JP2019524098A5 - - Google Patents
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- JP2019524098A5 JP2019524098A5 JP2019501985A JP2019501985A JP2019524098A5 JP 2019524098 A5 JP2019524098 A5 JP 2019524098A5 JP 2019501985 A JP2019501985 A JP 2019501985A JP 2019501985 A JP2019501985 A JP 2019501985A JP 2019524098 A5 JP2019524098 A5 JP 2019524098A5
- Authority
- JP
- Japan
- Prior art keywords
- nuclease
- target sequence
- dna sequence
- cells
- sequence
- 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
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- 101710163270 Nuclease Proteins 0.000 claims 26
- 238000000034 method Methods 0.000 claims 20
- 108091033409 CRISPR Proteins 0.000 claims 9
- 210000004027 cell Anatomy 0.000 claims 9
- 108091028043 Nucleic acid sequence Proteins 0.000 claims 8
- 108090000623 proteins and genes Proteins 0.000 claims 8
- 238000010354 CRISPR gene editing Methods 0.000 claims 6
- 108091034117 Oligonucleotide Proteins 0.000 claims 6
- 230000000295 complement effect Effects 0.000 claims 6
- 239000012634 fragment Substances 0.000 claims 6
- 230000008685 targeting Effects 0.000 claims 6
- 108091029865 Exogenous DNA Proteins 0.000 claims 4
- 239000013603 viral vector Substances 0.000 claims 4
- 101150074775 Csf1 gene Proteins 0.000 claims 3
- 108020004414 DNA Proteins 0.000 claims 3
- 238000010459 TALEN Methods 0.000 claims 3
- 108010043645 Transcription Activator-Like Effector Nucleases Proteins 0.000 claims 3
- 108010017070 Zinc Finger Nucleases Proteins 0.000 claims 3
- 239000002773 nucleotide Substances 0.000 claims 3
- 125000003729 nucleotide group Chemical group 0.000 claims 3
- 241000702421 Dependoparvovirus Species 0.000 claims 2
- 208000026350 Inborn Genetic disease Diseases 0.000 claims 2
- 241000713666 Lentivirus Species 0.000 claims 2
- 208000016361 genetic disease Diseases 0.000 claims 2
- -1 meganuclease Proteins 0.000 claims 2
- 230000035772 mutation Effects 0.000 claims 2
- 108091033319 polynucleotide Proteins 0.000 claims 2
- 239000002157 polynucleotide Substances 0.000 claims 2
- 102000040430 polynucleotide Human genes 0.000 claims 2
- 241000701161 unidentified adenovirus Species 0.000 claims 2
- 241001430294 unidentified retrovirus Species 0.000 claims 2
- 230000003612 virological effect Effects 0.000 claims 2
- 210000002237 B-cell of pancreatic islet Anatomy 0.000 claims 1
- 210000001789 adipocyte Anatomy 0.000 claims 1
- 210000003651 basophil Anatomy 0.000 claims 1
- 210000001772 blood platelet Anatomy 0.000 claims 1
- 210000004413 cardiac myocyte Anatomy 0.000 claims 1
- 210000002889 endothelial cell Anatomy 0.000 claims 1
- 210000003979 eosinophil Anatomy 0.000 claims 1
- 210000002919 epithelial cell Anatomy 0.000 claims 1
- 210000003494 hepatocyte Anatomy 0.000 claims 1
- 210000004698 lymphocyte Anatomy 0.000 claims 1
- 210000001616 monocyte Anatomy 0.000 claims 1
- 210000000440 neutrophil Anatomy 0.000 claims 1
- 210000000287 oocyte Anatomy 0.000 claims 1
- 210000004409 osteocyte Anatomy 0.000 claims 1
- 210000001116 retinal neuron Anatomy 0.000 claims 1
- 210000002363 skeletal muscle cell Anatomy 0.000 claims 1
- 210000000329 smooth muscle myocyte Anatomy 0.000 claims 1
- 210000000130 stem cell Anatomy 0.000 claims 1
Priority Applications (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2022159696A JP7493563B2 (ja) | 2016-07-15 | 2022-10-03 | 分裂細胞または非分裂細胞のゲノム編集のための方法と組成物 |
| JP2024082104A JP2024119833A (ja) | 2016-07-15 | 2024-05-20 | 非分裂細胞のゲノム編集のための方法と組成物 |
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201662363164P | 2016-07-15 | 2016-07-15 | |
| US62/363,164 | 2016-07-15 | ||
| PCT/US2017/042151 WO2018013932A1 (en) | 2016-07-15 | 2017-07-14 | Methods and compositions for genome editing in non-dividing cells |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2022159696A Division JP7493563B2 (ja) | 2016-07-15 | 2022-10-03 | 分裂細胞または非分裂細胞のゲノム編集のための方法と組成物 |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2019524098A JP2019524098A (ja) | 2019-09-05 |
| JP2019524098A5 true JP2019524098A5 (enExample) | 2020-08-20 |
Family
ID=59564227
Family Applications (3)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2019501985A Pending JP2019524098A (ja) | 2016-07-15 | 2017-07-14 | 非分裂細胞のゲノム編集のための方法と組成物 |
| JP2022159696A Active JP7493563B2 (ja) | 2016-07-15 | 2022-10-03 | 分裂細胞または非分裂細胞のゲノム編集のための方法と組成物 |
| JP2024082104A Pending JP2024119833A (ja) | 2016-07-15 | 2024-05-20 | 非分裂細胞のゲノム編集のための方法と組成物 |
Family Applications After (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2022159696A Active JP7493563B2 (ja) | 2016-07-15 | 2022-10-03 | 分裂細胞または非分裂細胞のゲノム編集のための方法と組成物 |
| JP2024082104A Pending JP2024119833A (ja) | 2016-07-15 | 2024-05-20 | 非分裂細胞のゲノム編集のための方法と組成物 |
Country Status (6)
| Country | Link |
|---|---|
| US (3) | US11674158B2 (enExample) |
| EP (2) | EP3485023B1 (enExample) |
| JP (3) | JP2019524098A (enExample) |
| AU (1) | AU2017295898B2 (enExample) |
| CA (1) | CA3030587A1 (enExample) |
| WO (1) | WO2018013932A1 (enExample) |
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| US9828582B2 (en) | 2013-03-19 | 2017-11-28 | Duke University | Compositions and methods for the induction and tuning of gene expression |
| US9340799B2 (en) | 2013-09-06 | 2016-05-17 | President And Fellows Of Harvard College | MRNA-sensing switchable gRNAs |
| US11053481B2 (en) | 2013-12-12 | 2021-07-06 | President And Fellows Of Harvard College | Fusions of Cas9 domains and nucleic acid-editing domains |
| EP3204496A1 (en) | 2014-10-10 | 2017-08-16 | Editas Medicine, Inc. | Compositions and methods for promoting homology directed repair |
| AU2015342749B2 (en) | 2014-11-07 | 2022-01-27 | Editas Medicine, Inc. | Methods for improving CRISPR/Cas-mediated genome-editing |
| EP3256487A4 (en) | 2015-02-09 | 2018-07-18 | Duke University | Compositions and methods for epigenome editing |
| CA2999500A1 (en) | 2015-09-24 | 2017-03-30 | Editas Medicine, Inc. | Use of exonucleases to improve crispr/cas-mediated genome editing |
| US12043852B2 (en) | 2015-10-23 | 2024-07-23 | President And Fellows Of Harvard College | Evolved Cas9 proteins for gene editing |
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| WO2017180915A2 (en) | 2016-04-13 | 2017-10-19 | Duke University | Crispr/cas9-based repressors for silencing gene targets in vivo and methods of use |
| EP4047092B1 (en) | 2016-04-13 | 2025-07-30 | Editas Medicine, Inc. | Cas9 fusion molecules, gene editing systems, and methods of use thereof |
| US11674158B2 (en) | 2016-07-15 | 2023-06-13 | Salk Institute For Biological Studies | Methods and compositions for genome editing in non-dividing cells |
| EP3487523B1 (en) | 2016-07-19 | 2023-09-06 | Duke University | Therapeutic applications of cpf1-based genome editing |
| 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 |
| WO2018119359A1 (en) | 2016-12-23 | 2018-06-28 | President And Fellows Of Harvard College | Editing of ccr5 receptor gene to protect against hiv infection |
| EP3592853A1 (en) | 2017-03-09 | 2020-01-15 | President and Fellows of Harvard College | Suppression of pain by gene editing |
| EP3592381A1 (en) | 2017-03-09 | 2020-01-15 | President and Fellows of Harvard College | Cancer vaccine |
| KR20190127797A (ko) | 2017-03-10 | 2019-11-13 | 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 | 시토신에서 구아닌으로의 염기 편집제 |
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| 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 |
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| 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 |
| CN111315889A (zh) * | 2017-09-08 | 2020-06-19 | 生命技术公司 | 提高同源重组的方法和其组合物 |
| 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 |
| US20210363546A1 (en) * | 2018-05-03 | 2021-11-25 | President And Fellows Of Harvard College | In vivo homology directed repair in heart, skeletal muscle, and muscle stem cells |
| WO2019226953A1 (en) | 2018-05-23 | 2019-11-28 | The Broad Institute, Inc. | Base editors and uses thereof |
| WO2019239361A1 (en) * | 2018-06-14 | 2019-12-19 | Novartis Ag | Method for sequence insertion using crispr |
| EP3836974A4 (en) | 2018-08-18 | 2022-06-15 | President and Fellows of Harvard College | IN SITU GENE EDIT |
| US20220175960A1 (en) * | 2018-08-24 | 2022-06-09 | Locanabio, Inc. | Fasl immunomodulatory gene therapy compositions and methods for use |
| WO2020051360A1 (en) * | 2018-09-05 | 2020-03-12 | The Broad Institute, Inc. | Base editing for treating hutchinson-gilford progeria syndrome |
| US12285495B2 (en) | 2018-10-15 | 2025-04-29 | Fondazione Telethon Ets | Genome editing methods and constructs |
| BR112021007301A2 (pt) | 2018-10-18 | 2021-07-27 | Intellia Therapeutics, Inc. | composições e métodos para expressar fator ix |
| US12281338B2 (en) | 2018-10-29 | 2025-04-22 | The Broad Institute, Inc. | Nucleobase editors comprising GeoCas9 and uses thereof |
| US20220000933A1 (en) * | 2018-10-30 | 2022-01-06 | Crispr Therapeutics Ag | Compositions and methods for nhej-mediated genome editing |
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| DE112020001306T5 (de) | 2019-03-19 | 2022-01-27 | Massachusetts Institute Of Technology | Verfahren und zusammensetzungen zur editierung von nukleotidsequenzen |
| EP3930766A4 (en) * | 2019-04-14 | 2023-02-22 | Duke University | CRISPR/CAS-BASED GENE EDITING COMPOSITION TO RESTORE DYSTROPHIN FUNCTION |
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| CA3152056A1 (en) * | 2019-08-22 | 2021-02-25 | Salk Institute For Biological Studies | Compositions and methods for in vivo gene editing |
| US12435330B2 (en) | 2019-10-10 | 2025-10-07 | The Broad Institute, Inc. | Methods and compositions for prime editing RNA |
| CN112741906B (zh) * | 2019-10-31 | 2022-07-05 | 华东师范大学 | 一种用于治疗b型血友病的产品 |
| CN113058041B (zh) * | 2020-08-27 | 2022-04-05 | 华东师范大学 | 一种用于治疗庞贝氏病的产品 |
| 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 |
| AU2021345112A1 (en) * | 2020-09-15 | 2023-04-27 | Research Institute At Nationwide Children's Hospital | Aav-mediated homology-independent targeted integration gene editing for correction of diverse dmd mutations in patients with muscular dystrophy |
| US20240425877A1 (en) * | 2021-05-07 | 2024-12-26 | Ucl Business Ltd | Abca4 genome editing |
| WO2022248645A1 (en) * | 2021-05-27 | 2022-12-01 | Astrazeneca Ab | Cas9 effector proteins with enhanced stability |
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| IL316555A (en) * | 2022-05-02 | 2024-12-01 | Fond Telethon Ets | Homology-independent directed integration for gene editing |
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| WO2025235862A1 (en) | 2024-05-10 | 2025-11-13 | Inndura Therapeutics Inc. | A modified immune cell receptor comprising a target-binding domain and the extracellular domain of cd16a |
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| US11674158B2 (en) | 2016-07-15 | 2023-06-13 | Salk Institute For Biological Studies | Methods and compositions for genome editing in non-dividing cells |
-
2017
- 2017-07-14 US US16/318,120 patent/US11674158B2/en active Active
- 2017-07-14 WO PCT/US2017/042151 patent/WO2018013932A1/en not_active Ceased
- 2017-07-14 CA CA3030587A patent/CA3030587A1/en active Pending
- 2017-07-14 EP EP17749553.8A patent/EP3485023B1/en active Active
- 2017-07-14 AU AU2017295898A patent/AU2017295898B2/en active Active
- 2017-07-14 JP JP2019501985A patent/JP2019524098A/ja active Pending
- 2017-07-14 EP EP23203168.2A patent/EP4321623A3/en active Pending
-
2022
- 2022-10-03 JP JP2022159696A patent/JP7493563B2/ja active Active
-
2023
- 2023-05-02 US US18/311,000 patent/US11959094B2/en active Active
-
2024
- 2024-03-14 US US18/604,927 patent/US20240409961A1/en not_active Abandoned
- 2024-05-20 JP JP2024082104A patent/JP2024119833A/ja active Pending
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