JP2017520243A5 - - Google Patents
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- JP2017520243A5 JP2017520243A5 JP2016571271A JP2016571271A JP2017520243A5 JP 2017520243 A5 JP2017520243 A5 JP 2017520243A5 JP 2016571271 A JP2016571271 A JP 2016571271A JP 2016571271 A JP2016571271 A JP 2016571271A JP 2017520243 A5 JP2017520243 A5 JP 2017520243A5
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- JP
- Japan
- Prior art keywords
- nuclease
- cell
- polynucleotide
- nuclease agent
- nucleic 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.)
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- 238000000034 method Methods 0.000 claims description 85
- 210000004027 cell Anatomy 0.000 claims description 60
- 239000003550 marker Substances 0.000 claims description 60
- 150000007523 nucleic acids Chemical class 0.000 claims description 51
- 108020005004 Guide RNA Proteins 0.000 claims description 41
- 102000039446 nucleic acids Human genes 0.000 claims description 36
- 108020004707 nucleic acids Proteins 0.000 claims description 36
- 230000000694 effects Effects 0.000 claims description 19
- 108090000623 proteins and genes Proteins 0.000 claims description 19
- 230000008685 targeting Effects 0.000 claims description 19
- 238000003780 insertion Methods 0.000 claims description 18
- 230000037431 insertion Effects 0.000 claims description 18
- 108091033409 CRISPR Proteins 0.000 claims description 14
- 229930193140 Neomycin Natural products 0.000 claims description 14
- 229960004927 neomycin Drugs 0.000 claims description 14
- YQYJSBFKSSDGFO-UHFFFAOYSA-N Epihygromycin Natural products OC1C(O)C(C(=O)C)OC1OC(C(=C1)O)=CC=C1C=C(C)C(=O)NC1C(O)C(O)C2OCOC2C1O YQYJSBFKSSDGFO-UHFFFAOYSA-N 0.000 claims description 12
- 239000002773 nucleotide Substances 0.000 claims description 12
- 125000003729 nucleotide group Chemical group 0.000 claims description 12
- 102000004169 proteins and genes Human genes 0.000 claims description 10
- 210000004962 mammalian cell Anatomy 0.000 claims description 7
- 230000005782 double-strand break Effects 0.000 claims description 6
- 230000001939 inductive effect Effects 0.000 claims description 5
- 230000001105 regulatory effect Effects 0.000 claims description 5
- 101000634835 Homo sapiens M1-specific T cell receptor alpha chain Proteins 0.000 claims description 4
- 101000634836 Homo sapiens T cell receptor alpha chain MC.7.G5 Proteins 0.000 claims description 4
- 102000018251 Hypoxanthine Phosphoribosyltransferase Human genes 0.000 claims description 4
- 108010091358 Hypoxanthine Phosphoribosyltransferase Proteins 0.000 claims description 4
- 102100029450 M1-specific T cell receptor alpha chain Human genes 0.000 claims description 4
- 230000003115 biocidal effect Effects 0.000 claims description 4
- 238000012258 culturing Methods 0.000 claims description 4
- 210000003527 eukaryotic cell Anatomy 0.000 claims description 4
- RXWNCPJZOCPEPQ-NVWDDTSBSA-N puromycin Chemical compound C1=CC(OC)=CC=C1C[C@H](N)C(=O)N[C@H]1[C@@H](O)[C@H](N2C3=NC=NC(=C3N=C2)N(C)C)O[C@@H]1CO RXWNCPJZOCPEPQ-NVWDDTSBSA-N 0.000 claims description 4
- 241000283984 Rodentia Species 0.000 claims description 3
- 108091079001 CRISPR RNA Proteins 0.000 claims description 2
- 241000700584 Simplexvirus Species 0.000 claims description 2
- 102000006601 Thymidine Kinase Human genes 0.000 claims description 2
- 108020004440 Thymidine kinase Proteins 0.000 claims description 2
- 229930189065 blasticidin Natural products 0.000 claims description 2
- 210000002950 fibroblast Anatomy 0.000 claims description 2
- BRZYSWJRSDMWLG-CAXSIQPQSA-N geneticin Natural products O1C[C@@](O)(C)[C@H](NC)[C@@H](O)[C@H]1O[C@@H]1[C@@H](O)[C@H](O[C@@H]2[C@@H]([C@@H](O)[C@H](O)[C@@H](C(C)O)O2)N)[C@@H](N)C[C@H]1N BRZYSWJRSDMWLG-CAXSIQPQSA-N 0.000 claims description 2
- 210000003958 hematopoietic stem cell Anatomy 0.000 claims description 2
- 210000001178 neural stem cell Anatomy 0.000 claims description 2
- 229950010131 puromycin Drugs 0.000 claims description 2
- 101710163270 Nuclease Proteins 0.000 claims 71
- 239000003795 chemical substances by application Substances 0.000 claims 46
- 108091033319 polynucleotide Proteins 0.000 claims 38
- 102000040430 polynucleotide Human genes 0.000 claims 38
- 239000002157 polynucleotide Substances 0.000 claims 38
- 108091028043 Nucleic acid sequence Proteins 0.000 claims 15
- 108010017070 Zinc Finger Nucleases Proteins 0.000 claims 8
- 108091008874 T cell receptors Proteins 0.000 claims 5
- 102000016266 T-Cell Antigen Receptors Human genes 0.000 claims 5
- 238000012986 modification Methods 0.000 claims 4
- 230000004048 modification Effects 0.000 claims 4
- 210000001161 mammalian embryo Anatomy 0.000 claims 3
- 108091032973 (ribonucleotides)n+m Proteins 0.000 claims 2
- 238000010354 CRISPR gene editing Methods 0.000 claims 2
- 108091026890 Coding region Proteins 0.000 claims 2
- 108060003951 Immunoglobulin Proteins 0.000 claims 2
- 108700026244 Open Reading Frames Proteins 0.000 claims 2
- 125000003275 alpha amino acid group Chemical group 0.000 claims 2
- 238000003556 assay Methods 0.000 claims 2
- 239000003623 enhancer Substances 0.000 claims 2
- 102000018358 immunoglobulin Human genes 0.000 claims 2
- 238000010348 incorporation Methods 0.000 claims 2
- 230000035772 mutation Effects 0.000 claims 2
- 125000006850 spacer group Chemical group 0.000 claims 2
- 108700028369 Alleles Proteins 0.000 claims 1
- 101000998953 Homo sapiens Immunoglobulin heavy variable 1-2 Proteins 0.000 claims 1
- 101001008255 Homo sapiens Immunoglobulin kappa variable 1D-8 Proteins 0.000 claims 1
- 101001047628 Homo sapiens Immunoglobulin kappa variable 2-29 Proteins 0.000 claims 1
- 101001008321 Homo sapiens Immunoglobulin kappa variable 2D-26 Proteins 0.000 claims 1
- 101001047619 Homo sapiens Immunoglobulin kappa variable 3-20 Proteins 0.000 claims 1
- 101001008263 Homo sapiens Immunoglobulin kappa variable 3D-15 Proteins 0.000 claims 1
- 102000006496 Immunoglobulin Heavy Chains Human genes 0.000 claims 1
- 108010019476 Immunoglobulin Heavy Chains Proteins 0.000 claims 1
- 102100036887 Immunoglobulin heavy variable 1-2 Human genes 0.000 claims 1
- 102100022949 Immunoglobulin kappa variable 2-29 Human genes 0.000 claims 1
- HCHKCACWOHOZIP-UHFFFAOYSA-N Zinc Chemical group [Zn] HCHKCACWOHOZIP-UHFFFAOYSA-N 0.000 claims 1
- 201000010099 disease Diseases 0.000 claims 1
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 claims 1
- 239000012636 effector Substances 0.000 claims 1
- 210000004263 induced pluripotent stem cell Anatomy 0.000 claims 1
- 108020004999 messenger RNA Proteins 0.000 claims 1
- 210000000472 morula Anatomy 0.000 claims 1
- 231100000331 toxic Toxicity 0.000 claims 1
- 230000002588 toxic effect Effects 0.000 claims 1
- 230000002103 transcriptional effect Effects 0.000 claims 1
- 239000011701 zinc Substances 0.000 claims 1
- 229910052725 zinc Inorganic materials 0.000 claims 1
- 241000699800 Cricetinae Species 0.000 description 1
- 230000006801 homologous recombination Effects 0.000 description 1
- 238000002744 homologous recombination Methods 0.000 description 1
- 230000003834 intracellular effect Effects 0.000 description 1
- 210000001236 prokaryotic cell Anatomy 0.000 description 1
Applications Claiming Priority (5)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201462008832P | 2014-06-06 | 2014-06-06 | |
| US62/008,832 | 2014-06-06 | ||
| US201462017916P | 2014-06-27 | 2014-06-27 | |
| US62/017,916 | 2014-06-27 | ||
| PCT/US2015/034503 WO2015188109A1 (en) | 2014-06-06 | 2015-06-05 | Methods and compositions for modifying a targeted locus |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2020067276A Division JP7154248B2 (ja) | 2014-06-06 | 2020-04-03 | 標的遺伝子座を修飾するための方法及び組成物 |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2017520243A JP2017520243A (ja) | 2017-07-27 |
| JP2017520243A5 true JP2017520243A5 (enExample) | 2018-06-28 |
| JP6688231B2 JP6688231B2 (ja) | 2020-04-28 |
Family
ID=53404960
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2016571271A Active JP6688231B2 (ja) | 2014-06-06 | 2015-06-05 | 標的遺伝子座を修飾するための方法及び組成物 |
| JP2020067276A Active JP7154248B2 (ja) | 2014-06-06 | 2020-04-03 | 標的遺伝子座を修飾するための方法及び組成物 |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2020067276A Active JP7154248B2 (ja) | 2014-06-06 | 2020-04-03 | 標的遺伝子座を修飾するための方法及び組成物 |
Country Status (25)
| Country | Link |
|---|---|
| US (4) | US10106820B2 (enExample) |
| EP (2) | EP3708671B1 (enExample) |
| JP (2) | JP6688231B2 (enExample) |
| KR (1) | KR102374379B1 (enExample) |
| CN (2) | CN106795521B (enExample) |
| AU (1) | AU2015269187B2 (enExample) |
| BR (1) | BR112016028564A2 (enExample) |
| CA (1) | CA2950173C (enExample) |
| CY (1) | CY1122897T1 (enExample) |
| DK (1) | DK3152312T3 (enExample) |
| ES (2) | ES2784754T3 (enExample) |
| HR (1) | HRP20200529T1 (enExample) |
| HU (1) | HUE049776T2 (enExample) |
| IL (1) | IL249042B (enExample) |
| LT (1) | LT3152312T (enExample) |
| MX (1) | MX385689B (enExample) |
| NZ (1) | NZ727481A (enExample) |
| PL (1) | PL3152312T3 (enExample) |
| PT (1) | PT3152312T (enExample) |
| RS (1) | RS60359B1 (enExample) |
| RU (1) | RU2704283C9 (enExample) |
| SG (2) | SG11201609634YA (enExample) |
| SI (1) | SI3152312T1 (enExample) |
| SM (1) | SMT202000264T1 (enExample) |
| WO (1) | WO2015188109A1 (enExample) |
Families Citing this family (79)
| 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 |
| JP6275120B2 (ja) | 2012-04-25 | 2018-02-07 | リジェネロン・ファーマシューティカルズ・インコーポレイテッドＲｅｇｅｎｅｒｏｎ Ｐｈａｒｍａｃｅｕｔｉｃａｌｓ， Ｉｎｃ． | 大きい標的化ベクターによるヌクレアーゼ媒介標的化 |
| JP6475172B2 (ja) | 2013-02-20 | 2019-02-27 | リジェネロン・ファーマシューティカルズ・インコーポレイテッドＲｅｇｅｎｅｒｏｎ Ｐｈａｒｍａｃｅｕｔｉｃａｌｓ， Ｉｎｃ． | ラットの遺伝子組換え |
| EP3456831B1 (en) | 2013-04-16 | 2021-07-14 | Regeneron Pharmaceuticals, Inc. | Targeted modification of rat genome |
| 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 |
| US9737604B2 (en) | 2013-09-06 | 2017-08-22 | President And Fellows Of Harvard College | Use of cationic lipids to deliver CAS9 |
| US9340799B2 (en) | 2013-09-06 | 2016-05-17 | President And Fellows Of Harvard College | MRNA-sensing switchable gRNAs |
| MX388127B (es) | 2013-12-11 | 2025-03-19 | Regeneron Pharma | Metodos y composiciones para la modificacion dirigida de un genoma. |
| ES2975317T3 (es) | 2013-12-11 | 2024-07-04 | Regeneron Pharma | Métodos y composiciones para la modificación dirigida de un genoma |
| US11053481B2 (en) | 2013-12-12 | 2021-07-06 | President And Fellows Of Harvard College | Fusions of Cas9 domains and nucleic acid-editing domains |
| BR112016028564A2 (pt) | 2014-06-06 | 2018-01-30 | Regeneron Pharma | método para modificar um locus-alvo em uma célula. |
| SG10201911411YA (en) | 2014-06-26 | 2020-02-27 | Regeneron Pharma | Methods and compositions for targeted genetic modifications and methods of use |
| AU2015298571B2 (en) | 2014-07-30 | 2020-09-03 | President And Fellows Of Harvard College | Cas9 proteins including ligand-dependent inteins |
| CA2963315A1 (en) | 2014-10-15 | 2016-04-21 | Regeneron Pharmaceuticals, Inc. | Methods and compositions for generating or maintaining pluripotent cells |
| CA3176380A1 (en) | 2014-11-21 | 2016-05-26 | Regeneron Pharmaceuticals, Inc. | Methods and compositions for targeted genetic modification using paired guide rnas |
| BR112017013104A2 (pt) | 2014-12-19 | 2018-05-15 | Regeneron Pharma | métodos para modificar um locus genômico alvo em uma célula, para intensificar a recombinação homóloga em um locus genômico alvo em uma célula e para produzir uma geração f0 de um animal não humano. |
| 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 |
| AU2017230011A1 (en) * | 2016-03-11 | 2018-09-27 | 2Seventy Bio, Inc. | Genome edited immune effector cells |
| KR20180132705A (ko) * | 2016-04-04 | 2018-12-12 | 에테하 취리히 | 단백질 생산 및 라이브러리(Library) 생성을 위한 포유동물 세포주 |
| CA3020923A1 (en) | 2016-04-15 | 2017-10-19 | Memorial Sloan Kettering Cancer Center | Transgenic t cell and chimeric antigen receptor t cell compositions and related methods |
| WO2017201476A1 (en) * | 2016-05-20 | 2017-11-23 | Regeneron Pharmaceuticals, Inc. | Methods for breaking immunological tolerance using multiple guide rnas |
| IL263160B2 (en) | 2016-06-03 | 2024-01-01 | Regeneron Pharma | Non-human animals expressing exogenous terminal deoxynucleotidyltransferase |
| CN107513538A (zh) * | 2016-06-17 | 2017-12-26 | 北京大学 | 基因敲除方法 |
| US20180004537A1 (en) | 2016-07-01 | 2018-01-04 | Microsoft Technology Licensing, Llc | Molecular State Machines |
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| 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 |
| US11542509B2 (en) | 2016-08-24 | 2023-01-03 | President And Fellows Of Harvard College | Incorporation of unnatural amino acids into proteins using base editing |
| AU2017342543B2 (en) | 2016-10-14 | 2024-06-27 | President And Fellows Of Harvard College | AAV delivery of nucleobase editors |
| MA53935A (fr) | 2016-11-04 | 2021-12-22 | Regeneron Pharma | Animaux non humains ayant un locus à chaîne légère lambda d'immunoglobuline modifiée |
| WO2018119359A1 (en) | 2016-12-23 | 2018-06-28 | President And Fellows Of Harvard College | Editing of ccr5 receptor gene to protect against hiv infection |
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| TW201839136A (zh) | 2017-02-06 | 2018-11-01 | 瑞士商諾華公司 | 治療血色素異常症之組合物及方法 |
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| EP3592381A1 (en) | 2017-03-09 | 2020-01-15 | President and Fellows of Harvard College | Cancer vaccine |
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| KR20190127797A (ko) | 2017-03-10 | 2019-11-13 | 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 | 시토신에서 구아닌으로의 염기 편집제 |
| CA3056182A1 (en) * | 2017-03-16 | 2018-09-20 | Pfizer Inc. | Tyrosine prototrophy |
| CA3057192A1 (en) | 2017-03-23 | 2018-09-27 | President And Fellows Of Harvard College | Nucleobase editors comprising nucleic acid programmable dna binding proteins |
| 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 |
| 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 |
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| US12435330B2 (en) | 2019-10-10 | 2025-10-07 | The Broad Institute, Inc. | Methods and compositions for prime editing RNA |
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| US20240018493A1 (en) * | 2020-11-10 | 2024-01-18 | The Board Of Trustees Of The Leland Stanford Junior University | Knock-in of large dna for long-term high genomic expression |
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