JPWO2020014577A5 - - Google Patents
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- Publication number
- JPWO2020014577A5 JPWO2020014577A5 JP2021500866A JP2021500866A JPWO2020014577A5 JP WO2020014577 A5 JPWO2020014577 A5 JP WO2020014577A5 JP 2021500866 A JP2021500866 A JP 2021500866A JP 2021500866 A JP2021500866 A JP 2021500866A JP WO2020014577 A5 JPWO2020014577 A5 JP WO2020014577A5
- Authority
- JP
- Japan
- Prior art keywords
- cas9
- combination
- seq
- mrna
- mutation
- 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
- 108091033409 CRISPR Proteins 0.000 claims description 53
- 108020004999 messenger RNA Proteins 0.000 claims description 21
- 230000035772 mutation Effects 0.000 claims description 17
- 108020004414 DNA Proteins 0.000 claims description 14
- 108091027544 Subgenomic mRNA Proteins 0.000 claims description 13
- 125000003729 nucleotide group Chemical group 0.000 claims description 8
- 102000053602 DNA Human genes 0.000 claims description 6
- 230000007018 DNA scission Effects 0.000 claims description 6
- 108020005004 Guide RNA Proteins 0.000 claims description 6
- 101150038500 cas9 gene Proteins 0.000 claims description 6
- 238000010362 genome editing Methods 0.000 claims description 6
- 108091032973 (ribonucleotides)n+m Proteins 0.000 claims description 5
- 210000004027 cell Anatomy 0.000 claims description 5
- 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 4
- 108010042407 Endonucleases Proteins 0.000 claims description 4
- 102000004533 Endonucleases Human genes 0.000 claims description 4
- 230000004927 fusion Effects 0.000 claims description 4
- 108090000623 proteins and genes Proteins 0.000 claims description 4
- 101150023320 B16R gene Proteins 0.000 claims description 3
- 238000010354 CRISPR gene editing Methods 0.000 claims description 3
- 101100316831 Vaccinia virus (strain Copenhagen) B18R gene Proteins 0.000 claims description 3
- 101100004099 Vaccinia virus (strain Western Reserve) VACWR200 gene Proteins 0.000 claims description 3
- LRSASMSXMSNRBT-UHFFFAOYSA-N 5-methylcytosine Chemical compound CC1=CNC(=O)N=C1N LRSASMSXMSNRBT-UHFFFAOYSA-N 0.000 claims description 2
- 108091023037 Aptamer Proteins 0.000 claims description 2
- 102000004190 Enzymes Human genes 0.000 claims description 2
- 108090000790 Enzymes Proteins 0.000 claims description 2
- 101710163270 Nuclease Proteins 0.000 claims description 2
- 108091036407 Polyadenylation Proteins 0.000 claims description 2
- 108020004682 Single-Stranded DNA Proteins 0.000 claims description 2
- 108091006090 chromatin-associated proteins Proteins 0.000 claims description 2
- 230000001973 epigenetic effect Effects 0.000 claims description 2
- 210000003527 eukaryotic cell Anatomy 0.000 claims description 2
- ZEMGGZBWXRYJHK-UHFFFAOYSA-N thiouracil Chemical compound O=C1C=CNC(=S)N1 ZEMGGZBWXRYJHK-UHFFFAOYSA-N 0.000 claims description 2
- 238000001890 transfection Methods 0.000 claims description 2
- 239000003550 marker Substances 0.000 claims 1
- 238000000034 method Methods 0.000 description 25
- 238000012986 modification Methods 0.000 description 1
- 230000004048 modification Effects 0.000 description 1
- 230000008685 targeting Effects 0.000 description 1
Priority Applications (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2024135748A JP2024164090A (ja) | 2018-07-13 | 2024-08-15 | 遺伝子編集の高特異性を達成する方法 |
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201862697955P | 2018-07-13 | 2018-07-13 | |
| US62/697,955 | 2018-07-13 | ||
| PCT/US2019/041551 WO2020014577A1 (en) | 2018-07-13 | 2019-07-12 | Methods of achieving high specificity of genome editing |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2024135748A Division JP2024164090A (ja) | 2018-07-13 | 2024-08-15 | 遺伝子編集の高特異性を達成する方法 |
Publications (4)
| Publication Number | Publication Date |
|---|---|
| JP2021530988A JP2021530988A (ja) | 2021-11-18 |
| JP2021530988A5 JP2021530988A5 (https=) | 2022-07-19 |
| JPWO2020014577A5 true JPWO2020014577A5 (https=) | 2022-07-19 |
| JP7590952B2 JP7590952B2 (ja) | 2024-11-27 |
Family
ID=69141767
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2021500866A Active JP7590952B2 (ja) | 2018-07-13 | 2019-07-12 | 遺伝子編集の高特異性を達成する方法 |
| JP2024135748A Pending JP2024164090A (ja) | 2018-07-13 | 2024-08-15 | 遺伝子編集の高特異性を達成する方法 |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2024135748A Pending JP2024164090A (ja) | 2018-07-13 | 2024-08-15 | 遺伝子編集の高特異性を達成する方法 |
Country Status (7)
| Country | Link |
|---|---|
| US (1) | US20220195403A1 (https=) |
| EP (1) | EP3820503A4 (https=) |
| JP (2) | JP7590952B2 (https=) |
| KR (1) | KR20210031482A (https=) |
| CA (1) | CA3106162A1 (https=) |
| TW (1) | TW202023605A (https=) |
| WO (1) | WO2020014577A1 (https=) |
Families Citing this family (18)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| EP3204496A1 (en) | 2014-10-10 | 2017-08-16 | Editas Medicine, Inc. | Compositions and methods for promoting homology directed repair |
| US11866726B2 (en) | 2017-07-14 | 2024-01-09 | Editas Medicine, Inc. | Systems and methods for targeted integration and genome editing and detection thereof using integrated priming sites |
| EP3823633A4 (en) | 2018-06-29 | 2023-05-03 | Editas Medicine, Inc. | SYNTHETIC LEAD MOLECULES, COMPOSITIONS AND METHODS RELATED THERETO |
| US12521451B2 (en) | 2019-11-08 | 2026-01-13 | Regeneron Pharmaceuticals, Inc. | CRISPR and AAV strategies for x-linked juvenile retinoschisis therapy |
| KR20240099393A (ko) | 2021-11-01 | 2024-06-28 | 톰 바이오사이언시스, 인코포레이티드 | 유전자 편집 기구와 핵산 카고의 동시 전달을 위한 단일 작제물 플랫폼 |
| WO2023122764A1 (en) | 2021-12-22 | 2023-06-29 | Tome Biosciences, Inc. | Co-delivery of a gene editor construct and a donor template |
| TW202346588A (zh) * | 2022-03-04 | 2023-12-01 | 大陸商益杰立科（上海）生物科技有限公司 | 基因組編輯的組成物和方法 |
| WO2023205744A1 (en) | 2022-04-20 | 2023-10-26 | Tome Biosciences, Inc. | Programmable gene insertion compositions |
| WO2023215831A1 (en) | 2022-05-04 | 2023-11-09 | Tome Biosciences, Inc. | Guide rna compositions for programmable gene insertion |
| WO2023225670A2 (en) | 2022-05-20 | 2023-11-23 | Tome Biosciences, Inc. | Ex vivo programmable gene insertion |
| WO2024020587A2 (en) | 2022-07-22 | 2024-01-25 | Tome Biosciences, Inc. | Pleiopluripotent stem cell programmable gene insertion |
| CN115312122B (zh) * | 2022-10-12 | 2022-12-16 | 之江实验室 | 一种CRISPR-Cas酶可突变位点推荐方法和装置 |
| WO2024138194A1 (en) | 2022-12-22 | 2024-06-27 | Tome Biosciences, Inc. | Platforms, compositions, and methods for in vivo programmable gene insertion |
| WO2024234006A1 (en) | 2023-05-11 | 2024-11-14 | Tome Biosciences, Inc. | Systems, compositions, and methods for targeting liver sinusodial endothelial cells (lsecs) |
| IL324634A (en) | 2023-05-15 | 2026-01-01 | Nchroma Bio Inc | Compositions and methods for epigenetic regulation of hbv gene expression |
| WO2025050069A1 (en) | 2023-09-01 | 2025-03-06 | Tome Biosciences, Inc. | Programmable gene insertion using engineered integration enzymes |
| EP4677108A1 (en) | 2024-04-22 | 2026-01-14 | Basecamp Research Ltd | Method and compositions for detecting off-target editing |
| WO2025224182A2 (en) | 2024-04-23 | 2025-10-30 | Basecamp Research Ltd | Single construct platform for simultaneous delivery of gene editing machinery and nucleic acid cargo |
Family Cites Families (12)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US9862926B2 (en) * | 2011-06-27 | 2018-01-09 | Cellscript, Llc. | Inhibition of innate immune response |
| US9234213B2 (en) * | 2013-03-15 | 2016-01-12 | System Biosciences, Llc | Compositions and methods directed to CRISPR/Cas genomic engineering systems |
| EP3011030B1 (en) * | 2013-06-17 | 2023-11-08 | The Broad Institute, Inc. | Optimized crispr-cas double nickase systems, methods and compositions for sequence manipulation |
| SMT202100691T1 (it) * | 2013-07-11 | 2022-01-10 | Modernatx Inc | Composizioni 5 comprendenti polinucleotidi sintetici che codificano proteine correlate a crispr e sgrna sintetici e metodi d'uso |
| US9340799B2 (en) * | 2013-09-06 | 2016-05-17 | President And Fellows Of Harvard College | MRNA-sensing switchable gRNAs |
| US10900034B2 (en) * | 2014-12-03 | 2021-01-26 | Agilent Technologies, Inc. | Guide RNA with chemical modifications |
| EP3233880A1 (en) * | 2014-12-16 | 2017-10-25 | Novartis AG | End capped nucleic acid molecules |
| CN104611368B (zh) * | 2015-01-15 | 2018-05-25 | 中国科学院广州生物医药与健康研究院 | 重组后不产生移码突变的载体、在爪蛙基因组中进行基因定点敲入的方法及应用 |
| US20180112213A1 (en) * | 2015-03-25 | 2018-04-26 | Editas Medicine, Inc. | Crispr/cas-related methods, compositions and components |
| US10188750B1 (en) * | 2015-10-23 | 2019-01-29 | University Of South Florida | Self-replicating cell selective gene delivery compositions, methods, and uses thereof |
| WO2017160662A1 (en) * | 2016-03-12 | 2017-09-21 | The Regents Of The University Of California | Biodegradable vectors for efficient rna delivery |
| EP4286523A3 (en) * | 2016-12-20 | 2024-03-20 | Bristol-Myers Squibb Company | Methods for increasing the efficiency of homology directed repair (hdr) in the cellular genome |
-
2019
- 2019-07-12 US US17/259,998 patent/US20220195403A1/en active Pending
- 2019-07-12 CA CA3106162A patent/CA3106162A1/en active Pending
- 2019-07-12 TW TW108124751A patent/TW202023605A/zh unknown
- 2019-07-12 JP JP2021500866A patent/JP7590952B2/ja active Active
- 2019-07-12 WO PCT/US2019/041551 patent/WO2020014577A1/en not_active Ceased
- 2019-07-12 KR KR1020217003832A patent/KR20210031482A/ko active Pending
- 2019-07-12 EP EP19834647.0A patent/EP3820503A4/en active Pending
-
2024
- 2024-08-15 JP JP2024135748A patent/JP2024164090A/ja active Pending
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