JP2016520319A5 - - Google Patents
Download PDFInfo
- Publication number
- 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
- Authority
- JP
- Japan
- Prior art keywords
- seq
- activity
- 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
Links
- 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 (OSRAM) | 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 (OSRAM) |
| EP (1) | EP3004149B1 (OSRAM) |
| JP (1) | JP6488283B2 (OSRAM) |
| AU (1) | AU2014273089B2 (OSRAM) |
| CA (1) | CA2913871C (OSRAM) |
| WO (1) | WO2014191525A1 (OSRAM) |
Families Citing this family (67)
| 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 |
| 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 |
| US9526784B2 (en) | 2013-09-06 | 2016-12-27 | President And Fellows Of Harvard College | Delivery system for functional nucleases |
| US9340800B2 (en) | 2013-09-06 | 2016-05-17 | President And Fellows Of Harvard College | Extended DNA-sensing GRNAS |
| US9388430B2 (en) | 2013-09-06 | 2016-07-12 | President And Fellows Of Harvard College | Cas9-recombinase fusion proteins and uses thereof |
| CA2930015A1 (en) | 2013-11-07 | 2015-05-14 | Editas Medicine, Inc. | Crispr-related methods and compositions with governing grnas |
| US9840699B2 (en) | 2013-12-12 | 2017-12-12 | President And Fellows Of Harvard College | Methods for nucleic acid editing |
| US9725710B2 (en) | 2014-01-08 | 2017-08-08 | Flodesign Sonics, Inc. | Acoustophoresis device with dual acoustophoretic chamber |
| WO2016022363A2 (en) | 2014-07-30 | 2016-02-11 | President And Fellows Of Harvard College | Cas9 proteins including ligand-dependent inteins |
| US11708572B2 (en) | 2015-04-29 | 2023-07-25 | Flodesign Sonics, Inc. | Acoustic cell separation techniques and processes |
| US11377651B2 (en) | 2016-10-19 | 2022-07-05 | Flodesign Sonics, Inc. | Cell therapy processes utilizing acoustophoresis |
| US11459540B2 (en) | 2015-07-28 | 2022-10-04 | Flodesign Sonics, Inc. | Expanded bed affinity selection |
| US11474085B2 (en) | 2015-07-28 | 2022-10-18 | 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 |
| AU2016333898B2 (en) | 2015-10-05 | 2020-11-12 | Precision Biosciences, Inc. | Genetically-modified cells comprising a modified human T cell receptor alpha constant region gene |
| DK3359660T3 (en) | 2015-10-05 | 2020-02-17 | Prec Biosciences Inc | Engineered meganucleases with recognition sequences found in the human t cell receptor alpha constant region gene |
| IL258821B (en) | 2015-10-23 | 2022-07-01 | Harvard College | Nucleobase editors and their uses |
| EP3448987A4 (en) | 2016-04-29 | 2020-05-27 | Voyager Therapeutics, Inc. | COMPOSITIONS FOR TREATING A DISEASE |
| US11214789B2 (en) | 2016-05-03 | 2022-01-04 | Flodesign Sonics, Inc. | Concentration and washing of particles with acoustics |
| US11085035B2 (en) | 2016-05-03 | 2021-08-10 | Flodesign Sonics, Inc. | Therapeutic cell washing, concentration, and separation utilizing acoustophoresis |
| JP2019525759A (ja) * | 2016-07-25 | 2019-09-12 | ブルーバード バイオ， インコーポレイテッド | Ｂｃｌ１１ａホーミングエンドヌクレアーゼバリアント、組成物、および使用方法 |
| 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 |
| WO2018035141A1 (en) | 2016-08-16 | 2018-02-22 | Bluebird Bio, Inc. | Il-10 receptor alpha homing endonuclease variants, compositions, and methods of use |
| WO2018039438A1 (en) | 2016-08-24 | 2018-03-01 | President And Fellows Of Harvard College | Incorporation of unnatural amino acids into proteins using base editing |
| WO2018049226A1 (en) | 2016-09-08 | 2018-03-15 | Bluebird Bio, Inc. | Pd-1 homing endonuclease variants, compositions, and methods of use |
| 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 |
| CN110050066A (zh) * | 2016-10-17 | 2019-07-23 | 蓝鸟生物公司 | TGFβR2核酸内切酶变体、组合物和使用方法 |
| 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 |
| BR112019019655A2 (pt) | 2017-03-23 | 2020-04-22 | Harvard College | editores de nucleobase que compreendem proteínas de ligação a dna programáveis por ácido nucleico |
| AU2018251150B2 (en) | 2017-04-13 | 2024-05-09 | Albert-Ludwigs-Universität Freiburg | 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 |
| IL270776B2 (en) * | 2017-05-25 | 2024-11-01 | Bluebird Bio Inc | Cblb endonuclease variants, compositions, and methods of use |
| WO2019005957A1 (en) | 2017-06-30 | 2019-01-03 | Precision Biosciences, Inc. | GENETICALLY MODIFIED T CELLS COMPRISING A MODIFIED INTRON IN THE ALPHA T CELL RECEPTOR GENE |
| 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 |
| KR20250107288A (ko) | 2017-10-16 | 2025-07-11 | 더 브로드 인스티튜트, 인코퍼레이티드 | 아데노신 염기 편집제의 용도 |
| US10785574B2 (en) | 2017-12-14 | 2020-09-22 | Flodesign Sonics, Inc. | Acoustic transducer driver and controller |
| WO2019118949A1 (en) | 2017-12-15 | 2019-06-20 | The Broad Institute, Inc. | Systems and methods for predicting repair outcomes in genetic engineering |
| WO2019126555A1 (en) * | 2017-12-20 | 2019-06-27 | Bluebird Bio, Inc. | Ctla4 homing endonuclease variants, compositions, and methods of use |
| JP7304888B2 (ja) | 2018-04-12 | 2023-07-07 | プレシジョン バイオサイエンシズ，インク． | ヒトｔ細胞受容体アルファ定常領域遺伝子に特異性を有する最適化された操作されたヌクレアーゼ |
| US11981892B2 (en) | 2018-04-16 | 2024-05-14 | 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 |
| EP3806903B1 (en) | 2018-06-14 | 2024-02-14 | 2seventy bio, Inc. | Cd79a chimeric antigen receptors |
| WO2020072059A1 (en) | 2018-10-04 | 2020-04-09 | Bluebird Bio, Inc. | Cblb endonuclease variants, compositions, and methods of use |
| WO2020092453A1 (en) | 2018-10-29 | 2020-05-07 | 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 |
| 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 |
| EP3987024A4 (en) | 2019-06-20 | 2023-11-01 | University Of Massachusetts | COMPOSITIONS AND METHODS FOR ENHANCED GENOMIC EDITING |
| US12435330B2 (en) | 2019-10-10 | 2025-10-07 | The Broad Institute, Inc. | Methods and compositions for prime editing RNA |
| CA3172449A1 (en) | 2020-03-27 | 2021-09-30 | Erik Hans MANTING | 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 |
| 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 |
| CN116133679A (zh) | 2020-06-30 | 2023-05-16 | 门德斯有限公司 | 白血病衍生细胞在卵巢癌疫苗中的用途 |
| WO2022190058A1 (en) | 2021-03-12 | 2022-09-15 | Dcprime B.V. | Methods of vaccination and use of cd47 blockade |
| CA3237482A1 (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 |
|---|---|---|---|---|
| US20140148361A1 (en) * | 2010-06-07 | 2014-05-29 | Barry L. Stoddard | Generation and Expression of Engineered I-ONUI Endonuclease and Its Homologues and Uses Thereof |
| EP3603662B1 (en) * | 2011-02-28 | 2022-01-19 | Seattle Children's Research Institute | Coupling endonucleases with end-processing enzymes drive high efficiency gene disruption |
| KR101982360B1 (ko) * | 2011-04-05 | 2019-05-24 | 셀렉티스 | 콤팩트 tale-뉴클레아제의 발생 방법 및 이의 용도 |
| US9540623B2 (en) * | 2011-07-08 | 2017-01-10 | Cellectis | Method for increasing the efficiency of double-strand-break induced mutagenesis |
| US20130210151A1 (en) * | 2011-11-07 | 2013-08-15 | University Of Western Ontario | Endonuclease for genome editing |
-
2014
- 2014-05-28 AU AU2014273089A patent/AU2014273089B2/en active Active
- 2014-05-28 JP JP2016516171A patent/JP6488283B2/ja active Active
- 2014-05-28 CA CA2913871A patent/CA2913871C/en active Active
- 2014-05-28 WO PCT/EP2014/061186 patent/WO2014191525A1/en not_active Ceased
- 2014-05-28 EP EP14727480.7A patent/EP3004149B1/en active Active
- 2014-05-28 US US14/891,202 patent/US10006052B2/en active Active
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| JP2016520319A5 (OSRAM) | ||
| JP2016520320A5 (OSRAM) | ||
| ES2993268T3 (en) | Genetically engineered t cells with regnase-1 and/or tgfbrii disruption have improved functionality and persistence | |
| AU2022215269A1 (en) | Genetically engineered cells and methods of making the same | |
| TW202043249A (zh) | 編輯rna的方法和組合物 | |
| TW202028466A (zh) | 編輯RNAs的方法以及組成物 | |
| JP2019150033A (ja) | 変異ｃａｓ９タンパク質 | |
| JP2017532955A5 (OSRAM) | ||
| JP2015533786A5 (OSRAM) | ||
| JP2017018125A5 (OSRAM) | ||
| US20250250350A1 (en) | Immune cells having co-expressed shrnas and logic gate systems | |
| WO2013186563A3 (en) | Factor viii sequences | |
| AU2021369494A9 (en) | Safe harbor loci | |
| JP2025517359A (ja) | Ｐｓｍａ及びｃａ９を標的とする操作された受容体のシステム | |
| KR20250068649A (ko) | 공동 발현된 tgfbr shrna를 갖는 면역 세포 | |
| KR20210151110A (ko) | Scn9a 또는 scn10a 유전자를 변형시키기 위한 유전자-편집 시스템 및 이의 방법 및 용도 | |
| EP3841114A1 (en) | Methods and compositions for rna expression of myc inhibitors | |
| US20250222103A1 (en) | Immune cells with combination gene perturbations | |
| JP2015174843A5 (OSRAM) | ||
| CN118843692A (zh) | 具有共表达的shRNA和逻辑门系统的免疫细胞 | |
| NZ742751A (en) | Materials and methods for treatment of titin-based myopathies and other titinopathies |