2853829C (en) | 2011-07-22 | 2023-09-26 | President And Fellows Of Harvard College | Evaluation and improvement of nuclease cleavage specificity |
| LT2986729T (lt) | 2013-04-16 | 2018-10-25 | Regeneron Pharmaceuticals, Inc. | Žiurkės genomo tikslinė modifikacija |
| US9163284B2 (en) | 2013-08-09 | 2015-10-20 | 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 |
| US9322037B2 (en) | 2013-09-06 | 2016-04-26 | President And Fellows Of Harvard College | Cas9-FokI fusion proteins and uses thereof |
| US9526784B2 (en) | 2013-09-06 | 2016-12-27 | President And Fellows Of Harvard College | Delivery system for functional nucleases |
| US9340800B2 (en) | 2013-09-06 | 2016-05-17 | President And Fellows Of Harvard College | Extended DNA-sensing GRNAS |
| EP3066201B1 (en) | 2013-11-07 | 2018-03-07 | Editas Medicine, Inc. | Crispr-related methods and compositions with governing grnas |
| CN110951779B (zh) | 2013-12-11 | 2024-04-16 | 瑞泽恩制药公司 | 用于靶向修饰基因组的方法和组合物 |
| US9840699B2 (en) | 2013-12-12 | 2017-12-12 | President And Fellows Of Harvard College | Methods for nucleic acid editing |
| WO2016022363A2 (en) | 2014-07-30 | 2016-02-11 | President And Fellows Of Harvard College | Cas9 proteins including ligand-dependent inteins |
| CA3176380A1 (en) | 2014-11-21 | 2016-05-26 | Regeneron Pharmaceuticals, Inc. | Methods and compositions for targeted genetic modification using paired guide rnas |
| US20170266320A1 (en) * | 2014-12-01 | 2017-09-21 | President And Fellows Of Harvard College | RNA-Guided Systems for In Vivo Gene Editing |
| EP3277816B1 (en) * | 2015-04-01 | 2020-06-17 | Editas Medicine, Inc. | Crispr/cas-related methods and compositions for treating duchenne muscular dystrophy and becker muscular dystrophy |
| CA2997909A1 (en) * | 2015-09-08 | 2017-03-16 | Precision Biosciences, Inc. | Treatment of retinitis pigmentosa using engineered meganucleases |
| CN108601821B (zh) | 2015-10-05 | 2023-09-19 | 精密生物科学公司 | 包含经修饰的人T细胞受体α恒定区基因的经遗传修饰的细胞 |
| ES2883116T3 (es) * | 2015-10-05 | 2021-12-07 | Prec Biosciences Inc | Meganucleasas diseñadas con secuencias de reconocimiento encontradas en el gen de la región constante alfa del receptor de células T humanas |
| IL294014B2 (en) | 2015-10-23 | 2024-07-01 | Harvard College | Nucleobase editors and uses thereof |
| CN109475109B (zh) | 2016-05-20 | 2021-10-29 | 瑞泽恩制药公司 | 用于使用多个引导rna来破坏免疫耐受性的方法 |
| AU2017306676B2 (en) | 2016-08-03 | 2024-02-22 | President And Fellows Of Harvard College | Adenosine nucleobase editors and uses thereof |
| EP3497214B1 (en) | 2016-08-09 | 2023-06-28 | President and Fellows of Harvard College | Programmable cas9-recombinase fusion proteins and uses thereof |
| US11542509B2 (en) | 2016-08-24 | 2023-01-03 | President And Fellows Of Harvard College | Incorporation of unnatural amino acids into proteins using base editing |
| WO2018071868A1 (en) | 2016-10-14 | 2018-04-19 | President And Fellows Of Harvard College | Aav delivery of nucleobase editors |
| 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 |
| 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 |
| WO2018165631A1 (en) | 2017-03-09 | 2018-09-13 | 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 |
| WO2018165629A1 (en) | 2017-03-10 | 2018-09-13 | President And Fellows Of Harvard College | Cytosine to guanine base editor |
| KR20240116572A (ko) | 2017-03-23 | 2024-07-29 | 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 | 핵산 프로그램가능한 dna 결합 단백질을 포함하는 핵염기 편집제 |
| AU2017407272B2 (en) * | 2017-03-30 | 2024-06-13 | Kyoto University | Method for inducing exon skipping by genome editing |
| US11560566B2 (en) | 2017-05-12 | 2023-01-24 | President And Fellows Of Harvard College | Aptazyme-embedded guide RNAs for use with CRISPR-Cas9 in genome editing and transcriptional activation |
| EP3645038A1 (en) | 2017-06-30 | 2020-05-06 | 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 |
| US11319532B2 (en) | 2017-08-30 | 2022-05-03 | President And Fellows Of Harvard College | High efficiency base editors comprising Gam |
| WO2019079347A1 (en) | 2017-10-16 | 2019-04-25 | The Broad Institute, Inc. | USES OF BASIC EDITORS ADENOSINE |
| US12406749B2 (en) | 2017-12-15 | 2025-09-02 | The Broad Institute, Inc. | Systems and methods for predicting repair outcomes in genetic engineering |
| AU2019252527B2 (en) | 2018-04-12 | 2024-02-22 | Precision Biosciences, Inc. | Optimized engineered nucleases having specificity for the human T cell receptor alpha constant region gene |
| EP3797160A1 (en) | 2018-05-23 | 2021-03-31 | The Broad Institute Inc. | Base editors and uses thereof |
| WO2020014261A1 (en) | 2018-07-09 | 2020-01-16 | The Broad Institute, Inc. | Rna programmable epigenetic rna modifiers and uses thereof |
| WO2020092453A1 (en) | 2018-10-29 | 2020-05-07 | The Broad Institute, Inc. | Nucleobase editors comprising geocas9 and uses thereof |
| US12467077B2 (en) * | 2018-11-19 | 2025-11-11 | Altius Institute For Biomedical Sciences | Compositions and methods for DNA modification detection |
| WO2020154500A1 (en) | 2019-01-23 | 2020-07-30 | The Broad Institute, Inc. | Supernegatively charged proteins and uses thereof |
| BR112021018607A2 (pt) | 2019-03-19 | 2021-11-23 | Massachusetts Inst Technology | Métodos e composições para editar sequências de nucleotídeos |
| EP3956349A1 (en) | 2019-04-17 | 2022-02-23 | The Broad Institute, Inc. | Adenine base editors with reduced off-target effects |
| CN120843482A (zh) | 2019-05-07 | 2025-10-28 | 精密生物科学公司 | 用于识别序列的工程化大范围核酸酶的优化 |
| US12435330B2 (en) | 2019-10-10 | 2025-10-07 | The Broad Institute, Inc. | Methods and compositions for prime editing RNA |
| BR112022019182A2 (pt) * | 2020-04-06 | 2022-11-29 | Homology Medicines Inc | Composições de vírus adeno-associadas para transferência do gene ids e métodos de uso da mesma |
| US20230175014A1 (en) * | 2020-04-27 | 2023-06-08 | 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 |
| AU2021267940A1 (en) | 2020-05-08 | 2022-12-08 | President And Fellows Of Harvard College | Methods and compositions for simultaneous editing of both strands of a target double-stranded nucleotide sequence |
| US20230183664A1 (en) | 2020-05-11 | 2023-06-15 | Precision Biosciences, Inc. | Self-limiting viral vectors encoding nucleases |
| WO2022104062A1 (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 |
| 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 |
| 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 |
Family Cites Families (20)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CA2514417A1 (en) * | 2003-01-28 | 2004-08-12 | Cellectis | Custom-made meganuclease and use thereof |
| EP2628794B1 (en) | 2005-10-18 | 2016-05-04 | Precision Biosciences | Rationally designed meganucleases with altered sequence specificity and DNA-binding affinity |
| WO2009059195A2 (en) | 2007-10-31 | 2009-05-07 | Precision Biosciences | Rationally-designed single-chain meganucleases with non-palindromic recognition sequences |
| WO2009101399A1 (en) * | 2008-02-12 | 2009-08-20 | Isis Innovation Limited | Treatment of muscular dystrophy using peptide nucleic acid ( pna) |
| WO2010009147A1 (en) | 2008-07-14 | 2010-01-21 | Precision Biosciences, Inc. | Recognition sequences for i-crei-derived meganucleases and uses thereof |
| EP2480659A2 (en) * | 2009-09-24 | 2012-08-01 | 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 |
| CA2799095A1 (en) | 2010-05-12 | 2011-11-17 | Cellectis | Meganuclease variants cleaving a dna target sequence from the dystrophin gene and uses thereof |
| CA2815512A1 (en) * | 2010-10-27 | 2012-05-03 | Philippe Duchateau | Method for increasing the efficiency of double-strand break-induced mutagenesis |
| EP3683319A1 (en) | 2011-06-01 | 2020-07-22 | Precision Biosciences, Inc. | Methods and products for producing engineered mammalian cell lines with amplified transgenes |
| DK2766483T3 (da) * | 2011-10-10 | 2022-04-19 | Hospital For Sick Children | Fremgangsmåder og sammensætninger til screening for og behandling af udviklingsforstyrrelser |
| 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 |
| EP2841572B1 (en) * | 2012-04-27 | 2019-06-19 | 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 |
| BR122019025678B1 (pt) * | 2012-11-01 | 2023-04-18 | Factor Bioscience Inc | Composições que compreendem ácido nucleico que codifica uma proteína de edição gênica |
| US8697359B1 (en) * | 2012-12-12 | 2014-04-15 | The Broad Institute, Inc. | CRISPR-Cas systems and methods for altering expression of gene products |
| CN105658805B (zh) * | 2013-06-05 | 2021-12-31 | 杜克大学 | Rna指导的基因编辑和基因调节 |
| CA2878645C (en) | 2014-01-22 | 2017-02-21 | Alfa Wassermann, Inc. | Centrifugation systems with non-contact seal assemblies |
| US11369692B2 (en) * | 2015-10-28 | 2022-06-28 | Vertex Pharmaceuticals Incorporated | Materials and methods for treatment of Duchenne Muscular Dystrophy |
| WO2022104062A1 (en) * | 2020-11-12 | 2022-05-19 | Precision Biosciences, Inc. | Engineered meganucleases having specificity for recognition sequences in the dystrophin gene |
-
2015
- 2015-03-12 ES ES20189872T patent/ES3037550T3/es active Active
- 2015-03-12 EP EP25191909.8A patent/EP4659765A2/en active Pending
- 2015-03-12 CA CA2942268A patent/CA2942268A1/en active Pending
- 2015-03-12 EP EP20189872.3A patent/EP3858376B1/en active Active
- 2015-03-12 AU AU2015229299A patent/AU2015229299A1/en not_active Abandoned
- 2015-03-12 EP EP15761320.9A patent/EP3116533B1/en active Active
- 2015-03-12 JP JP2016556801A patent/JP2017512767A/ja active Pending
- 2015-03-12 ES ES15761320T patent/ES2821149T3/es active Active
- 2015-03-12 US US15/124,984 patent/US20170106055A1/en not_active Abandoned
- 2015-03-12 WO PCT/US2015/020205 patent/WO2015138739A2/en not_active Ceased
- 2015-03-12 DK DK15761320.9T patent/DK3116533T3/da active
-
2019
- 2019-02-25 US US16/284,733 patent/US20190269762A1/en not_active Abandoned
- 2019-07-03 US US16/503,396 patent/US20190365870A1/en not_active Abandoned
- 2019-08-23 JP JP2019152614A patent/JP6832995B2/ja active Active
-
2020
- 2020-09-25 US US17/033,331 patent/US20210145940A1/en not_active Abandoned
- 2020-10-26 AU AU2020260387A patent/AU2020260387A1/en not_active Abandoned
-
2021
- 2021-02-02 JP JP2021014851A patent/JP2021088563A/ja active Pending
-
2022
- 2022-09-28 JP JP2022154637A patent/JP2023002555A/ja active Pending
-
2023
- 2023-10-19 US US18/490,484 patent/US20240156919A1/en not_active Abandoned
-
2024
- 2024-04-02 AU AU2024202080A patent/AU2024202080A1/en active Pending
- 2024-09-27 JP JP2024168117A patent/JP2025013784A/ja active Pending
-
2025
- 2025-01-17 US US19/030,187 patent/US20250152679A1/en active Pending
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