JP2023002555A5 - - Google Patents
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- JP2023002555A5 JP2023002555A5 JP2022154637A JP2022154637A JP2023002555A5 JP 2023002555 A5 JP2023002555 A5 JP 2023002555A5 JP 2022154637 A JP2022154637 A JP 2022154637A JP 2022154637 A JP2022154637 A JP 2022154637A JP 2023002555 A5 JP2023002555 A5 JP 2023002555A5
- Authority
- JP
- Japan
- Prior art keywords
- composition
- nuclease
- gene encoding
- recognition sequence
- gene
- 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 12
- 108090000623 proteins and genes Proteins 0.000 claims 8
- 108010069091 Dystrophin Proteins 0.000 claims 5
- 210000000663 muscle cell Anatomy 0.000 claims 4
- 210000003205 muscle Anatomy 0.000 claims 2
- 241000702421 Dependoparvovirus Species 0.000 claims 1
- 206010013801 Duchenne Muscular Dystrophy Diseases 0.000 claims 1
- 230000000295 complement effect Effects 0.000 claims 1
- 230000037433 frameshift Effects 0.000 claims 1
- 239000002502 liposome Substances 0.000 claims 1
- 108090000765 processed proteins & peptides Proteins 0.000 claims 1
- 238000011144 upstream manufacturing Methods 0.000 claims 1
Priority Applications (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2024168117A 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 |
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201461951648P | 2014-03-12 | 2014-03-12 | |
| US61/951,648 | 2014-03-12 | ||
| JP2021014851A 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 |
Related Parent Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2021014851A Division 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 |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2024168117A Division 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 |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2023002555A JP2023002555A (ja) | 2023-01-10 |
| JP2023002555A5 true JP2023002555A5 (https=) | 2023-06-06 |
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 Before (3)
| 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 |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| 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 (https=) |
| EP (3) | EP4659765A3 (https=) |
| JP (5) | JP2017512767A (https=) |
| AU (3) | AU2015229299A1 (https=) |
| CA (1) | CA2942268A1 (https=) |
| DK (1) | DK3116533T3 (https=) |
| ES (2) | ES3037550T3 (https=) |
| WO (1) | WO2015138739A2 (https=) |
Families Citing this family (55)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US10323236B2 (en) | 2011-07-22 | 2019-06-18 | 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 |
| US9228207B2 (en) | 2013-09-06 | 2016-01-05 | President And Fellows Of Harvard College | Switchable gRNAs comprising aptamers |
| US9526784B2 (en) | 2013-09-06 | 2016-12-27 | President And Fellows Of Harvard College | Delivery system for functional nucleases |
| US9322037B2 (en) | 2013-09-06 | 2016-04-26 | President And Fellows Of Harvard College | Cas9-FokI fusion proteins and uses thereof |
| CN106459995B (zh) | 2013-11-07 | 2020-02-21 | 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 |
| KR102170502B1 (ko) | 2013-12-11 | 2020-10-28 | 리제너론 파마슈티칼스 인코포레이티드 | 게놈의 표적화된 변형을 위한 방법 및 조성물 |
| US20150165054A1 (en) | 2013-12-12 | 2015-06-18 | President And Fellows Of Harvard College | Methods for correcting caspase-9 point mutations |
| US10077453B2 (en) | 2014-07-30 | 2018-09-18 | President And Fellows Of Harvard College | CAS9 proteins including ligand-dependent inteins |
| KR102531016B1 (ko) | 2014-11-21 | 2023-05-10 | 리제너론 파마슈티칼스 인코포레이티드 | 쌍 형성된 가이드 rna를 사용하는 표적화된 유전자 변형을 위한 방법 및 조성물 |
| US20170266320A1 (en) * | 2014-12-01 | 2017-09-21 | President And Fellows Of Harvard College | RNA-Guided Systems for In Vivo Gene Editing |
| EP3748004A1 (en) * | 2015-04-01 | 2020-12-09 | Editas Medicine, Inc. | Crispr/cas-related methods and compositions for treating duchenne muscular dystrophy and becker muscular dystrophy |
| US10603363B2 (en) * | 2015-09-08 | 2020-03-31 | Precision Biosciences, Inc. | Treatment of retinitis pigmentosa using engineered meganucleases |
| JP6816133B2 (ja) | 2015-10-05 | 2021-01-20 | プレシジョン バイオサイエンシズ，インク． | 改変ヒトｔ細胞受容体アルファ定常領域遺伝子を含む遺伝子改変細胞 |
| EP3359660B1 (en) * | 2015-10-05 | 2019-12-04 | Precision Biosciences, Inc. | Engineered meganucleases with recognition sequences found in the human t cell receptor alpha constant region gene |
| IL310721B2 (en) | 2015-10-23 | 2025-11-01 | Harvard College | Nucleobase editors and uses thereof |
| CN113831407B (zh) | 2016-05-20 | 2024-06-11 | 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 |
| CN110214183A (zh) | 2016-08-03 | 2019-09-06 | 哈佛大学的校长及成员们 | 腺苷核碱基编辑器及其用途 |
| WO2018031683A1 (en) | 2016-08-09 | 2018-02-15 | 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 |
| KR102622411B1 (ko) | 2016-10-14 | 2024-01-10 | 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 |
| US12390514B2 (en) | 2017-03-09 | 2025-08-19 | 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 |
| US11542496B2 (en) | 2017-03-10 | 2023-01-03 | 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 |
| EP3617311B1 (en) * | 2017-03-30 | 2024-11-20 | 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 |
| EP3645038B1 (en) | 2017-06-30 | 2026-02-18 | 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 |
| 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 |
| US12406749B2 (en) | 2017-12-15 | 2025-09-02 | The Broad Institute, Inc. | Systems and methods for predicting repair outcomes in genetic engineering |
| US11786554B2 (en) | 2018-04-12 | 2023-10-17 | Precision Biosciences, Inc. | Optimized engineered nucleases having specificity for the human T cell receptor alpha constant region gene |
| US12157760B2 (en) | 2018-05-23 | 2024-12-03 | The Broad Institute, Inc. | Base editors and uses thereof |
| US12522807B2 (en) | 2018-07-09 | 2026-01-13 | 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 |
| US12351837B2 (en) | 2019-01-23 | 2025-07-08 | The Broad Institute, Inc. | Supernegatively charged proteins and uses thereof |
| WO2020191233A1 (en) | 2019-03-19 | 2020-09-24 | The Broad Institute, Inc. | Methods and compositions for editing nucleotide sequences |
| US12473543B2 (en) | 2019-04-17 | 2025-11-18 | 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 |
| IL296986A (en) * | 2020-04-06 | 2022-12-01 | Homology Medicines Inc | Adeno-associated virus preparations 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 |
| IL297761A (en) | 2020-05-08 | 2022-12-01 | Broad Inst Inc | Methods and compositions for simultaneously editing two helices of a designated double-helix nucleotide sequence |
| US20230183664A1 (en) | 2020-05-11 | 2023-06-15 | Precision Biosciences, Inc. | Self-limiting viral vectors encoding nucleases |
| JP7642809B2 (ja) * | 2020-11-12 | 2025-03-10 | プレシジョン バイオサイエンシズ，インク． | ジストロフィン遺伝子内の認識配列に特異性を有する遺伝子操作されたメガヌクレアーゼ |
| WO2023108025A1 (en) * | 2021-12-08 | 2023-06-15 | 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 |
Family Cites Families (20)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| EP3202899B1 (en) * | 2003-01-28 | 2020-10-21 | Cellectis | Custom-made meganuclease and use thereof |
| ES2582091T3 (es) | 2005-10-18 | 2016-09-09 | Precision Biosciences | Meganucleasas diseñadas racionalmente con especificidad de secuencia y afinidad de unión a ADN alteradas |
| EP2215223B1 (en) | 2007-10-31 | 2013-05-01 | Precision Biosciences, Inc. | 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) |
| EP2313498B1 (en) | 2008-07-14 | 2017-03-15 | 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 |
| US20130337454A1 (en) * | 2010-10-27 | 2013-12-19 | Philippe Duchateau | Method for increasing the efficiency of double-strand break-induced mutagenesis |
| EP2714936B1 (en) | 2011-06-01 | 2018-12-12 | Precision Biosciences, Inc. | Methods and products for producing engineered mammalian cell lines with amplified transgenes |
| 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 |
| KR102121086B1 (ko) * | 2012-11-01 | 2020-06-09 | 팩터 바이오사이언스 인크. | 세포에서 단백질을 발현시키는 방법들과 생성물들 |
| 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 |
| AU2016344609B2 (en) * | 2015-10-28 | 2022-05-12 | Vertex Pharmaceuticals Incorporated | Materials and methods for treatment of duchenne muscular dystrophy |
| JP7642809B2 (ja) * | 2020-11-12 | 2025-03-10 | プレシジョン バイオサイエンシズ，インク． | ジストロフィン遺伝子内の認識配列に特異性を有する遺伝子操作されたメガヌクレアーゼ |
-
2015
- 2015-03-12 EP EP25191909.8A patent/EP4659765A3/en active Pending
- 2015-03-12 JP JP2016556801A patent/JP2017512767A/ja active Pending
- 2015-03-12 EP EP15761320.9A patent/EP3116533B1/en active Active
- 2015-03-12 CA CA2942268A patent/CA2942268A1/en active Pending
- 2015-03-12 US US15/124,984 patent/US20170106055A1/en not_active Abandoned
- 2015-03-12 EP EP20189872.3A patent/EP3858376B1/en active Active
- 2015-03-12 ES ES20189872T patent/ES3037550T3/es active Active
- 2015-03-12 ES ES15761320T patent/ES2821149T3/es active Active
- 2015-03-12 WO PCT/US2015/020205 patent/WO2015138739A2/en not_active Ceased
- 2015-03-12 DK DK15761320.9T patent/DK3116533T3/da active
- 2015-03-12 AU AU2015229299A patent/AU2015229299A1/en not_active Abandoned
-
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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