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
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) | EP3116533B1 (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 |
|---|---|---|---|---|
| WO2013066438A2 (en) | 2011-07-22 | 2013-05-10 | President And Fellows Of Harvard College | Evaluation and improvement of nuclease cleavage specificity |
| DK3456831T3 (da) | 2013-04-16 | 2021-09-06 | Regeneron Pharma | Målrettet modifikation af rottegenom |
| 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 |
| US9526784B2 (en) | 2013-09-06 | 2016-12-27 | President And Fellows Of Harvard College | Delivery system for functional nucleases |
| US9340799B2 (en) | 2013-09-06 | 2016-05-17 | President And Fellows Of Harvard College | MRNA-sensing switchable gRNAs |
| US9322037B2 (en) | 2013-09-06 | 2016-04-26 | President And Fellows Of Harvard College | Cas9-FokI fusion proteins and uses thereof |
| CN111218447B (zh) | 2013-11-07 | 2024-10-11 | 爱迪塔斯医药有限公司 | 使用统治型gRNA的CRISPR相关方法和组合物 |
| MX388127B (es) | 2013-12-11 | 2025-03-19 | Regeneron Pharma | Metodos y composiciones para la modificacion dirigida de un genoma. |
| US20150166984A1 (en) | 2013-12-12 | 2015-06-18 | President And Fellows Of Harvard College | Methods for correcting alpha-antitrypsin point mutations |
| AU2015298571B2 (en) | 2014-07-30 | 2020-09-03 | President And Fellows Of Harvard College | Cas9 proteins including ligand-dependent inteins |
| EP3221457B1 (en) | 2014-11-21 | 2019-03-20 | Regeneron Pharmaceuticals, Inc. | Methods and compositions for targeted genetic modification using paired guide rnas |
| WO2016089866A1 (en) * | 2014-12-01 | 2016-06-09 | President And Fellows Of Harvard College | Rna-guided systems for in vivo gene editing |
| CA2981508A1 (en) * | 2015-04-01 | 2016-10-06 | 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 |
| ES2803728T3 (es) | 2015-10-05 | 2021-01-29 | Prec Biosciences Inc | Células genéticamente modificadas que comprenden un gen de región constante alfa del receptor de linfocitos T humano modificado |
| ES2768984T3 (es) | 2015-10-05 | 2020-06-24 | Prec Biosciences Inc | Meganucleasas diseñadas con secuencias de reconocimiento encontradas en el gen de la región constante alfa del receptor de células T humanas |
| WO2017070632A2 (en) | 2015-10-23 | 2017-04-27 | President And Fellows Of Harvard College | Nucleobase editors and uses thereof |
| SG10202102885UA (en) | 2016-05-20 | 2021-05-28 | Regeneron Pharma | Methods for breaking immunological tolerance using multiple guide rnas |
| WO2018027078A1 (en) | 2016-08-03 | 2018-02-08 | President And Fellows Of Harard College | Adenosine nucleobase editors and uses thereof |
| AU2017308889B2 (en) | 2016-08-09 | 2023-11-09 | President And Fellows Of Harvard College | Programmable Cas9-recombinase fusion proteins and uses thereof |
| WO2018039438A1 (en) | 2016-08-24 | 2018-03-01 | President And Fellows Of Harvard College | Incorporation of unnatural amino acids into proteins using base editing |
| KR20240007715A (ko) | 2016-10-14 | 2024-01-16 | 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 | 핵염기 에디터의 aav 전달 |
| AU2017364106A1 (en) * | 2016-11-28 | 2019-06-20 | The Board Of Regents Of The University Of Texas System | Prevention of muscular dystrophy by CRISPR/Cpfl-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 |
| KR20190123328A (ko) | 2017-03-09 | 2019-10-31 | 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 | 암 백신 |
| WO2018165504A1 (en) | 2017-03-09 | 2018-09-13 | President And Fellows Of Harvard College | Suppression of pain by gene editing |
| 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 |
| IL306092A (en) | 2017-03-23 | 2023-11-01 | Harvard College | Nucleic base editors that include nucleic acid programmable DNA binding proteins |
| CN110892069B (zh) * | 2017-03-30 | 2024-02-09 | 国立大学法人京都大学 | 基于基因组编辑的外显子跳跃诱导方法 |
| 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 |
| EP4775669A2 (en) | 2017-06-30 | 2026-07-15 | Precision Biosciences, Inc. | Genetically-modified t cells comprising a modified intron in the t cell receptor alpha gene |
| 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) |
| EP3676376B1 (en) | 2017-08-30 | 2025-01-15 | 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 |
| US12406749B2 (en) | 2017-12-15 | 2025-09-02 | The Broad Institute, Inc. | Systems and methods for predicting repair outcomes in genetic engineering |
| KR102617818B1 (ko) | 2018-04-12 | 2023-12-27 | 프리시젼 바이오사이언시스 인코포레이티드 | 인간 t 세포 수용체 알파 불변 영역 유전자에 대한 특이성을 갖는 최적화된 조작된 뉴클레아제 |
| WO2019226953A1 (en) | 2018-05-23 | 2019-11-28 | The Broad Institute, Inc. | Base editors and uses thereof |
| EP3820495A4 (en) | 2018-07-09 | 2022-07-20 | The Broad Institute Inc. | RNA-PROGRAMMABLE EPIGENETIC RNA MODIFIERS AND THEIR USES |
| WO2020092453A1 (en) | 2018-10-29 | 2020-05-07 | 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 |
| WO2020191241A1 (en) | 2019-03-19 | 2020-09-24 | The Broad Institute, Inc. | Methods and compositions for editing nucleotide sequences |
| WO2020214842A1 (en) | 2019-04-17 | 2020-10-22 | The Broad Institute, Inc. | Adenine base editors with reduced off-target effects |
| CN114026228A (zh) | 2019-05-07 | 2022-02-08 | 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 |
| WO2021207077A1 (en) * | 2020-04-06 | 2021-10-14 | Homology Medicines, Inc. | Adeno-associated virus compositions for ids gene transfer and methods of use thereof |
| 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 |
| CA3177481A1 (en) | 2020-05-08 | 2021-11-11 | David R. Liu | Methods and compositions for simultaneous editing of both strands of a target double-stranded nucleotide sequence |
| WO2021231259A1 (en) | 2020-05-11 | 2021-11-18 | Precision Biosciences, Inc. | Self-limiting viral vectors encoding nucleases |
| KR20250099412A (ko) | 2020-11-12 | 2025-07-01 | 프리시젼 바이오사이언시스 인코포레이티드 | 디스트로핀 유전자 내의 인식 서열에 대해 특이성을 갖는 조작된 메가뉴클레아제 |
| EP4444886A4 (en) * | 2021-12-08 | 2026-08-12 | Mammoth Biosciences Inc | Systems and their uses for the treatment of diseases associated with DMD |
| TW202600818A (zh) * | 2024-02-26 | 2026-01-01 | 日商日冷食品股份有限公司 | 包含經修飾dna的細胞之製作方法、包含該細胞的生物體之製作方法,及基因產物之生產方法 |
| 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 |
|---|---|---|---|---|
| US20060206949A1 (en) * | 2003-01-28 | 2006-09-14 | Sylvain Arnould | Custom-made meganuclease and use thereof |
| DK3246403T3 (da) | 2005-10-18 | 2020-10-26 | Prec Biosciences | Rationelt designede meganucleaser med ændret sekvens-specificitet og dna-bindende affinitet |
| ES2422291T3 (es) | 2007-10-31 | 2013-09-10 | Prec Biosciences Inc | Meganucleasas monocatenarias diseñadas racionalmente con secuencias de reconocimiento no palindrómicas |
| WO2009101399A1 (en) * | 2008-02-12 | 2009-08-20 | Isis Innovation Limited | Treatment of muscular dystrophy using peptide nucleic acid ( pna) |
| JP2011527906A (ja) | 2008-07-14 | 2011-11-10 | プレシジョン バイオサイエンシズ,インク. | I−CreI由来メガヌクレアーゼの認識配列およびその使用 |
| 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 |
| EP2569424A1 (en) * | 2010-05-12 | 2013-03-20 | Cellectis | Meganuclease variants cleaving a dna target sequence from the dystrophin gene and uses thereof |
| SG189482A1 (en) * | 2010-10-27 | 2013-05-31 | Cellectis | 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 |
| WO2013054200A2 (en) * | 2011-10-10 | 2013-04-18 | 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 |
| 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 |
| CA2890110C (en) * | 2012-11-01 | 2023-05-02 | 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 |
| WO2014197748A2 (en) * | 2013-06-05 | 2014-12-11 | 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 |
| EP4279084B1 (en) * | 2015-10-28 | 2025-06-11 | Vertex Pharmaceuticals Inc. | Materials and methods for treatment of duchenne muscular dystrophy |
| KR20250099412A (ko) * | 2020-11-12 | 2025-07-01 | 프리시젼 바이오사이언시스 인코포레이티드 | 디스트로핀 유전자 내의 인식 서열에 대해 특이성을 갖는 조작된 메가뉴클레아제 |
-
2015
- 2015-03-12 AU AU2015229299A patent/AU2015229299A1/en not_active Abandoned
- 2015-03-12 JP JP2016556801A patent/JP2017512767A/ja active Pending
- 2015-03-12 WO PCT/US2015/020205 patent/WO2015138739A2/en not_active Ceased
- 2015-03-12 EP EP15761320.9A patent/EP3116533B1/en active Active
- 2015-03-12 CA CA2942268A patent/CA2942268A1/en active Pending
- 2015-03-12 ES ES20189872T patent/ES3037550T3/es active Active
- 2015-03-12 EP EP20189872.3A patent/EP3858376B1/en active Active
- 2015-03-12 US US15/124,984 patent/US20170106055A1/en not_active Abandoned
- 2015-03-12 EP EP25191909.8A patent/EP4659765A3/en active Pending
- 2015-03-12 DK DK15761320.9T patent/DK3116533T3/da active
- 2015-03-12 ES ES15761320T patent/ES2821149T3/es active 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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