JP2020532967A5 - - Google Patents
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- JP2020532967A5 JP2020532967A5 JP2020511253A JP2020511253A JP2020532967A5 JP 2020532967 A5 JP2020532967 A5 JP 2020532967A5 JP 2020511253 A JP2020511253 A JP 2020511253A JP 2020511253 A JP2020511253 A JP 2020511253A JP 2020532967 A5 JP2020532967 A5 JP 2020532967A5
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- 230000035772 mutation Effects 0.000 claims 12
- 102000004169 proteins and genes Human genes 0.000 claims 12
- 108090000623 proteins and genes Proteins 0.000 claims 12
- 210000004027 cell Anatomy 0.000 claims 10
- 108020004414 DNA Proteins 0.000 claims 8
- 108020001507 fusion proteins Proteins 0.000 claims 7
- 102000037865 fusion proteins Human genes 0.000 claims 7
- 238000000034 method Methods 0.000 claims 6
- 102000053602 DNA Human genes 0.000 claims 5
- 102000004190 Enzymes Human genes 0.000 claims 5
- 108090000790 Enzymes Proteins 0.000 claims 5
- 101710169336 5'-deoxyadenosine deaminase Proteins 0.000 claims 4
- 102000055025 Adenosine deaminases Human genes 0.000 claims 4
- 102100026846 Cytidine deaminase Human genes 0.000 claims 4
- 108010031325 Cytidine deaminase Proteins 0.000 claims 4
- 102000039446 nucleic acids Human genes 0.000 claims 4
- 108020004707 nucleic acids Proteins 0.000 claims 4
- 150000007523 nucleic acids Chemical class 0.000 claims 4
- 101710142939 Adenosine deaminase 1 Proteins 0.000 claims 2
- 108091033409 CRISPR Proteins 0.000 claims 2
- 102000000311 Cytosine Deaminase Human genes 0.000 claims 2
- 108010080611 Cytosine Deaminase Proteins 0.000 claims 2
- 108020005004 Guide RNA Proteins 0.000 claims 2
- 101710160287 Heterochromatin protein 1 Proteins 0.000 claims 2
- 108010033040 Histones Proteins 0.000 claims 2
- 102000016397 Methyltransferase Human genes 0.000 claims 2
- 108060004795 Methyltransferase Proteins 0.000 claims 2
- 241000193996 Streptococcus pyogenes Species 0.000 claims 2
- 230000000295 complement effect Effects 0.000 claims 2
- 230000029279 positive regulation of transcription, DNA-dependent Effects 0.000 claims 2
- 230000003584 silencer Effects 0.000 claims 2
- 101710199622 tRNA-specific adenosine deaminase Proteins 0.000 claims 2
- 238000013518 transcription Methods 0.000 claims 2
- 230000035897 transcription Effects 0.000 claims 2
- 230000037426 transcriptional repression Effects 0.000 claims 2
- 108091032973 (ribonucleotides)n+m Proteins 0.000 claims 1
- 108010029988 AICDA (activation-induced cytidine deaminase) Proteins 0.000 claims 1
- 108010079649 APOBEC-1 Deaminase Proteins 0.000 claims 1
- 108010004483 APOBEC-3G Deaminase Proteins 0.000 claims 1
- 102100036664 Adenosine deaminase Human genes 0.000 claims 1
- 102100025976 Adenosine deaminase 2 Human genes 0.000 claims 1
- 102100040397 C->U-editing enzyme APOBEC-1 Human genes 0.000 claims 1
- 102100040399 C->U-editing enzyme APOBEC-2 Human genes 0.000 claims 1
- 108010051109 Cell-Penetrating Peptides Proteins 0.000 claims 1
- 102000020313 Cell-Penetrating Peptides Human genes 0.000 claims 1
- 102220605874 Cytosolic arginine sensor for mTORC1 subunit 2_D10A_mutation Human genes 0.000 claims 1
- 102100040263 DNA dC->dU-editing enzyme APOBEC-3A Human genes 0.000 claims 1
- 102100040262 DNA dC->dU-editing enzyme APOBEC-3B Human genes 0.000 claims 1
- 102100040261 DNA dC->dU-editing enzyme APOBEC-3C Human genes 0.000 claims 1
- 102100040264 DNA dC->dU-editing enzyme APOBEC-3D Human genes 0.000 claims 1
- 102100040266 DNA dC->dU-editing enzyme APOBEC-3F Human genes 0.000 claims 1
- 102100038076 DNA dC->dU-editing enzyme APOBEC-3G Human genes 0.000 claims 1
- 102100029791 Double-stranded RNA-specific adenosine deaminase Human genes 0.000 claims 1
- 102100038191 Double-stranded RNA-specific editase 1 Human genes 0.000 claims 1
- 102100024692 Double-stranded RNA-specific editase B2 Human genes 0.000 claims 1
- 102220502946 Geranylgeranyl transferase type-2 subunit alpha_D10N_mutation Human genes 0.000 claims 1
- 108010068250 Herpes Simplex Virus Protein Vmw65 Proteins 0.000 claims 1
- 101000720051 Homo sapiens Adenosine deaminase 2 Proteins 0.000 claims 1
- 101000964322 Homo sapiens C->U-editing enzyme APOBEC-2 Proteins 0.000 claims 1
- 101000964378 Homo sapiens DNA dC->dU-editing enzyme APOBEC-3A Proteins 0.000 claims 1
- 101000964385 Homo sapiens DNA dC->dU-editing enzyme APOBEC-3B Proteins 0.000 claims 1
- 101000964383 Homo sapiens DNA dC->dU-editing enzyme APOBEC-3C Proteins 0.000 claims 1
- 101000964382 Homo sapiens DNA dC->dU-editing enzyme APOBEC-3D Proteins 0.000 claims 1
- 101000964377 Homo sapiens DNA dC->dU-editing enzyme APOBEC-3F Proteins 0.000 claims 1
- 101000865408 Homo sapiens Double-stranded RNA-specific adenosine deaminase Proteins 0.000 claims 1
- 101000742223 Homo sapiens Double-stranded RNA-specific editase 1 Proteins 0.000 claims 1
- 101000686486 Homo sapiens Double-stranded RNA-specific editase B2 Proteins 0.000 claims 1
- 101000615488 Homo sapiens Methyl-CpG-binding domain protein 2 Proteins 0.000 claims 1
- 101000653360 Homo sapiens Methylcytosine dioxygenase TET1 Proteins 0.000 claims 1
- 101000799048 Homo sapiens Probable inactive tRNA-specific adenosine deaminase-like protein 3 Proteins 0.000 claims 1
- 101000800426 Homo sapiens Putative C->U-editing enzyme APOBEC-4 Proteins 0.000 claims 1
- 101000657352 Homo sapiens Transcriptional adapter 2-alpha Proteins 0.000 claims 1
- 101000799160 Homo sapiens tRNA-specific adenosine deaminase 1 Proteins 0.000 claims 1
- 101000799057 Homo sapiens tRNA-specific adenosine deaminase 2 Proteins 0.000 claims 1
- 102100021299 Methyl-CpG-binding domain protein 2 Human genes 0.000 claims 1
- 102100030819 Methylcytosine dioxygenase TET1 Human genes 0.000 claims 1
- 108010057466 NF-kappa B Proteins 0.000 claims 1
- 102000003945 NF-kappa B Human genes 0.000 claims 1
- 101710141454 Nucleoprotein Proteins 0.000 claims 1
- 102100034006 Probable inactive tRNA-specific adenosine deaminase-like protein 3 Human genes 0.000 claims 1
- 102100033091 Putative C->U-editing enzyme APOBEC-4 Human genes 0.000 claims 1
- 101150023114 RNA1 gene Proteins 0.000 claims 1
- 101710122931 Replication and transcription activator Proteins 0.000 claims 1
- 102000004389 Ribonucleoproteins Human genes 0.000 claims 1
- 108010081734 Ribonucleoproteins Proteins 0.000 claims 1
- 102100022433 Single-stranded DNA cytosine deaminase Human genes 0.000 claims 1
- 102000005747 Transcription Factor RelA Human genes 0.000 claims 1
- 108010031154 Transcription Factor RelA Proteins 0.000 claims 1
- 102000004357 Transferases Human genes 0.000 claims 1
- 108090000992 Transferases Proteins 0.000 claims 1
- -1 VP64 Proteins 0.000 claims 1
- 125000000218 acetic acid group Chemical group C(C)(=O)* 0.000 claims 1
- 125000003275 alpha amino acid group Chemical group 0.000 claims 1
- 230000003197 catalytic effect Effects 0.000 claims 1
- UHDGCWIWMRVCDJ-ZAKLUEHWSA-N cytidine Chemical class O=C1N=C(N)C=CN1[C@H]1[C@H](O)[C@@H](O)[C@H](CO)O1 UHDGCWIWMRVCDJ-ZAKLUEHWSA-N 0.000 claims 1
- 230000000694 effects Effects 0.000 claims 1
- 210000001671 embryonic stem cell Anatomy 0.000 claims 1
- 238000000338 in vitro Methods 0.000 claims 1
- 230000003993 interaction Effects 0.000 claims 1
- 230000017156 mRNA modification Effects 0.000 claims 1
- 210000001161 mammalian embryo Anatomy 0.000 claims 1
- 210000002901 mesenchymal stem cell Anatomy 0.000 claims 1
- 210000001778 pluripotent stem cell Anatomy 0.000 claims 1
- 230000001105 regulatory effect Effects 0.000 claims 1
- 210000000130 stem cell Anatomy 0.000 claims 1
- 102100034113 tRNA-specific adenosine deaminase 1 Human genes 0.000 claims 1
- 102100034045 tRNA-specific adenosine deaminase 2 Human genes 0.000 claims 1
Priority Applications (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2024080681A JP2024112895A (ja) | 2017-08-23 | 2024-05-17 | 変更されたPAM特異性を有する遺伝子操作されたCRISPR-Cas9ヌクレアーゼ |
Applications Claiming Priority (5)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201762549303P | 2017-08-23 | 2017-08-23 | |
| US62/549,303 | 2017-08-23 | ||
| US201862641687P | 2018-03-12 | 2018-03-12 | |
| US62/641,687 | 2018-03-12 | ||
| PCT/US2018/047577 WO2019040650A1 (en) | 2017-08-23 | 2018-08-22 | GENETICALLY MODIFIED CRISPR-CAS9 NUCLEASES HAVING MODIFIED PAM SPECIFICITY |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2024080681A Division JP2024112895A (ja) | 2017-08-23 | 2024-05-17 | 変更されたPAM特異性を有する遺伝子操作されたCRISPR-Cas9ヌクレアーゼ |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2020532967A JP2020532967A (ja) | 2020-11-19 |
| JP2020532967A5 true JP2020532967A5 (enExample) | 2021-09-30 |
| JP7581046B2 JP7581046B2 (ja) | 2024-11-12 |
Family
ID=65439890
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2020511253A Active JP7581046B2 (ja) | 2017-08-23 | 2018-08-22 | 変更されたPAM特異性を有する遺伝子操作されたCRISPR-Cas9ヌクレアーゼ |
| JP2024080681A Pending JP2024112895A (ja) | 2017-08-23 | 2024-05-17 | 変更されたPAM特異性を有する遺伝子操作されたCRISPR-Cas9ヌクレアーゼ |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2024080681A Pending JP2024112895A (ja) | 2017-08-23 | 2024-05-17 | 変更されたPAM特異性を有する遺伝子操作されたCRISPR-Cas9ヌクレアーゼ |
Country Status (8)
| Country | Link |
|---|---|
| US (3) | US11286468B2 (enExample) |
| EP (1) | EP3672612A4 (enExample) |
| JP (2) | JP7581046B2 (enExample) |
| CN (2) | CN111278450B (enExample) |
| AU (4) | AU2018320865B2 (enExample) |
| BR (1) | BR112020003596A2 (enExample) |
| CA (1) | CA3073448A1 (enExample) |
| WO (1) | WO2019040650A1 (enExample) |
Families Citing this family (45)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US9890364B2 (en) | 2012-05-29 | 2018-02-13 | The General Hospital Corporation | TAL-Tet1 fusion proteins and methods of use thereof |
| AU2015280069B2 (en) | 2014-06-23 | 2021-08-12 | The General Hospital Corporation | Genomewide unbiased identification of dsbs evaluated by sequencing (guide-seq) |
| EP3204496A1 (en) | 2014-10-10 | 2017-08-16 | Editas Medicine, Inc. | Compositions and methods for promoting homology directed repair |
| AU2016226077B2 (en) | 2015-03-03 | 2021-12-23 | The General Hospital Corporation | Engineered CRISPR-Cas9 nucleases with altered PAM specificity |
| CA3000816A1 (en) | 2015-09-11 | 2017-03-16 | The General Hospital Corporation | Full interrogation of nuclease dsbs and sequencing (find-seq) |
| AU2017302551B2 (en) | 2016-07-26 | 2023-04-27 | The General Hospital Corporation | Variants of CRISPR from Prevotella and Francisella 1 (Cpf1) |
| EP3555297A1 (en) | 2016-12-19 | 2019-10-23 | Editas Medicine, Inc. | Assessing nuclease cleavage |
| WO2018129368A2 (en) | 2017-01-06 | 2018-07-12 | Editas Medicine, Inc. | Methods of assessing nuclease cleavage |
| WO2018195545A2 (en) | 2017-04-21 | 2018-10-25 | The General Hospital Corporation | Variants of cpf1 (cas12a) with altered pam specificity |
| US11499151B2 (en) | 2017-04-28 | 2022-11-15 | Editas Medicine, Inc. | Methods and systems for analyzing guide RNA molecules |
| CA3063733A1 (en) | 2017-05-25 | 2018-11-29 | The General Hospital Corporation | Base editors with improved precision and specificity |
| CN110997908A (zh) | 2017-06-09 | 2020-04-10 | 爱迪塔斯医药公司 | 工程化的cas9核酸酶 |
| EP3652312A1 (en) | 2017-07-14 | 2020-05-20 | Editas Medicine, Inc. | Systems and methods for targeted integration and genome editing and detection thereof using integrated priming sites |
| WO2019040650A1 (en) | 2017-08-23 | 2019-02-28 | The General Hospital Corporation | GENETICALLY MODIFIED CRISPR-CAS9 NUCLEASES HAVING MODIFIED PAM SPECIFICITY |
| AU2019256287B2 (en) | 2018-04-17 | 2026-02-05 | The General Hospital Corporation | Sensitive in vitro assays for substrate preferences and sites of nucleic acid binding, modifying, and cleaving agents |
| KR102853088B1 (ko) * | 2018-05-11 | 2025-09-02 | 빔 테라퓨틱스, 인크. | 프로그래밍가능한 염기 편집기 시스템을 이용하여 단일염기다형성을 편집하는 방법 |
| WO2019217942A1 (en) | 2018-05-11 | 2019-11-14 | Beam Therapeutics Inc. | Methods of substituting pathogenic amino acids using programmable base editor systems |
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| EP3850088A4 (en) | 2018-09-07 | 2023-07-19 | Beam Therapeutics, Inc. | Compositions and methods for improving base editing |
| KR20210055733A (ko) | 2018-09-07 | 2021-05-17 | 빔 테라퓨틱스, 인크. | 핵염기 편집 시스템을 전달하기 위한 조성물 및 방법 |
| JP7646552B2 (ja) | 2019-01-31 | 2025-03-17 | ビーム セラピューティクス インク. | 低減された非標的脱アミノ化を有する核酸塩基エディターおよび核酸塩基エディターの特徴づけのためのアッセイ |
| WO2020163396A1 (en) | 2019-02-04 | 2020-08-13 | The General Hospital Corporation | Adenine dna base editor variants with reduced off-target rna editing |
| US12600971B2 (en) | 2019-02-13 | 2026-04-14 | Beam Therapeutics Inc. | Modified immune cells having adenosine deaminase base editors for modifying a nucleobase in a target sequence |
| WO2020168135A1 (en) | 2019-02-13 | 2020-08-20 | Beam Therapeutics Inc. | Compositions and methods for treating alpha-1 antitrypsin deficiency |
| CN114026226B (zh) * | 2019-04-26 | 2024-10-18 | 株式会社图尔金 | 靶特异性crispr突变体 |
| CN112266418A (zh) * | 2019-07-08 | 2021-01-26 | 中国科学院微生物研究所 | 改进的基因组编辑系统及其应用 |
| CN117264998A (zh) * | 2019-07-10 | 2023-12-22 | 苏州齐禾生科生物科技有限公司 | 双功能基因组编辑系统及其用途 |
| CN119913134A (zh) * | 2019-08-29 | 2025-05-02 | 比姆医疗股份有限公司 | 用于编辑突变以允许转录或表达的组合物和方法 |
| US20220290121A1 (en) * | 2019-08-30 | 2022-09-15 | The General Hospital Corporation | Combinatorial Adenine and Cytosine DNA Base Editors |
| CA3153563A1 (en) * | 2019-09-09 | 2021-03-18 | Beam Therapeutics Inc. | Novel crispr enzymes, methods, systems and uses thereof |
| US12594301B2 (en) | 2019-09-27 | 2026-04-07 | Beam Therapeutics Inc. | Compositions and methods for treatment of liquid cancers |
| KR102421129B1 (ko) * | 2019-10-14 | 2022-07-15 | 연세대학교 산학협력단 | 신규 프로토스페이서 인접 모티프 서열 및 이를 이용한 세포의 유전체에서 표적 핵산을 변형시키는 방법 |
| EP4093864A4 (en) * | 2020-01-24 | 2024-04-10 | The General Hospital Corporation | Unconstrained genome targeting with near-pamless engineered crispr-cas9 variants |
| WO2021151085A2 (en) | 2020-01-24 | 2021-07-29 | The General Hospital Corporation | Crispr-cas enzymes with enhanced on-target activity |
| BR112022017715A2 (pt) | 2020-03-04 | 2022-11-16 | Flagship Pioneering Innovations Vi Llc | Métodos e composições para modular um genoma |
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| CN112430613A (zh) * | 2020-12-08 | 2021-03-02 | 安徽省农业科学院水稻研究所 | 一种宽编辑范围的SpG基因及其应用 |
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| KR20260037664A (ko) | 2021-09-08 | 2026-03-18 | 플래그쉽 파이어니어링 이노베이션스 브이아이, 엘엘씨 | 게놈을 조절하기 위한 방법 및 조성물 |
| CN116286736B (zh) * | 2022-01-17 | 2026-05-01 | 四川大学 | 一种具有扩大的pam识别范围的融合蛋白及其应用 |
| KR20260094824A (ko) * | 2023-10-25 | 2026-06-23 | 씨에스피씨 종콰이 팔마씨우티컬 테크놀로지 (스자좡) 컴퍼니 리미티드 | 고효율, 저오프타겟 유전자 편집 도구 |
| WO2025158391A2 (en) * | 2024-01-24 | 2025-07-31 | The General Hospital Corporation | Generation of custom crispr-cas9 pam variant enzymes |
| CN118048343B (zh) * | 2024-03-01 | 2025-01-28 | 上海交通大学 | Cas9核酸酶及其制备方法和用途 |
| CN120005854B (zh) * | 2025-04-18 | 2025-08-05 | 广州瑞风生物科技有限公司 | Cas9突变体、基因编辑系统及应用 |
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- 2018-08-22 US US16/109,657 patent/US11286468B2/en active Active
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