JP2016520319A5 - - Google Patents
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- JP2016520319A5 JP2016520319A5 JP2016516171A JP2016516171A JP2016520319A5 JP 2016520319 A5 JP2016520319 A5 JP 2016520319A5 JP 2016516171 A JP2016516171 A JP 2016516171A JP 2016516171 A JP2016516171 A JP 2016516171A JP 2016520319 A5 JP2016520319 A5 JP 2016520319A5
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- JP
- Japan
- Prior art keywords
- seq
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- variant
- cell
- onui
- 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.)
- Granted
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- 230000000694 effects Effects 0.000 claims description 24
- 108010042407 Endonucleases Proteins 0.000 claims description 22
- 102100031780 Endonuclease Human genes 0.000 claims description 21
- 238000000034 method Methods 0.000 claims description 21
- 210000004027 cell Anatomy 0.000 claims description 20
- 108020004414 DNA Proteins 0.000 claims description 14
- 102000004190 Enzymes Human genes 0.000 claims description 10
- 108090000790 Enzymes Proteins 0.000 claims description 10
- 108090000623 proteins and genes Proteins 0.000 claims description 10
- 150000007523 nucleic acids Chemical group 0.000 claims description 8
- 108020001580 protein domains Proteins 0.000 claims description 8
- 108091028043 Nucleic acid sequence Proteins 0.000 claims description 7
- 101150017501 CCR5 gene Proteins 0.000 claims description 6
- 230000003197 catalytic effect Effects 0.000 claims description 6
- 102000004169 proteins and genes Human genes 0.000 claims description 6
- 108091007494 Nucleic acid- binding domains Proteins 0.000 claims description 5
- 108060002716 Exonuclease Proteins 0.000 claims description 4
- 101000830950 Homo sapiens Three prime repair exonuclease 2 Proteins 0.000 claims description 4
- 241000725303 Human immunodeficiency virus Species 0.000 claims description 4
- 210000001744 T-lymphocyte Anatomy 0.000 claims description 4
- 102100024872 Three prime repair exonuclease 2 Human genes 0.000 claims description 4
- 102000013165 exonuclease Human genes 0.000 claims description 4
- 108091033319 polynucleotide Proteins 0.000 claims description 4
- 102000040430 polynucleotide Human genes 0.000 claims description 4
- 239000002157 polynucleotide Substances 0.000 claims description 4
- 108090000765 processed proteins & peptides Proteins 0.000 claims description 4
- 208000031886 HIV Infections Diseases 0.000 claims description 3
- 208000037357 HIV infectious disease Diseases 0.000 claims description 3
- 102100031573 Hematopoietic progenitor cell antigen CD34 Human genes 0.000 claims description 3
- 101000777663 Homo sapiens Hematopoietic progenitor cell antigen CD34 Proteins 0.000 claims description 3
- 101710163270 Nuclease Proteins 0.000 claims description 3
- 210000003958 hematopoietic stem cell Anatomy 0.000 claims description 3
- 208000033519 human immunodeficiency virus infectious disease Diseases 0.000 claims description 3
- 210000004698 lymphocyte Anatomy 0.000 claims description 3
- 210000000130 stem cell Anatomy 0.000 claims description 3
- FWMNVWWHGCHHJJ-SKKKGAJSSA-N 4-amino-1-[(2r)-6-amino-2-[[(2r)-2-[[(2r)-2-[[(2r)-2-amino-3-phenylpropanoyl]amino]-3-phenylpropanoyl]amino]-4-methylpentanoyl]amino]hexanoyl]piperidine-4-carboxylic acid Chemical compound C([C@H](C(=O)N[C@H](CC(C)C)C(=O)N[C@H](CCCCN)C(=O)N1CCC(N)(CC1)C(O)=O)NC(=O)[C@H](N)CC=1C=CC=CC=1)C1=CC=CC=C1 FWMNVWWHGCHHJJ-SKKKGAJSSA-N 0.000 claims description 2
- 102000004274 CCR5 Receptors Human genes 0.000 claims description 2
- 108010017088 CCR5 Receptors Proteins 0.000 claims description 2
- 102100034343 Integrase Human genes 0.000 claims description 2
- 108010061833 Integrases Proteins 0.000 claims description 2
- 102000003960 Ligases Human genes 0.000 claims description 2
- 108090000364 Ligases Proteins 0.000 claims description 2
- 108060004795 Methyltransferase Proteins 0.000 claims description 2
- 101100508818 Mus musculus Inpp5k gene Proteins 0.000 claims description 2
- 102000004160 Phosphoric Monoester Hydrolases Human genes 0.000 claims description 2
- 108090000608 Phosphoric Monoester Hydrolases Proteins 0.000 claims description 2
- 108091000080 Phosphotransferase Proteins 0.000 claims description 2
- 101100366438 Rattus norvegicus Sphkap gene Proteins 0.000 claims description 2
- 102000018120 Recombinases Human genes 0.000 claims description 2
- 108010091086 Recombinases Proteins 0.000 claims description 2
- 101710183280 Topoisomerase Proteins 0.000 claims description 2
- 108700019146 Transgenes Proteins 0.000 claims description 2
- 102000008579 Transposases Human genes 0.000 claims description 2
- 108010020764 Transposases Proteins 0.000 claims description 2
- HCHKCACWOHOZIP-UHFFFAOYSA-N Zinc Chemical compound [Zn] HCHKCACWOHOZIP-UHFFFAOYSA-N 0.000 claims description 2
- 125000003275 alpha amino acid group Chemical group 0.000 claims description 2
- 238000003776 cleavage reaction Methods 0.000 claims description 2
- 102000034287 fluorescent proteins Human genes 0.000 claims description 2
- 108091006047 fluorescent proteins Proteins 0.000 claims description 2
- 208000015181 infectious disease Diseases 0.000 claims description 2
- 230000003834 intracellular effect Effects 0.000 claims description 2
- 239000011159 matrix material Substances 0.000 claims description 2
- 238000002703 mutagenesis Methods 0.000 claims description 2
- 231100000350 mutagenesis Toxicity 0.000 claims description 2
- 108020004707 nucleic acids Proteins 0.000 claims description 2
- 102000039446 nucleic acids Human genes 0.000 claims description 2
- 102000020233 phosphotransferase Human genes 0.000 claims description 2
- 229920001184 polypeptide Polymers 0.000 claims description 2
- 102000004196 processed proteins & peptides Human genes 0.000 claims description 2
- 230000007017 scission Effects 0.000 claims description 2
- 238000006467 substitution reaction Methods 0.000 claims description 2
- 239000011701 zinc Substances 0.000 claims description 2
- 229910052725 zinc Inorganic materials 0.000 claims description 2
- 239000003814 drug Substances 0.000 claims 4
- 101710149870 C-C chemokine receptor type 5 Proteins 0.000 claims 1
- 230000002265 prevention Effects 0.000 claims 1
- 101150096316 5 gene Proteins 0.000 description 1
- 108091008927 CC chemokine receptors Proteins 0.000 description 1
- 102000005674 CCR Receptors Human genes 0.000 description 1
- 102000004533 Endonucleases Human genes 0.000 description 1
- 239000003153 chemical reaction reagent Substances 0.000 description 1
- 230000003013 cytotoxicity Effects 0.000 description 1
- 231100000135 cytotoxicity Toxicity 0.000 description 1
- 238000010362 genome editing Methods 0.000 description 1
- 210000005260 human cell Anatomy 0.000 description 1
- 230000002452 interceptive effect Effects 0.000 description 1
- 230000035772 mutation Effects 0.000 description 1
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| DKPA201370302 | 2013-05-31 | ||
| DKPA201370302 | 2013-05-31 | ||
| PCT/EP2014/061186 WO2014191525A1 (en) | 2013-05-31 | 2014-05-28 | A laglidadg homing endonuclease cleaving the c-c chemokine receptor type-5 (ccr5) gene and uses thereof |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2016520319A JP2016520319A (ja) | 2016-07-14 |
| JP2016520319A5 true JP2016520319A5 (enExample) | 2017-07-06 |
| JP6488283B2 JP6488283B2 (ja) | 2019-03-20 |
Family
ID=48628225
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2016516171A Active JP6488283B2 (ja) | 2013-05-31 | 2014-05-28 | Ｃ−ｃケモカイン受容体５型（ｃｃｒ５）遺伝子を開裂するｌａｇｌｉｄａｄｇホーミングエンドヌクレアーゼおよびその用途 |
Country Status (6)
| Country | Link |
|---|---|
| US (1) | US10006052B2 (enExample) |
| EP (1) | EP3004149B1 (enExample) |
| JP (1) | JP6488283B2 (enExample) |
| AU (1) | AU2014273089B2 (enExample) |
| CA (1) | CA2913871C (enExample) |
| WO (1) | WO2014191525A1 (enExample) |
Families Citing this family (67)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2013066438A2 (en) | 2011-07-22 | 2013-05-10 | President And Fellows Of Harvard College | Evaluation and improvement of nuclease cleavage specificity |
| US9458450B2 (en) | 2012-03-15 | 2016-10-04 | Flodesign Sonics, Inc. | Acoustophoretic separation technology using multi-dimensional standing waves |
| US10704021B2 (en) | 2012-03-15 | 2020-07-07 | Flodesign Sonics, Inc. | Acoustic perfusion devices |
| 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 |
| US9340799B2 (en) | 2013-09-06 | 2016-05-17 | President And Fellows Of Harvard College | MRNA-sensing switchable gRNAs |
| US9388430B2 (en) | 2013-09-06 | 2016-07-12 | President And Fellows Of Harvard College | Cas9-recombinase fusion proteins and uses thereof |
| US9737604B2 (en) | 2013-09-06 | 2017-08-22 | President And Fellows Of Harvard College | Use of cationic lipids to deliver CAS9 |
| KR20230054509A (ko) | 2013-11-07 | 2023-04-24 | 에디타스 메디신, 인코포레이티드 | 지배적인 gRNA를 이용하는 CRISPR-관련 방법 및 조성물 |
| US11053481B2 (en) | 2013-12-12 | 2021-07-06 | President And Fellows Of Harvard College | Fusions of Cas9 domains and nucleic acid-editing domains |
| CA2935960C (en) | 2014-01-08 | 2023-01-10 | Bart Lipkens | Acoustophoresis device with dual acoustophoretic chamber |
| AU2015298571B2 (en) | 2014-07-30 | 2020-09-03 | President And Fellows Of Harvard College | Cas9 proteins including ligand-dependent inteins |
| US11377651B2 (en) | 2016-10-19 | 2022-07-05 | Flodesign Sonics, Inc. | Cell therapy processes utilizing acoustophoresis |
| US11708572B2 (en) | 2015-04-29 | 2023-07-25 | Flodesign Sonics, Inc. | Acoustic cell separation techniques and processes |
| US11474085B2 (en) | 2015-07-28 | 2022-10-18 | Flodesign Sonics, Inc. | Expanded bed affinity selection |
| US11459540B2 (en) | 2015-07-28 | 2022-10-04 | Flodesign Sonics, Inc. | Expanded bed affinity selection |
| 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 |
| EP4108255A1 (en) | 2015-10-05 | 2022-12-28 | Precision Biosciences, Inc. | Genetically-modified cells comprising a modified human t cell receptor alpha constant region gene |
| AU2016333886B2 (en) | 2015-10-05 | 2020-10-08 | Precision Biosciences, Inc. | Engineered meganucleases with recognition sequences found in the human T cell receptor alpha constant region gene |
| US12043852B2 (en) | 2015-10-23 | 2024-07-23 | President And Fellows Of Harvard College | Evolved Cas9 proteins for gene editing |
| WO2017189964A2 (en) * | 2016-04-29 | 2017-11-02 | Voyager Therapeutics, Inc. | Compositions for the treatment of disease |
| US11085035B2 (en) | 2016-05-03 | 2021-08-10 | Flodesign Sonics, Inc. | Therapeutic cell washing, concentration, and separation utilizing acoustophoresis |
| US11214789B2 (en) | 2016-05-03 | 2022-01-04 | Flodesign Sonics, Inc. | Concentration and washing of particles with acoustics |
| CN109689865A (zh) * | 2016-07-25 | 2019-04-26 | 蓝鸟生物公司 | Bcl11a归巢核酸内切酶变体、组合物和使用方法 |
| 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 |
| CA3034094A1 (en) * | 2016-08-16 | 2018-02-22 | Bluebird Bio, Inc. | Il-10 receptor alpha homing endonuclease variants, compositions, and methods of use |
| US11542509B2 (en) | 2016-08-24 | 2023-01-03 | President And Fellows Of Harvard College | Incorporation of unnatural amino acids into proteins using base editing |
| IL265045B2 (en) | 2016-09-08 | 2023-09-01 | Bluebird Bio Inc | Variants of endonuclease pd-1, compositions and methods of use |
| AU2017342543B2 (en) | 2016-10-14 | 2024-06-27 | President And Fellows Of Harvard College | AAV delivery of nucleobase editors |
| AU2017346683B2 (en) * | 2016-10-17 | 2024-04-18 | Regeneron Pharmaceuticals, Inc. | TGFβR2 endonuclease variants, compositions, and methods of use |
| WO2018119359A1 (en) | 2016-12-23 | 2018-06-28 | President And Fellows Of Harvard College | Editing of ccr5 receptor gene to protect against hiv infection |
| EP3592381A1 (en) | 2017-03-09 | 2020-01-15 | 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 |
| KR20190127797A (ko) | 2017-03-10 | 2019-11-13 | 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 | 시토신에서 구아닌으로의 염기 편집제 |
| CA3057192A1 (en) | 2017-03-23 | 2018-09-27 | President And Fellows Of Harvard College | Nucleobase editors comprising nucleic acid programmable dna binding proteins |
| CA3059643A1 (en) | 2017-04-13 | 2018-10-18 | Cellectis | New sequence specific reagents targeting ccr5 in primary hematopoietic cells |
| 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 |
| KR102590466B1 (ko) | 2017-05-25 | 2023-10-19 | 2세븐티 바이오, 인코포레이티드 | Cblb 엔도뉴클레아제 변이체, 조성물 및 사용 방법 |
| AU2018292526A1 (en) | 2017-06-30 | 2020-01-16 | 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 |
| WO2019139645A2 (en) | 2017-08-30 | 2019-07-18 | President And Fellows Of Harvard College | High efficiency base editors comprising gam |
| CA3082251A1 (en) | 2017-10-16 | 2019-04-25 | The Broad Institute, Inc. | Uses of adenosine base editors |
| KR20220066413A (ko) | 2017-12-14 | 2022-05-24 | 프로디자인 소닉스, 인크. | 음향 트랜스듀서 구동기 및 제어기 |
| EP3724214A4 (en) | 2017-12-15 | 2021-09-01 | The Broad Institute Inc. | SYSTEMS AND METHODS FOR PREDICTING REPAIR RESULTS IN GENETIC ENGINEERING |
| WO2019126555A1 (en) * | 2017-12-20 | 2019-06-27 | Bluebird Bio, Inc. | Ctla4 homing endonuclease variants, compositions, and methods of use |
| WO2019200122A1 (en) | 2018-04-12 | 2019-10-17 | Precision Biosciences, Inc. | Optimized engineered nucleases having specificity for the human t cell receptor alpha constant region gene |
| WO2019204226A1 (en) | 2018-04-16 | 2019-10-24 | University Of Massachusetts | Compositions and methods for improved gene editing |
| WO2019226953A1 (en) | 2018-05-23 | 2019-11-28 | The Broad Institute, Inc. | Base editors and uses thereof |
| BR112020025439A2 (pt) | 2018-06-14 | 2021-03-16 | Bluebird Bio, Inc. | Receptores de antígenos quiméricos cd79a |
| WO2020072059A1 (en) | 2018-10-04 | 2020-04-09 | Bluebird Bio, Inc. | Cblb endonuclease variants, compositions, and methods of use |
| US12281338B2 (en) | 2018-10-29 | 2025-04-22 | The Broad Institute, Inc. | Nucleobase editors comprising GeoCas9 and uses thereof |
| KR20210102925A (ko) | 2018-12-10 | 2021-08-20 | 블루버드 바이오, 인코포레이티드. | 호밍 엔도뉴클레아제 변이체 |
| US12351837B2 (en) | 2019-01-23 | 2025-07-08 | The Broad Institute, Inc. | Supernegatively charged proteins and uses thereof |
| DE112020001306T5 (de) | 2019-03-19 | 2022-01-27 | Massachusetts Institute Of Technology | Verfahren und zusammensetzungen zur editierung von nukleotidsequenzen |
| US12473543B2 (en) | 2019-04-17 | 2025-11-18 | The Broad Institute, Inc. | Adenine base editors with reduced off-target effects |
| WO2020257684A1 (en) | 2019-06-20 | 2020-12-24 | University Of Massachusetts | Compositions and methods for improved gene editing |
| US12435330B2 (en) | 2019-10-10 | 2025-10-07 | The Broad Institute, Inc. | Methods and compositions for prime editing RNA |
| WO2021191870A1 (en) | 2020-03-27 | 2021-09-30 | Dcprime B.V. | Ex vivo use of modified cells of leukemic origin for enhancing the efficacy of adoptive cell therapy |
| WO2021222451A2 (en) * | 2020-04-28 | 2021-11-04 | City Of Hope | Tagged gene editing technology for clinical cell sorting and enrichment |
| 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 |
| EP4171617A1 (en) | 2020-06-30 | 2023-05-03 | Mendus B.V. | Use of leukemia-derived cells in ovarian cancer vaccines |
| US20220305100A1 (en) | 2021-03-12 | 2022-09-29 | Dcprime B.V. | Methods of vaccination and use of cd47 blockade |
| WO2023081756A1 (en) | 2021-11-03 | 2023-05-11 | The J. David Gladstone Institutes, A Testamentary Trust Established Under The Will Of J. David Gladstone | Precise genome editing using retrons |
| GB202117314D0 (en) | 2021-11-30 | 2022-01-12 | Clarke David John | Cyclic nucleic acid fragmentation |
| WO2023141602A2 (en) | 2022-01-21 | 2023-07-27 | Renagade Therapeutics Management Inc. | Engineered retrons and methods of use |
| WO2024044723A1 (en) | 2022-08-25 | 2024-02-29 | Renagade Therapeutics Management Inc. | Engineered retrons and methods of use |
Family Cites Families (5)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2011156430A2 (en) | 2010-06-07 | 2011-12-15 | Fred Hutchinson Cancer Research Center | Generation and expression of engineered i-onui endonuclease and its homologues and uses thereof |
| EP4062927A1 (en) * | 2011-02-28 | 2022-09-28 | Seattle Children's Research Institute | Coupling endonucleases with end-processing enzymes drives high efficiency gene disruption |
| EP2694091B1 (en) * | 2011-04-05 | 2019-03-13 | Cellectis | Method for the generation of compact tale-nucleases and uses thereof |
| EP2729567B1 (en) * | 2011-07-08 | 2016-10-05 | Cellectis | Method for increasing the efficiency of double-strand break-induced mutagenssis |
| EP2776560A2 (en) * | 2011-11-07 | 2014-09-17 | The University of Western Ontario | Endonuclease for genome editing |
-
2014
- 2014-05-28 EP EP14727480.7A patent/EP3004149B1/en active Active
- 2014-05-28 AU AU2014273089A patent/AU2014273089B2/en active Active
- 2014-05-28 US US14/891,202 patent/US10006052B2/en active Active
- 2014-05-28 WO PCT/EP2014/061186 patent/WO2014191525A1/en not_active Ceased
- 2014-05-28 JP JP2016516171A patent/JP6488283B2/ja active Active
- 2014-05-28 CA CA2913871A patent/CA2913871C/en active Active
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