JP2018518181A5 - - Google Patents
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- JP2018518181A5 JP2018518181A5 JP2017565262A JP2017565262A JP2018518181A5 JP 2018518181 A5 JP2018518181 A5 JP 2018518181A5 JP 2017565262 A JP2017565262 A JP 2017565262A JP 2017565262 A JP2017565262 A JP 2017565262A JP 2018518181 A5 JP2018518181 A5 JP 2018518181A5
- Authority
- JP
- Japan
- Prior art keywords
- complex
- cell precursor
- nucleotide sequence
- supercharged protein
- specific nuclease
- 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
- 238000000034 method Methods 0.000 claims description 47
- 210000000173 T-lymphoid precursor cell Anatomy 0.000 claims description 36
- 101710163270 Nuclease Proteins 0.000 claims description 34
- 102000004169 proteins and genes Human genes 0.000 claims description 33
- 108090000623 proteins and genes Proteins 0.000 claims description 33
- 239000002773 nucleotide Substances 0.000 claims description 28
- 125000003729 nucleotide group Chemical group 0.000 claims description 28
- 108020004414 DNA Proteins 0.000 claims description 18
- 108010019670 Chimeric Antigen Receptors Proteins 0.000 claims description 17
- 108020005004 Guide RNA Proteins 0.000 claims description 14
- 108010043121 Green Fluorescent Proteins Proteins 0.000 claims description 12
- 102000004144 Green Fluorescent Proteins Human genes 0.000 claims description 12
- 239000005090 green fluorescent protein Substances 0.000 claims description 12
- 230000006798 recombination Effects 0.000 claims description 10
- 238000005215 recombination Methods 0.000 claims description 10
- 210000004027 cell Anatomy 0.000 claims description 9
- 206010028980 Neoplasm Diseases 0.000 claims description 8
- 125000002924 primary amino group Chemical group [H]N([H])* 0.000 claims description 8
- 108091028043 Nucleic acid sequence Proteins 0.000 claims description 5
- 230000004570 RNA-binding Effects 0.000 claims description 5
- 230000005782 double-strand break Effects 0.000 claims description 5
- 230000034431 double-strand break repair via homologous recombination Effects 0.000 claims description 5
- 150000007523 nucleic acids Chemical group 0.000 claims description 5
- 108090000765 processed proteins & peptides Proteins 0.000 claims description 5
- 108091033409 CRISPR Proteins 0.000 claims description 4
- 239000012190 activator Substances 0.000 claims description 4
- 230000027455 binding Effects 0.000 claims description 4
- 210000004899 c-terminal region Anatomy 0.000 claims description 4
- 238000012258 culturing Methods 0.000 claims description 4
- 210000001778 pluripotent stem cell Anatomy 0.000 claims description 4
- 230000023603 positive regulation of transcription initiation, DNA-dependent Effects 0.000 claims description 4
- 230000035897 transcription Effects 0.000 claims description 4
- 238000013518 transcription Methods 0.000 claims description 4
- 230000010354 integration Effects 0.000 claims description 3
- 238000002054 transplantation Methods 0.000 claims description 3
- 210000001744 T-lymphocyte Anatomy 0.000 claims description 2
- 201000011510 cancer Diseases 0.000 claims description 2
- 230000004069 differentiation Effects 0.000 claims description 2
- 210000003958 hematopoietic stem cell Anatomy 0.000 claims description 2
- 210000004263 induced pluripotent stem cell Anatomy 0.000 claims description 2
- 230000006780 non-homologous end joining Effects 0.000 claims description 2
- 101800004910 Nuclease A Proteins 0.000 description 1
- 210000004369 blood Anatomy 0.000 description 1
- 239000008280 blood Substances 0.000 description 1
- 230000003833 cell viability Effects 0.000 description 1
- 238000004519 manufacturing process Methods 0.000 description 1
- 229920001184 polypeptide Polymers 0.000 description 1
- 239000002243 precursor Substances 0.000 description 1
- 102000004196 processed proteins & peptides Human genes 0.000 description 1
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201562181145P | 2015-06-17 | 2015-06-17 | |
| US62/181,145 | 2015-06-17 | ||
| PCT/US2016/038125 WO2016205680A1 (en) | 2015-06-17 | 2016-06-17 | Crispr/cas9 complex for introducing a functional polypeptide into cells of blood cell lineage |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2018518181A JP2018518181A (ja) | 2018-07-12 |
| JP2018518181A5 true JP2018518181A5 (OSRAM) | 2019-07-18 |
Family
ID=57546413
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2017565262A Pending JP2018518181A (ja) | 2015-06-17 | 2016-06-17 | 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 |
Country Status (7)
| Country | Link |
|---|---|
| US (1) | US11555207B2 (OSRAM) |
| EP (1) | EP3310931B1 (OSRAM) |
| JP (1) | JP2018518181A (OSRAM) |
| CN (1) | CN108026545A (OSRAM) |
| AU (1) | AU2016278959A1 (OSRAM) |
| CA (1) | CA2989858A1 (OSRAM) |
| WO (1) | WO2016205680A1 (OSRAM) |
Families Citing this family (46)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| EP2734621B1 (en) | 2011-07-22 | 2019-09-04 | President and Fellows of Harvard College | Evaluation and improvement of nuclease cleavage specificity |
| US20150044192A1 (en) | 2013-08-09 | 2015-02-12 | 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 |
| US9388430B2 (en) | 2013-09-06 | 2016-07-12 | President And Fellows Of Harvard College | Cas9-recombinase fusion proteins and uses thereof |
| US9340800B2 (en) | 2013-09-06 | 2016-05-17 | President And Fellows Of Harvard College | Extended DNA-sensing GRNAS |
| US9526784B2 (en) | 2013-09-06 | 2016-12-27 | President And Fellows Of Harvard College | Delivery system for functional nucleases |
| US9840699B2 (en) | 2013-12-12 | 2017-12-12 | President And Fellows Of Harvard College | Methods for nucleic acid editing |
| WO2016022363A2 (en) | 2014-07-30 | 2016-02-11 | 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 |
| CN108026526B (zh) | 2015-06-09 | 2023-05-12 | 爱迪塔斯医药公司 | 用于改善移植的crispr/cas相关方法和组合物 |
| JP2018518182A (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 | ゲノム編集のためのcrispr/cas9複合体 |
| IL258821B (en) | 2015-10-23 | 2022-07-01 | Harvard College | Nucleobase editors and their uses |
| 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 |
| JP7092281B2 (ja) * | 2017-02-07 | 2022-06-28 | エージェンシー フォー サイエンス,テクノロジー アンド リサーチ | 多能性幹細胞から模倣自然免疫細胞を生成する方法及びキット |
| AU2018217404B2 (en) | 2017-02-13 | 2024-03-28 | Assistance Publique - Hopitaux De Paris | Method for generating T cells progenitors |
| WO2018165504A1 (en) | 2017-03-09 | 2018-09-13 | President And Fellows Of Harvard College | Suppression of pain by gene editing |
| CN110662556A (zh) | 2017-03-09 | 2020-01-07 | 哈佛大学的校长及成员们 | 癌症疫苗 |
| 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 |
| BR112019019655A2 (pt) | 2017-03-23 | 2020-04-22 | Harvard College | editores de nucleobase que compreendem proteínas de ligação a dna programáveis por ácido nucleico |
| 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 |
| US20200362355A1 (en) * | 2017-06-15 | 2020-11-19 | The Regents Of The University Of California | Targeted non-viral dna insertions |
| 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 |
| US20200246488A1 (en) * | 2017-07-17 | 2020-08-06 | The Broad Institute, Inc. | Compositions and methods for treating inflammatory bowel diseases |
| 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 |
| US11905513B2 (en) * | 2017-10-13 | 2024-02-20 | Mbp Titan, Llc | Advanced genome editing |
| KR20250107288A (ko) | 2017-10-16 | 2025-07-11 | 더 브로드 인스티튜트, 인코퍼레이티드 | 아데노신 염기 편집제의 용도 |
| IL274179B2 (en) | 2017-10-27 | 2024-02-01 | Univ 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 |
| US20210254049A1 (en) * | 2018-04-20 | 2021-08-19 | Cellino Biotech, Inc. | Directed cell fate specification and targeted maturation |
| WO2019226953A1 (en) | 2018-05-23 | 2019-11-28 | The Broad Institute, Inc. | Base editors and uses thereof |
| BR112021007229A2 (pt) | 2018-10-16 | 2021-08-10 | Blueallele, Llc | métodos para inserção dirigida de dna em genes |
| WO2020092453A1 (en) | 2018-10-29 | 2020-05-07 | The Broad Institute, Inc. | Nucleobase editors comprising geocas9 and uses thereof |
| EP3877748A4 (en) | 2018-11-06 | 2022-11-09 | Cellino Biotech, Inc. | Systems for cell control |
| US12351837B2 (en) | 2019-01-23 | 2025-07-08 | The Broad Institute, Inc. | Supernegatively charged proteins and uses thereof |
| EP3942042A1 (en) | 2019-03-19 | 2022-01-26 | The Broad Institute, Inc. | Methods and compositions for editing nucleotide sequences |
| EP3956349A1 (en) | 2019-04-17 | 2022-02-23 | The Broad Institute, Inc. | Adenine base editors with reduced off-target effects |
| US12435330B2 (en) | 2019-10-10 | 2025-10-07 | The Broad Institute, Inc. | Methods and compositions for prime editing RNA |
| 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 |
| WO2022147014A2 (en) * | 2020-12-28 | 2022-07-07 | The Regents Of The University Of California | Engineered gamma delta t cells and methods of making and using thereof |
| EP4416292A2 (en) | 2021-10-14 | 2024-08-21 | Arsenal Biosciences, Inc. | Immune cells having co-expressed shrnas and logic gate systems |
| 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 |
|---|---|---|---|---|
| US10059923B2 (en) * | 2008-01-30 | 2018-08-28 | Memorial Sloan Kettering Cancer Center | Methods for off-the-shelf tumor immunotherapy using allogeneic T-cell precursors |
| US9221886B2 (en) * | 2009-04-28 | 2015-12-29 | President And Fellows Of Harvard College | Supercharged proteins for cell penetration |
| SG10201606959PA (en) | 2012-02-24 | 2016-09-29 | Hutchinson Fred Cancer Res | Compositions and methods for the treatment of hemoglobinopathies |
| SI3401400T1 (sl) | 2012-05-25 | 2019-10-30 | Univ California | Postopki in sestavki za RNA usmerjeno modifikacijo tarčne DNA in za RNA usmerjeno modulacijo prepisovanja |
| EP2906684B8 (en) * | 2012-10-10 | 2020-09-02 | Sangamo Therapeutics, Inc. | T cell modifying compounds and uses thereof |
| CN113355357B (zh) | 2012-12-12 | 2024-12-03 | 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 |
| ES2536353T3 (es) | 2012-12-12 | 2015-05-22 | The Broad Institute, Inc. | Ingeniería de sistemas, métodos y composiciones de guía optimizadas para manipulación de secuencias |
| CN105658796B (zh) | 2012-12-12 | 2021-10-26 | 布罗德研究所有限公司 | 用于序列操纵的crispr-cas组分系统、方法以及组合物 |
| US8697359B1 (en) | 2012-12-12 | 2014-04-15 | The Broad Institute, Inc. | CRISPR-Cas systems and methods for altering expression of gene products |
| US20140310830A1 (en) | 2012-12-12 | 2014-10-16 | Feng Zhang | CRISPR-Cas Nickase Systems, Methods And Compositions For Sequence Manipulation in Eukaryotes |
| EP2931898B1 (en) | 2012-12-12 | 2016-03-09 | The Broad Institute, Inc. | Engineering and optimization of systems, methods and compositions for sequence manipulation with functional domains |
| 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 |
| JP2016522679A (ja) * | 2013-04-04 | 2016-08-04 | 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 | CRISPR/Cas系を用いたゲノム編集の治療的使用 |
| ES2645393T3 (es) | 2013-05-29 | 2017-12-05 | Cellectis | Métodos de manipulación de linfocitos T para inmunoterapia usando el sistema de nucleasa Cas guiada por ARN |
| JP2016528890A (ja) | 2013-07-09 | 2016-09-23 | 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 | CRISPR/Cas系を用いるゲノム編集の治療用の使用 |
| US9526784B2 (en) * | 2013-09-06 | 2016-12-27 | President And Fellows Of Harvard College | Delivery system for functional nucleases |
| EP3215623A4 (en) * | 2014-11-06 | 2018-09-26 | 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) |
| US20190388469A1 (en) | 2015-01-30 | 2019-12-26 | The Regents Of The University Of California | Protein delivery in primary hematopoietic cells |
| BR112017020750A2 (pt) * | 2015-03-27 | 2018-06-26 | Harvard College | células t modificadas e métodos de produção e utilização das mesmas |
| 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 |
| SG11201808831TA (en) | 2016-04-15 | 2018-11-29 | Memorial Sloan Kettering Cancer Center | Transgenic t cell and chimeric antigen receptor t cell compositions and related methods |
-
2016
- 2016-06-17 CA CA2989858A patent/CA2989858A1/en not_active Abandoned
- 2016-06-17 US US15/737,134 patent/US11555207B2/en active Active
- 2016-06-17 JP JP2017565262A patent/JP2018518181A/ja active Pending
- 2016-06-17 AU AU2016278959A patent/AU2016278959A1/en not_active Abandoned
- 2016-06-17 EP EP16812542.5A patent/EP3310931B1/en active Active
- 2016-06-17 WO PCT/US2016/038125 patent/WO2016205680A1/en not_active Ceased
- 2016-06-17 CN CN201680044599.6A patent/CN108026545A/zh active Pending
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