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 (OSRAM) | 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 (OSRAM) |
| EP (2) | EP4321623A3 (OSRAM) |
| JP (3) | JP2019524098A (OSRAM) |
| AU (1) | AU2017295898B2 (OSRAM) |
| CA (1) | CA3030587A1 (OSRAM) |
| WO (1) | WO2018013932A1 (OSRAM) |
Families Citing this family (59)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US9828582B2 (en) | 2013-03-19 | 2017-11-28 | Duke University | Compositions and methods for the induction and tuning of gene expression |
| US9340800B2 (en) | 2013-09-06 | 2016-05-17 | President And Fellows Of Harvard College | Extended DNA-sensing GRNAS |
| US9840699B2 (en) | 2013-12-12 | 2017-12-12 | President And Fellows Of Harvard College | Methods for nucleic acid editing |
| AU2015330699B2 (en) | 2014-10-10 | 2021-12-02 | Editas Medicine, Inc. | Compositions and methods for promoting homology directed repair |
| EP4464338A3 (en) | 2014-11-07 | 2025-02-12 | Editas Medicine, Inc. | Systems for improving crispr/cas-mediated genome-editing |
| WO2016130600A2 (en) | 2015-02-09 | 2016-08-18 | Duke University | Compositions and methods for epigenome editing |
| AU2016326711B2 (en) | 2015-09-24 | 2022-11-03 | Editas Medicine, Inc. | Use of exonucleases to improve CRISPR/Cas-mediated genome editing |
| IL258821B (en) | 2015-10-23 | 2022-07-01 | Harvard College | Nucleobase editors and their uses |
| AU2016362282B2 (en) | 2015-11-30 | 2023-03-16 | Duke University | Therapeutic targets for the correction of the human dystrophin gene by gene editing and methods of use |
| WO2017165826A1 (en) | 2016-03-25 | 2017-09-28 | Editas Medicine, Inc. | Genome editing systems comprising repair-modulating enzyme molecules and methods of their use |
| EP4047092B1 (en) | 2016-04-13 | 2025-07-30 | Editas Medicine, Inc. | Cas9 fusion molecules, gene editing systems, and methods of use thereof |
| US20190127713A1 (en) | 2016-04-13 | 2019-05-02 | Duke University | Crispr/cas9-based repressors for silencing gene targets in vivo and methods of use |
| EP4321623A3 (en) | 2016-07-15 | 2024-05-15 | Salk Institute for Biological Studies | Methods and compositions for genome editing in non-dividing cells |
| EP4275747A3 (en) | 2016-07-19 | 2024-01-24 | 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 |
| CA3033327A1 (en) | 2016-08-09 | 2018-02-15 | 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 |
| JP7588390B2 (ja) | 2016-10-14 | 2024-11-22 | 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 | 核酸塩基エディターのａａｖ送達 |
| WO2018119359A1 (en) | 2016-12-23 | 2018-06-28 | President And Fellows Of Harvard College | Editing of ccr5 receptor gene to protect against hiv infection |
| CN110662556A (zh) | 2017-03-09 | 2020-01-07 | 哈佛大学的校长及成员们 | 癌症疫苗 |
| WO2018165504A1 (en) | 2017-03-09 | 2018-09-13 | President And Fellows Of Harvard College | Suppression of pain by gene editing |
| JP2020510439A (ja) | 2017-03-10 | 2020-04-09 | 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 | シトシンからグアニンへの塩基編集因子 |
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| EP3652312A1 (en) | 2017-07-14 | 2020-05-20 | Editas Medicine, Inc. | Systems and methods for targeted integration and genome editing and detection thereof using integrated priming sites |
| JP2020534795A (ja) | 2017-07-28 | 2020-12-03 | 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 | ファージによって支援される連続的進化（ｐａｃｅ）を用いて塩基編集因子を進化させるための方法および組成物 |
| US11319532B2 (en) | 2017-08-30 | 2022-05-03 | President And Fellows Of Harvard College | High efficiency base editors comprising Gam |
| US20190119701A1 (en) * | 2017-09-08 | 2019-04-25 | Life Technologies Corporation | Methods for improved homologous recombination and compositions thereof |
| KR20250107288A (ko) | 2017-10-16 | 2025-07-11 | 더 브로드 인스티튜트, 인코퍼레이티드 | 아데노신 염기 편집제의 용도 |
| WO2019118949A1 (en) | 2017-12-15 | 2019-06-20 | The Broad Institute, Inc. | Systems and methods for predicting repair outcomes in genetic engineering |
| EP3787692A4 (en) * | 2018-05-03 | 2022-03-23 | 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 |
| US12486519B2 (en) | 2018-08-18 | 2025-12-02 | President And Fellows Of Harvard College | In situ gene editing |
| AU2019326617A1 (en) * | 2018-08-24 | 2021-03-18 | Locanabio, Inc. | FASL immunomodulatory gene therapy compositions and methods for use |
| US20240173430A1 (en) * | 2018-09-05 | 2024-05-30 | The Broad Institute, Inc. | Base editing for treating hutchinson-gilford progeria syndrome |
| WO2020079033A1 (en) * | 2018-10-15 | 2020-04-23 | Fondazione Telethon | Genome editing methods and constructs |
| EP3867380A2 (en) | 2018-10-18 | 2021-08-25 | Intellia Therapeutics, Inc. | Compositions and methods for expressing factor ix |
| WO2020092453A1 (en) | 2018-10-29 | 2020-05-07 | The Broad Institute, Inc. | Nucleobase editors comprising geocas9 and uses thereof |
| WO2020092611A1 (en) * | 2018-10-30 | 2020-05-07 | Casebia Therapeutics Limited Liability Partnership | Compositions and methods for nhej-mediated genome editing |
| US12351837B2 (en) | 2019-01-23 | 2025-07-08 | The Broad Institute, Inc. | Supernegatively charged proteins and uses thereof |
| US12270033B2 (en) | 2019-02-15 | 2025-04-08 | Exhaura, Ltd. | Dual leucine zipper kinase inhibitors for gene therapy |
| EP3942042A1 (en) | 2019-03-19 | 2022-01-26 | The Broad Institute, Inc. | Methods and compositions for editing nucleotide sequences |
| WO2020214613A1 (en) * | 2019-04-14 | 2020-10-22 | Duke University | Crispr/cas-based genome editing composition for restoring dystrophin function |
| EP3956349A1 (en) | 2019-04-17 | 2022-02-23 | The Broad Institute, Inc. | Adenine base editors with reduced off-target effects |
| MX2021015122A (es) | 2019-06-07 | 2022-04-06 | Regeneron Pharma | Animales no humanos que comprenden un locus de albumina humanizado. |
| CN114144203A (zh) | 2019-07-12 | 2022-03-04 | 国立研究开发法人理化学研究所 | 源于显性变异基因的疾患的治疗剂 |
| WO2021034336A1 (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 |
| 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 |
| 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 |
| WO2021226558A1 (en) | 2020-05-08 | 2021-11-11 | The Broad Institute, Inc. | Methods and compositions for simultaneous editing of both strands of a target double-stranded nucleotide sequence |
| CA3195233A1 (en) * | 2020-09-15 | 2022-03-24 | 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 |
| WO2022234295A1 (en) * | 2021-05-07 | 2022-11-10 | Ucl Business Ltd | Abca4 genome editing |
| WO2022248645A1 (en) * | 2021-05-27 | 2022-12-01 | Astrazeneca Ab | Cas9 effector proteins with enhanced stability |
| US20250177573A1 (en) | 2021-11-19 | 2025-06-05 | The University Of Bristol | Materials & Methods for Treatment of Macular Degeneration |
| JP2025515030A (ja) * | 2022-05-02 | 2025-05-13 | フォンダジオン テレソン イーティーエス | 遺伝子編集用の相同性非依存性標的組込み |
| WO2024102954A1 (en) | 2022-11-10 | 2024-05-16 | Massachusetts Institute Of Technology | Activation induced clipping system (aics) |
| 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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| EP4321623A3 (en) | 2016-07-15 | 2024-05-15 | Salk Institute for Biological Studies | Methods and compositions for genome editing in non-dividing cells |
-
2017
- 2017-07-14 EP EP23203168.2A patent/EP4321623A3/en active Pending
- 2017-07-14 EP EP17749553.8A patent/EP3485023B1/en active Active
- 2017-07-14 JP JP2019501985A patent/JP2019524098A/ja active Pending
- 2017-07-14 AU AU2017295898A patent/AU2017295898B2/en active Active
- 2017-07-14 CA CA3030587A patent/CA3030587A1/en active Pending
- 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
-
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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