JP2018518182A5 - - Google Patents
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- JP2018518182A5 JP2018518182A5 JP2017565277A JP2017565277A JP2018518182A5 JP 2018518182 A5 JP2018518182 A5 JP 2018518182A5 JP 2017565277 A JP2017565277 A JP 2017565277A JP 2017565277 A JP2017565277 A JP 2017565277A JP 2018518182 A5 JP2018518182 A5 JP 2018518182A5
- Authority
- JP
- Japan
- Prior art keywords
- composition
- cell
- complex
- cells
- target dna
- 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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- 210000004027 cell Anatomy 0.000 claims description 77
- 230000035772 mutation Effects 0.000 claims description 47
- 102000004169 proteins and genes Human genes 0.000 claims description 46
- 108090000623 proteins and genes Proteins 0.000 claims description 46
- 238000000034 method Methods 0.000 claims description 45
- 101710163270 Nuclease Proteins 0.000 claims description 44
- 108020004414 DNA Proteins 0.000 claims description 42
- 108020005004 Guide RNA Proteins 0.000 claims description 28
- 208000037913 T-cell disorder Diseases 0.000 claims description 26
- 239000002773 nucleotide Substances 0.000 claims description 22
- 125000003729 nucleotide group Chemical group 0.000 claims description 22
- 108010043121 Green Fluorescent Proteins Proteins 0.000 claims description 18
- 102000004144 Green Fluorescent Proteins Human genes 0.000 claims description 18
- 239000005090 green fluorescent protein Substances 0.000 claims description 18
- 102000001554 Hemoglobins Human genes 0.000 claims description 15
- 108010054147 Hemoglobins Proteins 0.000 claims description 15
- 108090000765 processed proteins & peptides Proteins 0.000 claims description 13
- 210000004899 c-terminal region Anatomy 0.000 claims description 12
- 125000002924 primary amino group Chemical group [H]N([H])* 0.000 claims description 10
- 238000005215 recombination Methods 0.000 claims description 9
- 230000006798 recombination Effects 0.000 claims description 9
- 108091033409 CRISPR Proteins 0.000 claims description 8
- 230000005782 double-strand break Effects 0.000 claims description 8
- 230000006780 non-homologous end joining Effects 0.000 claims description 8
- 230000023603 positive regulation of transcription initiation, DNA-dependent Effects 0.000 claims description 8
- 239000012190 activator Substances 0.000 claims description 7
- 238000013518 transcription Methods 0.000 claims description 7
- 230000035897 transcription Effects 0.000 claims description 7
- 108091034117 Oligonucleotide Proteins 0.000 claims description 6
- 230000004570 RNA-binding Effects 0.000 claims description 6
- 210000003958 hematopoietic stem cell Anatomy 0.000 claims description 6
- 210000004263 induced pluripotent stem cell Anatomy 0.000 claims description 6
- 210000001744 T-lymphocyte Anatomy 0.000 claims description 5
- 201000010099 disease Diseases 0.000 claims description 5
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 claims description 5
- 230000027455 binding Effects 0.000 claims description 4
- 238000012986 modification Methods 0.000 claims description 4
- 230000004048 modification Effects 0.000 claims description 4
- 210000001778 pluripotent stem cell Anatomy 0.000 claims description 4
- 150000001413 amino acids Chemical class 0.000 claims description 3
- 210000003527 eukaryotic cell Anatomy 0.000 claims description 3
- 208000007056 sickle cell anemia Diseases 0.000 claims description 3
- 208000018020 Sickle cell-beta-thalassemia disease syndrome Diseases 0.000 claims description 2
- 206010043391 Thalassaemia beta Diseases 0.000 claims description 2
- 230000003833 cell viability Effects 0.000 claims description 2
- 238000011161 development Methods 0.000 claims description 2
- 230000004069 differentiation Effects 0.000 claims description 2
- 238000010348 incorporation Methods 0.000 claims description 2
- 239000000203 mixture Substances 0.000 claims 37
- 239000002131 composite material Substances 0.000 claims 1
- 238000012937 correction Methods 0.000 claims 1
- 231100000433 cytotoxic Toxicity 0.000 claims 1
- 230000001472 cytotoxic effect Effects 0.000 claims 1
- 108091006106 transcriptional activators Proteins 0.000 claims 1
- 210000000130 stem cell Anatomy 0.000 description 3
- 108010061697 INF7 peptide Proteins 0.000 description 2
- 208000005980 beta thalassemia Diseases 0.000 description 1
- 230000005779 cell damage Effects 0.000 description 1
- 208000037887 cell injury Diseases 0.000 description 1
- 238000012258 culturing Methods 0.000 description 1
- 210000004602 germ cell Anatomy 0.000 description 1
- 210000002980 germ line cell Anatomy 0.000 description 1
- 210000005260 human cell Anatomy 0.000 description 1
- 238000000338 in vitro Methods 0.000 description 1
- 238000001727 in vivo Methods 0.000 description 1
- 239000002243 precursor Substances 0.000 description 1
- 230000001737 promoting effect Effects 0.000 description 1
- 208000002491 severe combined immunodeficiency Diseases 0.000 description 1
Applications Claiming Priority (5)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201562181138P | 2015-06-17 | 2015-06-17 | |
| US62/181,138 | 2015-06-17 | ||
| US201562266316P | 2015-12-11 | 2015-12-11 | |
| US62/266,316 | 2015-12-11 | ||
| PCT/US2016/038161 WO2016205703A1 (en) | 2015-06-17 | 2016-06-17 | Crispr/cas9 complex for genomic editing |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2018518182A JP2018518182A (ja) | 2018-07-12 |
| JP2018518182A5 true JP2018518182A5 (enExample) | 2019-07-18 |
Family
ID=57546388
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2017565277A Pending JP2018518182A (ja) | 2015-06-17 | 2016-06-17 | ゲノム編集のためのｃｒｉｓｐｒ／ｃａｓ９複合体 |
Country Status (8)
| Country | Link |
|---|---|
| US (2) | US11643668B2 (enExample) |
| EP (1) | EP3310932B1 (enExample) |
| JP (1) | JP2018518182A (enExample) |
| CN (1) | CN107949641A (enExample) |
| AU (1) | AU2016278982A1 (enExample) |
| CA (1) | CA2989831A1 (enExample) |
| WO (1) | WO2016205703A1 (enExample) |
| ZA (1) | ZA201800026B (enExample) |
Families Citing this family (50)
| 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 |
| 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 |
| 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 |
| EP4079847A1 (en) | 2014-07-30 | 2022-10-26 | President And Fellows Of Harvard College | Cas9 proteins including ligand-dependent inteins |
| JP7030522B2 (ja) | 2015-05-11 | 2022-03-07 | エディタス・メディシン、インコーポレイテッド | 幹細胞における遺伝子編集のための最適化ｃｒｉｓｐｒ／ｃａｓ９システムおよび方法 |
| JP7396783B2 (ja) | 2015-06-09 | 2023-12-12 | エディタス・メディシン、インコーポレイテッド | 移植を改善するためのｃｒｉｓｐｒ／ｃａｓ関連方法および組成物 |
| WO2017053729A1 (en) | 2015-09-25 | 2017-03-30 | The Board Of Trustees Of The Leland Stanford Junior University | Nuclease-mediated genome editing of primary cells and enrichment thereof |
| IL294014B2 (en) | 2015-10-23 | 2024-07-01 | Harvard College | Nucleobase editors and uses thereof |
| WO2018027078A1 (en) | 2016-08-03 | 2018-02-08 | President And Fellows Of Harard College | Adenosine nucleobase editors and uses thereof |
| US11661590B2 (en) | 2016-08-09 | 2023-05-30 | 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 |
| SG11201903089RA (en) | 2016-10-14 | 2019-05-30 | Harvard College | Aav delivery of nucleobase editors |
| US10745677B2 (en) | 2016-12-23 | 2020-08-18 | President And Fellows Of Harvard College | Editing of CCR5 receptor gene to protect against HIV infection |
| TW201839136A (zh) | 2017-02-06 | 2018-11-01 | 瑞士商諾華公司 | 治療血色素異常症之組合物及方法 |
| 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 |
| 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 |
| GB2575930A (en) | 2017-03-23 | 2020-01-29 | Harvard College | Nucleobase editors comprising nucleic acid programmable DNA binding proteins |
| WO2018182361A1 (ko) * | 2017-03-31 | 2018-10-04 | 한국과학기술원 | CRISPR/Cas 시스템과 재조합 효소 및 단일가닥 올리고디옥시리보핵산을 이용한 코리네박테리움 변이균주 제조방법 |
| 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 |
| AU2018283405B2 (en) | 2017-06-15 | 2025-06-26 | The Regents Of The University Of California | Targeted non-viral DNA insertions |
| WO2019014564A1 (en) | 2017-07-14 | 2019-01-17 | Editas Medicine, Inc. | SYSTEMS AND METHODS OF TARGETED INTEGRATION AND GENOME EDITING AND DETECTION THEREOF WITH INTEGRATED PRIMING SITES |
| US11732274B2 (en) | 2017-07-28 | 2023-08-22 | 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 |
| WO2019079347A1 (en) | 2017-10-16 | 2019-04-25 | The Broad Institute, Inc. | USES OF BASIC EDITORS ADENOSINE |
| EP3700924A4 (en) | 2017-10-27 | 2021-11-03 | The Regents of the University of California | TARGETED REPLACEMENT OF ENDOGENOUS T-CELL RECEPTORS |
| WO2019118949A1 (en) | 2017-12-15 | 2019-06-20 | The Broad Institute, Inc. | Systems and methods for predicting repair outcomes in genetic engineering |
| WO2019226953A1 (en) | 2018-05-23 | 2019-11-28 | The Broad Institute, Inc. | Base editors and uses thereof |
| CA3096118A1 (en) | 2018-05-29 | 2019-12-05 | Monsanto Technology Llc | Transgenic plants with enhanced traits |
| CN108753778B (zh) * | 2018-06-01 | 2021-11-02 | 上海科技大学 | 利用碱基编辑修复fbn1t7498c突变的试剂和方法 |
| WO2020092453A1 (en) | 2018-10-29 | 2020-05-07 | The Broad Institute, Inc. | Nucleobase editors comprising geocas9 and uses thereof |
| US20220010305A1 (en) * | 2018-10-31 | 2022-01-13 | Novozymes A/S | Genome Editing by Guided Endonuclease and Single-stranded Oligonucleotide |
| US12351837B2 (en) | 2019-01-23 | 2025-07-08 | The Broad Institute, Inc. | Supernegatively charged proteins and uses thereof |
| DE112020001342T5 (de) | 2019-03-19 | 2022-01-13 | President and Fellows of Harvard College | Verfahren und Zusammensetzungen zum Editing von Nukleotidsequenzen |
| RU2707542C1 (ru) | 2019-03-28 | 2019-11-27 | Федеральное бюджетное учреждение науки "Центральный научно-исследовательский институт эпидемиологии" Федеральной службы по надзору в сфере защиты прав потребителей и благополучия человека (ФБУН ЦНИИ Эпидемиологии Роспотребнадзора) | Способ получения препарата рекомбинантной нуклеазы CAS, по существу, свободного от бактериальных эндотоксинов, полученный данным способом препарат и содержащий его набор для использования в системе CRISPR/Cas |
| WO2020214842A1 (en) | 2019-04-17 | 2020-10-22 | The Broad Institute, Inc. | Adenine base editors with reduced off-target effects |
| EP3958909A4 (en) * | 2019-04-24 | 2024-01-10 | Magenta Therapeutics, Inc. | Anti-cd117 antibody-drug conjugates and uses thereof |
| CN110283838B (zh) * | 2019-06-27 | 2022-03-25 | 安徽省农业科学院水稻研究所 | 一种高剪切效率ScCas9基因及其应用 |
| CN112442516A (zh) * | 2019-08-29 | 2021-03-05 | 广州辑因医疗科技有限公司 | 一种高效修复环状铁粒幼细胞性贫血基因突变的方法 |
| WO2021037234A1 (zh) * | 2019-08-29 | 2021-03-04 | 广州辑因医疗科技有限公司 | 一种高效修复环状铁粒幼细胞性贫血基因突变的方法 |
| WO2021072328A1 (en) | 2019-10-10 | 2021-04-15 | The Broad Institute, Inc. | Methods and compositions for prime editing rna |
| GB2614813B (en) | 2020-05-08 | 2025-05-07 | Harvard College | Methods and compositions for simultaneous editing of both strands of a target double-stranded nucleotide sequence |
| KR102421847B1 (ko) * | 2020-11-03 | 2022-07-18 | 한국생명공학연구원 | Jak3 유전자 돌연변이 중증복합면역결핍 동물모델 및 이의 제조방법 |
| CR20240195A (es) | 2021-10-14 | 2024-06-20 | Arsenal Biosciences Inc | Células inmunitarias que tienen arnhc coexpresados y sistemas de compuerta lógica |
| WO2024011176A2 (en) * | 2022-07-08 | 2024-01-11 | The Regents Of The University Of California | Modified crispr-cas effector polypeptides and methods of use thereof |
| CN117801056A (zh) * | 2023-06-29 | 2024-04-02 | 北京百普赛斯生物科技股份有限公司 | 一种从溶液中纯化目标蛋白的方法 |
Family Cites Families (22)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| JP2012525146A (ja) | 2009-04-28 | 2012-10-22 | プレジデント アンド フェロウズ オブ ハーバード カレッジ | 細胞透過のための過剰に荷電されたタンパク質 |
| RU2018109507A (ru) * | 2012-02-24 | 2019-02-28 | Фред Хатчинсон Кэнсер Рисерч Сентер | Композиции и способы лечения гемоглобинопатии |
| WO2013176772A1 (en) | 2012-05-25 | 2013-11-28 | The Regents Of The University Of California | Methods and compositions for rna-directed target dna modification and for rna-directed modulation of transcription |
| WO2014059173A2 (en) * | 2012-10-10 | 2014-04-17 | Sangamo Biosciences, Inc. | T cell modifying compounds and uses thereof |
| US8697359B1 (en) | 2012-12-12 | 2014-04-15 | The Broad Institute, Inc. | CRISPR-Cas systems and methods for altering expression of gene products |
| PL2931898T3 (pl) | 2012-12-12 | 2016-09-30 | Le Cong | Projektowanie i optymalizacja systemów, sposoby i kompozycje do manipulacji sekwencją z domenami funkcjonalnymi |
| US20140310830A1 (en) | 2012-12-12 | 2014-10-16 | Feng Zhang | CRISPR-Cas Nickase Systems, Methods And Compositions For Sequence Manipulation in Eukaryotes |
| IL307735A (en) | 2012-12-12 | 2023-12-01 | Broad Inst Inc | Systems engineering, methods and optimal guiding components for sequence manipulation |
| CN105209621B (zh) | 2012-12-12 | 2021-05-25 | 布罗德研究所有限公司 | 对用于序列操纵的改进的系统、方法和酶组合物进行的工程化和优化 |
| EP4234696A3 (en) | 2012-12-12 | 2023-09-06 | The Broad Institute Inc. | Crispr-cas component systems, methods and compositions for sequence manipulation |
| WO2014165707A2 (en) | 2013-04-03 | 2014-10-09 | Memorial Sloan-Kettering Cancer Center | Effective generation of tumor-targeted t-cells derived from pluripotent stem cells |
| WO2014165825A2 (en) | 2013-04-04 | 2014-10-09 | President And Fellows Of Harvard College | Therapeutic uses of genome editing with crispr/cas systems |
| WO2014191128A1 (en) | 2013-05-29 | 2014-12-04 | Cellectis | Methods for engineering t cells for immunotherapy by using rna-guided cas nuclease system |
| WO2015006498A2 (en) | 2013-07-09 | 2015-01-15 | President And Fellows Of Harvard College | Therapeutic uses of genome editing with crispr/cas systems |
| US9526784B2 (en) | 2013-09-06 | 2016-12-27 | President And Fellows Of Harvard College | Delivery system for functional nucleases |
| US12180263B2 (en) | 2014-11-06 | 2024-12-31 | President And Fellows Of Harvard College | Cells lacking B2M surface expression and methods for allogeneic administration of such cells |
| WO2016094880A1 (en) | 2014-12-12 | 2016-06-16 | The Broad Institute Inc. | Delivery, use and therapeutic applications of crispr systems and compositions for genome editing as to hematopoietic stem cells (hscs) |
| WO2016123578A1 (en) | 2015-01-30 | 2016-08-04 | The Regents Of The University Of California | Protein delivery in primary hematopoietic cells |
| CN107847524A (zh) | 2015-03-27 | 2018-03-27 | 哈佛学院校长同事会 | 经过修饰的t细胞及其制备和使用方法 |
| CN104894068A (zh) | 2015-05-04 | 2015-09-09 | 南京凯地生物科技有限公司 | 一种利用CRISPR/Cas9制备CAR-T细胞的方法 |
| JP2018518181A (ja) | 2015-06-17 | 2018-07-12 | ザ ユーエービー リサーチ ファンデーション | 血液細胞系列の細胞に機能的ポリペプチドを導入するためのＣＲＩＳＰＲ／Ｃａｓ９複合体 |
| EP3443075B1 (en) | 2016-04-15 | 2022-09-28 | Memorial Sloan Kettering Cancer Center | Transgenic t cell and chimeric antigen receptor t cell compositions and related methods |
-
2016
- 2016-06-17 CA CA2989831A patent/CA2989831A1/en not_active Abandoned
- 2016-06-17 JP JP2017565277A patent/JP2018518182A/ja active Pending
- 2016-06-17 EP EP16812552.4A patent/EP3310932B1/en active Active
- 2016-06-17 US US15/737,132 patent/US11643668B2/en active Active
- 2016-06-17 CN CN201680048445.4A patent/CN107949641A/zh active Pending
- 2016-06-17 WO PCT/US2016/038161 patent/WO2016205703A1/en not_active Ceased
- 2016-06-17 AU AU2016278982A patent/AU2016278982A1/en not_active Abandoned
-
2018
- 2018-01-03 ZA ZA2018/00026A patent/ZA201800026B/en unknown
-
2023
- 2023-04-04 US US18/295,387 patent/US20240117380A1/en active Pending
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