JP2010517524A5 - - Google Patents
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- Publication number
- JP2010517524A5 JP2010517524A5 JP2009547783A JP2009547783A JP2010517524A5 JP 2010517524 A5 JP2010517524 A5 JP 2010517524A5 JP 2009547783 A JP2009547783 A JP 2009547783A JP 2009547783 A JP2009547783 A JP 2009547783A JP 2010517524 A5 JP2010517524 A5 JP 2010517524A5
- Authority
- JP
- Japan
- Prior art keywords
- meganuclease
- monomer
- obligate
- substitution
- amino acid
- 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
- 239000000178 monomer Substances 0.000 claims 31
- 235000001014 amino acid Nutrition 0.000 claims 18
- 238000006467 substitution reaction Methods 0.000 claims 16
- 150000001413 amino acids Chemical class 0.000 claims 15
- 239000012634 fragment Substances 0.000 claims 15
- 102000040430 polynucleotide Human genes 0.000 claims 14
- 239000002157 polynucleotide Substances 0.000 claims 14
- 108091033319 polynucleotide Proteins 0.000 claims 14
- 239000000833 heterodimer Substances 0.000 claims 13
- 230000035772 mutation Effects 0.000 claims 13
- 239000013598 vector Substances 0.000 claims 12
- 108090000623 proteins and genes Proteins 0.000 claims 8
- 230000009261 transgenic effect Effects 0.000 claims 6
- 230000002378 acidificating effect Effects 0.000 claims 5
- 125000003588 lysine group Chemical group [H]N([H])C([H])([H])C([H])([H])C([H])([H])C([H])([H])C([H])(N([H])[H])C(*)=O 0.000 claims 5
- 230000008685 targeting Effects 0.000 claims 5
- 108010042407 Endonucleases Proteins 0.000 claims 4
- 102000004533 Endonucleases Human genes 0.000 claims 4
- WHUUTDBJXJRKMK-UHFFFAOYSA-N Glutamic acid Natural products OC(=O)C(N)CCC(O)=O WHUUTDBJXJRKMK-UHFFFAOYSA-N 0.000 claims 4
- 235000013922 glutamic acid Nutrition 0.000 claims 4
- 239000004220 glutamic acid Substances 0.000 claims 4
- 125000000291 glutamic acid group Chemical group N[C@@H](CCC(O)=O)C(=O)* 0.000 claims 4
- 238000004519 manufacturing process Methods 0.000 claims 4
- COLNVLDHVKWLRT-UHFFFAOYSA-N phenylalanine Natural products OC(=O)C(N)CC1=CC=CC=C1 COLNVLDHVKWLRT-UHFFFAOYSA-N 0.000 claims 4
- COLNVLDHVKWLRT-QMMMGPOBSA-N phenylalanine group Chemical group N[C@@H](CC1=CC=CC=C1)C(=O)O COLNVLDHVKWLRT-QMMMGPOBSA-N 0.000 claims 4
- 239000004475 Arginine Substances 0.000 claims 3
- ROHFNLRQFUQHCH-UHFFFAOYSA-N Leucine Natural products CC(C)CC(N)C(O)=O ROHFNLRQFUQHCH-UHFFFAOYSA-N 0.000 claims 3
- 239000004472 Lysine Substances 0.000 claims 3
- KDXKERNSBIXSRK-UHFFFAOYSA-N Lysine Natural products NCCCCC(N)C(O)=O KDXKERNSBIXSRK-UHFFFAOYSA-N 0.000 claims 3
- 241001465754 Metazoa Species 0.000 claims 3
- ODKSFYDXXFIFQN-UHFFFAOYSA-N arginine Natural products OC(=O)C(N)CCCNC(N)=N ODKSFYDXXFIFQN-UHFFFAOYSA-N 0.000 claims 3
- -1 aromatic amino acid Chemical class 0.000 claims 3
- 108010050663 endodeoxyribonuclease CreI Proteins 0.000 claims 3
- 230000006801 homologous recombination Effects 0.000 claims 3
- 238000002744 homologous recombination Methods 0.000 claims 3
- 238000000338 in vitro Methods 0.000 claims 3
- 230000001939 inductive effect Effects 0.000 claims 3
- 125000001909 leucine group Chemical group [H]N(*)C(C(*)=O)C([H])([H])C(C([H])([H])[H])C([H])([H])[H] 0.000 claims 3
- 238000000034 method Methods 0.000 claims 3
- 102000039446 nucleic acids Human genes 0.000 claims 3
- 108020004707 nucleic acids Proteins 0.000 claims 3
- 150000007523 nucleic acids Chemical class 0.000 claims 3
- DHMQDGOQFOQNFH-UHFFFAOYSA-N Glycine Chemical compound NCC(O)=O DHMQDGOQFOQNFH-UHFFFAOYSA-N 0.000 claims 2
- 102220580831 Serine/threonine-protein kinase STK11_K96R_mutation Human genes 0.000 claims 2
- 230000015572 biosynthetic process Effects 0.000 claims 2
- 239000013604 expression vector Substances 0.000 claims 2
- 238000010353 genetic engineering Methods 0.000 claims 2
- 238000001727 in vivo Methods 0.000 claims 2
- 239000012678 infectious agent Substances 0.000 claims 2
- 239000000203 mixture Substances 0.000 claims 2
- 230000001105 regulatory effect Effects 0.000 claims 2
- 230000008439 repair process Effects 0.000 claims 2
- 102200052872 rs118203897 Human genes 0.000 claims 2
- 102200081526 rs121913583 Human genes 0.000 claims 2
- 102220017736 rs137854215 Human genes 0.000 claims 2
- 102200155456 rs35947557 Human genes 0.000 claims 2
- 238000012270 DNA recombination Methods 0.000 claims 1
- 239000004471 Glycine Substances 0.000 claims 1
- 208000026350 Inborn Genetic disease Diseases 0.000 claims 1
- FFEARJCKVFRZRR-BYPYZUCNSA-N L-methionine Chemical compound CSCC[C@H](N)C(O)=O FFEARJCKVFRZRR-BYPYZUCNSA-N 0.000 claims 1
- QIVBCDIJIAJPQS-VIFPVBQESA-N L-tryptophane Chemical compound C1=CC=C2C(C[C@H](N)C(O)=O)=CNC2=C1 QIVBCDIJIAJPQS-VIFPVBQESA-N 0.000 claims 1
- QIVBCDIJIAJPQS-UHFFFAOYSA-N Tryptophan Natural products C1=CC=C2C(CC(N)C(O)=O)=CNC2=C1 QIVBCDIJIAJPQS-UHFFFAOYSA-N 0.000 claims 1
- 241000700605 Viruses Species 0.000 claims 1
- 150000001484 arginines Chemical group 0.000 claims 1
- 235000003704 aspartic acid Nutrition 0.000 claims 1
- CKLJMWTZIZZHCS-REOHCLBHSA-N aspartic acid group Chemical group N[C@@H](CC(=O)O)C(=O)O CKLJMWTZIZZHCS-REOHCLBHSA-N 0.000 claims 1
- OQFSQFPPLPISGP-UHFFFAOYSA-N beta-carboxyaspartic acid Natural products OC(=O)C(N)C(C(O)=O)C(O)=O OQFSQFPPLPISGP-UHFFFAOYSA-N 0.000 claims 1
- 230000030833 cell death Effects 0.000 claims 1
- 239000013611 chromosomal DNA Substances 0.000 claims 1
- 230000002759 chromosomal effect Effects 0.000 claims 1
- 238000003776 cleavage reaction Methods 0.000 claims 1
- 230000037430 deletion Effects 0.000 claims 1
- 238000012217 deletion Methods 0.000 claims 1
- 239000000539 dimer Substances 0.000 claims 1
- 201000010099 disease Diseases 0.000 claims 1
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 claims 1
- 239000003814 drug Substances 0.000 claims 1
- 230000001747 exhibiting effect Effects 0.000 claims 1
- 208000016361 genetic disease Diseases 0.000 claims 1
- 239000000710 homodimer Substances 0.000 claims 1
- 208000015181 infectious disease Diseases 0.000 claims 1
- 230000002452 interceptive effect Effects 0.000 claims 1
- 230000009878 intermolecular interaction Effects 0.000 claims 1
- 229930182817 methionine Natural products 0.000 claims 1
- 238000010397 one-hybrid screening Methods 0.000 claims 1
- 239000000825 pharmaceutical preparation Substances 0.000 claims 1
- 229940127557 pharmaceutical product Drugs 0.000 claims 1
- 108090000765 processed proteins & peptides Proteins 0.000 claims 1
- 230000007017 scission Effects 0.000 claims 1
Applications Claiming Priority (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| PCT/IB2007/000849 WO2008093152A1 (en) | 2007-02-01 | 2007-02-01 | Obligate heterodimer meganucleases and uses thereof |
| PCT/IB2008/001333 WO2008093249A2 (en) | 2007-02-01 | 2008-01-31 | Obligate heterodimer meganucleases and uses thereof |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2010517524A JP2010517524A (ja) | 2010-05-27 |
| JP2010517524A5 true JP2010517524A5 (https=) | 2011-03-17 |
Family
ID=38283534
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2009547783A Pending JP2010517524A (ja) | 2007-02-01 | 2008-01-31 | 偏性ヘテロ二量体メガヌクレアーゼ及びその使用 |
Country Status (7)
| Country | Link |
|---|---|
| US (1) | US20100167357A1 (https=) |
| EP (2) | EP2433641A1 (https=) |
| JP (1) | JP2010517524A (https=) |
| CN (1) | CN101678087A (https=) |
| AU (1) | AU2008211635A1 (https=) |
| CA (1) | CA2677177A1 (https=) |
| WO (2) | WO2008093152A1 (https=) |
Families Citing this family (53)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2009095742A1 (en) * | 2008-01-31 | 2009-08-06 | Cellectis | New i-crei derived single-chain meganuclease and uses thereof |
| EP3202899B1 (en) | 2003-01-28 | 2020-10-21 | Cellectis | Custom-made meganuclease and use thereof |
| EP1863909B2 (en) | 2005-03-15 | 2014-09-10 | Cellectis | I-crei meganuclease variants with modified specificity, method of preparation and uses thereof |
| EP2167666A2 (en) | 2007-06-29 | 2010-03-31 | Pioneer Hi-Bred International Inc. | Methods for altering the genome of a monocot plant cell |
| WO2009019528A1 (en) * | 2007-08-03 | 2009-02-12 | Cellectis | Meganuclease variants cleaving a dna target sequence from the human interleukin-2 receptor gamma chain gene and uses thereof |
| EP2215223B1 (en) | 2007-10-31 | 2013-05-01 | Precision Biosciences, Inc. | Rationally-designed single-chain meganucleases with non-palindromic recognition sequences |
| WO2009074842A1 (en) * | 2007-12-13 | 2009-06-18 | Cellectis | Improved chimeric meganuclease enzymes and uses thereof |
| WO2009114321A2 (en) | 2008-03-11 | 2009-09-17 | Precision Biosciencs, Inc. | Rationally-designed meganucleases for maize genome engineering |
| EP2313498B1 (en) | 2008-07-14 | 2017-03-15 | Precision Biosciences, Inc. | Recognition sequences for i-crei-derived meganucleases and uses thereof |
| CN102177235A (zh) * | 2008-09-08 | 2011-09-07 | 赛莱克蒂斯公司 | 切割来自谷氨酰胺合成酶基因的dna靶序列的大范围核酸酶变体及其用途 |
| EP2180058A1 (en) | 2008-10-23 | 2010-04-28 | Cellectis | Meganuclease recombination system |
| WO2011036510A1 (en) * | 2009-09-24 | 2011-03-31 | Cellectis | Meganuclease variants cleaving the genome of the herpes simplex virus and uses thereof |
| US20120171191A1 (en) | 2009-05-26 | 2012-07-05 | Cellectis | Meganuclease variants cleaving the genome of a pathogenic non-integrating virus and uses thereof |
| JP5940977B2 (ja) * | 2009-08-11 | 2016-06-29 | サンガモ バイオサイエンシーズ， インコーポレイテッド | 標的改変によるホモ接合生物 |
| EP2480659A2 (en) | 2009-09-24 | 2012-08-01 | Cellectis | Meganuclease reagents of uses thereof for treating genetic diseases caused by frame shift/non sense mutations |
| AU2010325563B2 (en) | 2009-11-27 | 2017-02-02 | Basf Plant Science Company Gmbh | Chimeric endonucleases and uses thereof |
| EP2504439B1 (en) | 2009-11-27 | 2016-03-02 | BASF Plant Science Company GmbH | Optimized endonucleases and uses thereof |
| CN102762726A (zh) | 2009-11-27 | 2012-10-31 | 巴斯夫植物科学有限公司 | 嵌合内切核酸酶及其用途 |
| WO2011104382A1 (en) | 2010-02-26 | 2011-09-01 | Cellectis | Use of endonucleases for inserting transgenes into safe harbor loci |
| WO2012017329A2 (en) | 2010-08-02 | 2012-02-09 | Cellectis S.A. | Method for targeted genomic events in algae |
| US9044492B2 (en) | 2011-02-04 | 2015-06-02 | Cellectis Sa | Method for modulating the efficiency of double-strand break-induced mutagenesis |
| CA3111953C (en) | 2011-04-05 | 2023-10-24 | Cellectis | Method for the generation of compact tale-nucleases and uses thereof |
| WO2012138901A1 (en) | 2011-04-05 | 2012-10-11 | Cellectis Sa | Method for enhancing rare-cutting endonuclease efficiency and uses thereof |
| BR112013030652A2 (pt) | 2011-06-10 | 2016-12-13 | Basf Plant Science Co Gmbh | polinucleotídeo que codifica um polipeptídeo, molécula de ácido nucleico, vetor, organismo não humano, polipeptídeo e método para introduzir um ácido nucleico de interesse em um genoma de um organismo não humano |
| EP2612918A1 (en) | 2012-01-06 | 2013-07-10 | BASF Plant Science Company GmbH | In planta recombination |
| EP2877488B1 (en) | 2012-07-24 | 2021-02-24 | Cellectis | New modular base-specific nucleic acid binding domains from burkholderia rhizoxinica proteins |
| CN105121649A (zh) | 2012-11-16 | 2015-12-02 | 赛莱蒂克斯公司 | 靶向修饰藻基因组的方法 |
| EP2931899A1 (en) | 2012-12-12 | 2015-10-21 | The Broad Institute, Inc. | Functional genomics using crispr-cas systems, compositions, methods, knock out libraries and applications thereof |
| WO2014093709A1 (en) | 2012-12-12 | 2014-06-19 | The Broad Institute, Inc. | Methods, models, systems, and apparatus for identifying target sequences for cas enzymes or crispr-cas systems for target sequences and conveying results thereof |
| EP3031921B1 (en) | 2012-12-12 | 2025-03-12 | The Broad Institute, Inc. | Delivery, engineering and optimization of systems, methods and compositions for sequence manipulation and therapeutic applications |
| EP2840140B2 (en) * | 2012-12-12 | 2023-02-22 | The Broad Institute, Inc. | Crispr-Cas based method for mutation of prokaryotic cells |
| WO2014102688A1 (en) | 2012-12-27 | 2014-07-03 | Cellectis | New design matrix for improvement of homology-directed gene targeting |
| CN112301024A (zh) * | 2013-03-15 | 2021-02-02 | 通用医疗公司 | 使用RNA引导的FokI核酸酶(RFN)提高RNA引导的基因组编辑的特异性 |
| EP3725885A1 (en) | 2013-06-17 | 2020-10-21 | The Broad Institute, Inc. | Functional genomics using crispr-cas systems, compositions methods, screens and applications thereof |
| RU2716420C2 (ru) | 2013-06-17 | 2020-03-11 | Те Брод Инститьют Инк. | Доставка и применение систем crispr-cas, векторов и композиций для целенаправленного воздействия и терапии в печени |
| WO2014204724A1 (en) | 2013-06-17 | 2014-12-24 | The Broad Institute Inc. | Delivery, engineering and optimization of tandem guide systems, methods and compositions for sequence manipulation |
| CN105492611A (zh) | 2013-06-17 | 2016-04-13 | 布罗德研究所有限公司 | 用于序列操纵的优化的crispr-cas双切口酶系统、方法以及组合物 |
| MX374532B (es) | 2013-06-17 | 2025-03-06 | Broad Inst Inc | Suministro, uso y aplicaciones terapéuticas de los sistemas y composiciones crispr-cas, para actuar sobre trastornos y enfermedades utilizando componentes víricos. |
| JP6712948B2 (ja) | 2013-12-12 | 2020-06-24 | ザ・ブロード・インスティテュート・インコーポレイテッド | 組成物、及びヌクレオチドリピート障害におけるｃｒｉｓｐｒ−ｃａｓ系の使用方法 |
| SG10201804975PA (en) | 2013-12-12 | 2018-07-30 | Broad Inst Inc | Delivery, Use and Therapeutic Applications of the Crispr-Cas Systems and Compositions for HBV and Viral Diseases and Disorders |
| EP3080259B1 (en) | 2013-12-12 | 2023-02-01 | The Broad Institute, Inc. | Engineering of systems, methods and optimized guide compositions with new architectures for sequence manipulation |
| JP7103750B2 (ja) | 2013-12-12 | 2022-07-20 | ザ・ブロード・インスティテュート・インコーポレイテッド | ゲノム編集のためのＣＲＩＳＰＲ－Ｃａｓ系及び組成物の送達、使用及び治療適用 |
| WO2015089364A1 (en) | 2013-12-12 | 2015-06-18 | The Broad Institute Inc. | Crystal structure of a crispr-cas system, and uses thereof |
| JP6793547B2 (ja) | 2013-12-12 | 2020-12-02 | ザ・ブロード・インスティテュート・インコーポレイテッド | 最適化機能ＣＲＩＳＰＲ−Ｃａｓ系による配列操作のための系、方法および組成物 |
| EP3889260A1 (en) | 2014-12-12 | 2021-10-06 | The Broad Institute, Inc. | Protected guide rnas (pgrnas) |
| CA2970370A1 (en) | 2014-12-24 | 2016-06-30 | Massachusetts Institute Of Technology | Crispr having or associated with destabilization domains |
| WO2016205759A1 (en) | 2015-06-18 | 2016-12-22 | The Broad Institute Inc. | Engineering and optimization of systems, methods, enzymes and guide scaffolds of cas9 orthologs and variants for sequence manipulation |
| TWI906646B (zh) | 2015-06-18 | 2025-12-01 | 美商博得學院股份有限公司 | 降低脫靶效應的ｃｒｉｓｐｒ酶突變 |
| JP7422128B2 (ja) * | 2018-04-03 | 2024-01-25 | ジーフラス ライフ サイエンシズ，インク． | 配列特異的なインビボ細胞標的化 |
| 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 |
| 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 |
| CN119446263B (zh) * | 2024-10-30 | 2025-10-03 | 华东理工大学 | 一种预测psi蛋白单位点突变后单体和二聚体的比例变化的方法和psi蛋白的突变体 |
Family Cites Families (5)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| AU2003215869B2 (en) * | 2002-03-15 | 2008-04-24 | Cellectis | Hybrid and single chain meganucleases and use thereof |
| AU2003290518A1 (en) * | 2002-09-06 | 2004-04-23 | Fred Hutchinson Cancer Research Center | Methods and compositions concerning designed highly-specific nucleic acid binding proteins |
| EP3202899B1 (en) * | 2003-01-28 | 2020-10-21 | Cellectis | Custom-made meganuclease and use thereof |
| EP1863909B2 (en) * | 2005-03-15 | 2014-09-10 | Cellectis | I-crei meganuclease variants with modified specificity, method of preparation and uses thereof |
| ES2582091T3 (es) * | 2005-10-18 | 2016-09-09 | Precision Biosciences | Meganucleasas diseñadas racionalmente con especificidad de secuencia y afinidad de unión a ADN alteradas |
-
2007
- 2007-02-01 WO PCT/IB2007/000849 patent/WO2008093152A1/en not_active Ceased
-
2008
- 2008-01-31 EP EP11193586A patent/EP2433641A1/en not_active Withdrawn
- 2008-01-31 CA CA002677177A patent/CA2677177A1/en not_active Abandoned
- 2008-01-31 JP JP2009547783A patent/JP2010517524A/ja active Pending
- 2008-01-31 EP EP08751043.4A patent/EP2121004B1/en not_active Not-in-force
- 2008-01-31 WO PCT/IB2008/001333 patent/WO2008093249A2/en not_active Ceased
- 2008-01-31 AU AU2008211635A patent/AU2008211635A1/en not_active Abandoned
- 2008-01-31 CN CN200880006978A patent/CN101678087A/zh active Pending
- 2008-01-31 US US12/449,329 patent/US20100167357A1/en not_active Abandoned
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