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 (enExample) | 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 (enExample) |
| EP (2) | EP2433641A1 (enExample) |
| JP (1) | JP2010517524A (enExample) |
| CN (1) | CN101678087A (enExample) |
| AU (1) | AU2008211635A1 (enExample) |
| CA (1) | CA2677177A1 (enExample) |
| WO (2) | WO2008093152A1 (enExample) |
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 |
| EP1590468A2 (en) | 2003-01-28 | 2005-11-02 | Cellectis | Use of meganucleases for inducing homologous recombination ex vivo and in toto in vertebrate somatic tissues and application thereof |
| WO2006097854A1 (en) | 2005-03-15 | 2006-09-21 | Cellectis | Heterodimeric meganucleases and use thereof |
| WO2009006297A2 (en) | 2007-06-29 | 2009-01-08 | 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 |
| EP2660317B1 (en) | 2007-10-31 | 2016-04-06 | 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 |
| DK2313498T3 (en) | 2008-07-14 | 2017-05-22 | Prec Biosciences Inc | RECOGNITION SEQUENCES FOR I-CREI-DERIVED MECHANUCLEASES AND APPLICATIONS 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 |
| KR20170125406A (ko) * | 2009-08-11 | 2017-11-14 | 상가모 테라퓨틱스, 인코포레이티드 | 표적화 변형에 대한 동형접합성 유기체 |
| WO2011036640A2 (en) | 2009-09-24 | 2011-03-31 | Cellectis | Meganuclease reagents of uses thereof for treating genetic diseases caused by frame shift/non sense mutations |
| US10316304B2 (en) | 2009-11-27 | 2019-06-11 | Basf Plant Science Company Gmbh | Chimeric endonucleases and uses thereof |
| AU2010325549B2 (en) | 2009-11-27 | 2017-04-20 | 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 |
| CN103097527A (zh) | 2010-02-26 | 2013-05-08 | 塞勒克提斯公司 | 用于将转基因插入安全港座位的内切核酸酶的用途 |
| CA2807270A1 (en) | 2010-08-02 | 2012-02-09 | Cellectis | Method for targeted genomic events in diatoms |
| US9044492B2 (en) | 2011-02-04 | 2015-06-02 | Cellectis Sa | Method for modulating the efficiency of double-strand break-induced mutagenesis |
| EP2694091B1 (en) | 2011-04-05 | 2019-03-13 | 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 |
| CN103620027B (zh) | 2011-06-10 | 2017-11-21 | 巴斯夫植物科学有限公司 | 核酸酶融合蛋白及其用途 |
| EP2612918A1 (en) | 2012-01-06 | 2013-07-10 | BASF Plant Science Company GmbH | In planta recombination |
| WO2014018601A2 (en) | 2012-07-24 | 2014-01-30 | Cellectis | New modular base-specific nucleic acid binding domains from burkholderia rhizoxinica proteins |
| AU2013346504A1 (en) | 2012-11-16 | 2015-06-18 | Cellectis | Method for targeted modification of algae genomes |
| EP4234696A3 (en) * | 2012-12-12 | 2023-09-06 | The Broad Institute Inc. | Crispr-cas component systems, methods and compositions for sequence manipulation |
| 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 |
| EP3434776A1 (en) | 2012-12-12 | 2019-01-30 | 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 |
| KR20150105956A (ko) | 2012-12-12 | 2015-09-18 | 더 브로드 인스티튜트, 인코퍼레이티드 | 서열 조작 및 치료적 적용을 위한 시스템, 방법 및 조성물의 전달, 유전자 조작 및 최적화 |
| WO2014102688A1 (en) | 2012-12-27 | 2014-07-03 | Cellectis | New design matrix for improvement of homology-directed gene targeting |
| KR102874079B1 (ko) * | 2013-03-15 | 2025-10-22 | 더 제너럴 하스피탈 코포레이션 | Rna-안내 게놈 편집을 위해 특이성을 증가시키기 위한 절단된 안내 rna(tru-grnas)의 이용 |
| CN107995927B (zh) | 2013-06-17 | 2021-07-30 | 布罗德研究所有限公司 | 用于肝靶向和治疗的crispr-cas系统、载体和组合物的递送与用途 |
| EP3725885A1 (en) | 2013-06-17 | 2020-10-21 | The Broad Institute, Inc. | Functional genomics using crispr-cas systems, compositions methods, screens and applications thereof |
| EP3674411A1 (en) | 2013-06-17 | 2020-07-01 | The Broad Institute, Inc. | Delivery, engineering and optimization of tandem guide systems, methods and compositions for sequence manipulation |
| CA2915795C (en) | 2013-06-17 | 2021-07-13 | The Broad Institute, Inc. | Delivery, use and therapeutic applications of the crispr-cas systems and compositions for targeting disorders and diseases using viral components |
| 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 |
| EP3540051B1 (en) | 2013-12-12 | 2022-08-17 | The Broad Institute, Inc. | Delivery, use and therapeutic applications of the crispr-cas systems and compositions for hsv and viral diseases and disorders. |
| WO2015089364A1 (en) | 2013-12-12 | 2015-06-18 | The Broad Institute Inc. | Crystal structure of a crispr-cas system, and uses thereof |
| BR112016013201B1 (pt) | 2013-12-12 | 2023-01-31 | The Broad Institute, Inc. | Uso de uma composição compreendendo um sistema crispr-cas no tratamento de uma doença genética ocular |
| EP3080271B1 (en) | 2013-12-12 | 2020-02-12 | The Broad Institute, Inc. | Systems, methods and compositions for sequence manipulation with optimized functional crispr-cas systems |
| 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 |
| SG10201804973TA (en) | 2013-12-12 | 2018-07-30 | Broad Inst Inc | Compositions and Methods of Use of Crispr-Cas Systems in Nucleotide Repeat Disorders |
| EP3889260A1 (en) | 2014-12-12 | 2021-10-06 | The Broad Institute, Inc. | Protected guide rnas (pgrnas) |
| EP3237615B2 (en) | 2014-12-24 | 2023-07-26 | The Broad Institute, Inc. | 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 |
| CN109536474A (zh) | 2015-06-18 | 2019-03-29 | 布罗德研究所有限公司 | 降低脱靶效应的crispr酶突变 |
| 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 |
| 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 |
| 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 |
|---|---|---|---|---|
| WO2003078619A1 (en) * | 2002-03-15 | 2003-09-25 | Cellectis | Hybrid and single chain meganucleases and use thereof |
| WO2004031346A2 (en) * | 2002-09-06 | 2004-04-15 | Fred Hutchinson Cancer Research Center | Methods and compositions concerning designed highly-specific nucleic acid binding proteins |
| EP1590468A2 (en) * | 2003-01-28 | 2005-11-02 | Cellectis | Use of meganucleases for inducing homologous recombination ex vivo and in toto in vertebrate somatic tissues and application thereof |
| WO2006097854A1 (en) * | 2005-03-15 | 2006-09-21 | Cellectis | Heterodimeric meganucleases and use thereof |
| ES2626025T3 (es) * | 2005-10-18 | 2017-07-21 | 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 CA CA002677177A patent/CA2677177A1/en not_active Abandoned
- 2008-01-31 EP EP11193586A patent/EP2433641A1/en not_active Withdrawn
- 2008-01-31 JP JP2009547783A patent/JP2010517524A/ja active Pending
- 2008-01-31 CN CN200880006978A patent/CN101678087A/zh active Pending
- 2008-01-31 WO PCT/IB2008/001333 patent/WO2008093249A2/en not_active Ceased
- 2008-01-31 US US12/449,329 patent/US20100167357A1/en not_active Abandoned
- 2008-01-31 AU AU2008211635A patent/AU2008211635A1/en not_active Abandoned
- 2008-01-31 EP EP08751043.4A patent/EP2121004B1/en not_active Not-in-force
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