JP2024038326A5 - - Google Patents
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- JP2024038326A5 JP2024038326A5 JP2024002316A JP2024002316A JP2024038326A5 JP 2024038326 A5 JP2024038326 A5 JP 2024038326A5 JP 2024002316 A JP2024002316 A JP 2024002316A JP 2024002316 A JP2024002316 A JP 2024002316A JP 2024038326 A5 JP2024038326 A5 JP 2024038326A5
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- Prior art keywords
- sequence
- seq
- composition
- mrna
- encoding
- 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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- 125000003729 nucleotide group Chemical group 0.000 claims 58
- 239000002773 nucleotide Substances 0.000 claims 51
- 108090000623 proteins and genes Proteins 0.000 claims 48
- 108091033319 polynucleotide Proteins 0.000 claims 42
- 102000040430 polynucleotide Human genes 0.000 claims 42
- 239000002157 polynucleotide Substances 0.000 claims 42
- 108020004999 messenger RNA Proteins 0.000 claims 37
- 102000004169 proteins and genes Human genes 0.000 claims 34
- 230000004568 DNA-binding Effects 0.000 claims 24
- 125000006850 spacer group Chemical group 0.000 claims 21
- ISAKRJDGNUQOIC-UHFFFAOYSA-N Uracil Chemical compound O=C1C=CNC(=O)N1 ISAKRJDGNUQOIC-UHFFFAOYSA-N 0.000 claims 20
- RWQNBRDOKXIBIV-UHFFFAOYSA-N thymine Chemical compound CC1=CNC(=O)NC1=O RWQNBRDOKXIBIV-UHFFFAOYSA-N 0.000 claims 20
- NYHBQMYGNKIUIF-UUOKFMHZSA-N Guanosine Chemical compound C1=NC=2C(=O)NC(N)=NC=2N1[C@@H]1O[C@H](CO)[C@@H](O)[C@H]1O NYHBQMYGNKIUIF-UUOKFMHZSA-N 0.000 claims 16
- 102000006437 Proprotein Convertases Human genes 0.000 claims 14
- 108010044159 Proprotein Convertases Proteins 0.000 claims 14
- DRTQHJPVMGBUCF-XVFCMESISA-N Uridine Chemical compound O[C@@H]1[C@H](O)[C@@H](CO)O[C@H]1N1C(=O)NC(=O)C=C1 DRTQHJPVMGBUCF-XVFCMESISA-N 0.000 claims 12
- OIRDTQYFTABQOQ-KQYNXXCUSA-N adenosine Chemical compound C1=NC=2C(N)=NC=NC=2N1[C@@H]1O[C@H](CO)[C@@H](O)[C@H]1O OIRDTQYFTABQOQ-KQYNXXCUSA-N 0.000 claims 10
- 229940113082 thymine Drugs 0.000 claims 10
- 229940035893 uracil Drugs 0.000 claims 10
- UHDGCWIWMRVCDJ-UHFFFAOYSA-N 1-beta-D-Xylofuranosyl-NH-Cytosine Natural products O=C1N=C(N)C=CN1C1C(O)C(O)C(CO)O1 UHDGCWIWMRVCDJ-UHFFFAOYSA-N 0.000 claims 8
- MIKUYHXYGGJMLM-GIMIYPNGSA-N Crotonoside Natural products C1=NC2=C(N)NC(=O)N=C2N1[C@H]1O[C@@H](CO)[C@H](O)[C@@H]1O MIKUYHXYGGJMLM-GIMIYPNGSA-N 0.000 claims 8
- UHDGCWIWMRVCDJ-PSQAKQOGSA-N Cytidine Natural products O=C1N=C(N)C=CN1[C@@H]1[C@@H](O)[C@@H](O)[C@H](CO)O1 UHDGCWIWMRVCDJ-PSQAKQOGSA-N 0.000 claims 8
- NYHBQMYGNKIUIF-UHFFFAOYSA-N D-guanosine Natural products C1=2NC(N)=NC(=O)C=2N=CN1C1OC(CO)C(O)C1O NYHBQMYGNKIUIF-UHFFFAOYSA-N 0.000 claims 8
- UHDGCWIWMRVCDJ-ZAKLUEHWSA-N cytidine Chemical compound 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 8
- 229940029575 guanosine Drugs 0.000 claims 8
- 102000039446 nucleic acids Human genes 0.000 claims 8
- 108020004707 nucleic acids Proteins 0.000 claims 8
- 150000007523 nucleic acids Chemical class 0.000 claims 8
- DRTQHJPVMGBUCF-PSQAKQOGSA-N beta-L-uridine Natural products O[C@H]1[C@@H](O)[C@H](CO)O[C@@H]1N1C(=O)NC(=O)C=C1 DRTQHJPVMGBUCF-PSQAKQOGSA-N 0.000 claims 6
- RYYWUUFWQRZTIU-UHFFFAOYSA-K thiophosphate Chemical compound [O-]P([O-])([O-])=S RYYWUUFWQRZTIU-UHFFFAOYSA-K 0.000 claims 6
- DRTQHJPVMGBUCF-UHFFFAOYSA-N uracil arabinoside Natural products OC1C(O)C(CO)OC1N1C(=O)NC(=O)C=C1 DRTQHJPVMGBUCF-UHFFFAOYSA-N 0.000 claims 6
- 229940045145 uridine Drugs 0.000 claims 6
- 239000002126 C01EB10 - Adenosine Substances 0.000 claims 5
- 108091026890 Coding region Proteins 0.000 claims 5
- 229960005305 adenosine Drugs 0.000 claims 5
- 238000010459 TALEN Methods 0.000 claims 4
- IQFYYKKMVGJFEH-XLPZGREQSA-N Thymidine Chemical compound O=C1NC(=O)C(C)=CN1[C@@H]1O[C@H](CO)[C@@H](O)C1 IQFYYKKMVGJFEH-XLPZGREQSA-N 0.000 claims 4
- 108010043645 Transcription Activator-Like Effector Nucleases Proteins 0.000 claims 4
- 108010017070 Zinc Finger Nucleases Proteins 0.000 claims 4
- OPTASPLRGRRNAP-UHFFFAOYSA-N cytosine Chemical compound NC=1C=CNC(=O)N=1 OPTASPLRGRRNAP-UHFFFAOYSA-N 0.000 claims 4
- UYTPUPDQBNUYGX-UHFFFAOYSA-N guanine Chemical compound O=C1NC(N)=NC2=C1N=CN2 UYTPUPDQBNUYGX-UHFFFAOYSA-N 0.000 claims 4
- 108091033409 CRISPR Proteins 0.000 claims 3
- 101710169336 5'-deoxyadenosine deaminase Proteins 0.000 claims 2
- 229930024421 Adenine Natural products 0.000 claims 2
- GFFGJBXGBJISGV-UHFFFAOYSA-N Adenine Chemical compound NC1=NC=NC2=C1N=CN2 GFFGJBXGBJISGV-UHFFFAOYSA-N 0.000 claims 2
- 102000055025 Adenosine deaminases Human genes 0.000 claims 2
- DWRXFEITVBNRMK-UHFFFAOYSA-N Beta-D-1-Arabinofuranosylthymine Natural products O=C1NC(=O)C(C)=CN1C1C(O)C(O)C(CO)O1 DWRXFEITVBNRMK-UHFFFAOYSA-N 0.000 claims 2
- 108700004991 Cas12a Proteins 0.000 claims 2
- 108010008532 Deoxyribonuclease I Proteins 0.000 claims 2
- 102000007260 Deoxyribonuclease I Human genes 0.000 claims 2
- 108091026898 Leader sequence (mRNA) Proteins 0.000 claims 2
- 108020005067 RNA Splice Sites Proteins 0.000 claims 2
- 108091036066 Three prime untranslated region Proteins 0.000 claims 2
- 229960000643 adenine Drugs 0.000 claims 2
- IQFYYKKMVGJFEH-UHFFFAOYSA-N beta-L-thymidine Natural products O=C1NC(=O)C(C)=CN1C1OC(CO)C(O)C1 IQFYYKKMVGJFEH-UHFFFAOYSA-N 0.000 claims 2
- 229940104302 cytosine Drugs 0.000 claims 2
- 238000001802 infusion Methods 0.000 claims 2
- 238000001990 intravenous administration Methods 0.000 claims 2
- 230000030648 nucleus localization Effects 0.000 claims 2
- 229940104230 thymidine Drugs 0.000 claims 2
- 102100026846 Cytidine deaminase Human genes 0.000 claims 1
- 108010031325 Cytidine deaminase Proteins 0.000 claims 1
- 108020005004 Guide RNA Proteins 0.000 claims 1
- 206010045261 Type IIa hyperlipidaemia Diseases 0.000 claims 1
- 238000006243 chemical reaction Methods 0.000 claims 1
Applications Claiming Priority (12)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US202063007803P | 2020-04-09 | 2020-04-09 | |
| US202063007797P | 2020-04-09 | 2020-04-09 | |
| US63/007,803 | 2020-04-09 | ||
| US63/007,797 | 2020-04-09 | ||
| US202063045033P | 2020-06-26 | 2020-06-26 | |
| US202063045032P | 2020-06-26 | 2020-06-26 | |
| US63/045,033 | 2020-06-26 | ||
| US63/045,032 | 2020-06-26 | ||
| US202163136087P | 2021-01-11 | 2021-01-11 | |
| US63/136,087 | 2021-01-11 | ||
| PCT/US2021/026732 WO2021207712A2 (en) | 2020-04-09 | 2021-04-09 | Base editing of pcsk9 and methods of using same for treatment of disease |
| JP2022562027A JP7457832B2 (ja) | 2020-04-09 | 2021-04-09 | Ｐｃｓｋ９の塩基編集および疾患の処置のためにこれを使用する方法 |
Related Parent Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2022562027A Division JP7457832B2 (ja) | 2020-04-09 | 2021-04-09 | Ｐｃｓｋ９の塩基編集および疾患の処置のためにこれを使用する方法 |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2024038326A JP2024038326A (ja) | 2024-03-19 |
| JP2024038326A5 true JP2024038326A5 (enExample) | 2024-12-26 |
Family
ID=78022701
Family Applications (4)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2022562027A Active JP7457832B2 (ja) | 2020-04-09 | 2021-04-09 | Ｐｃｓｋ９の塩基編集および疾患の処置のためにこれを使用する方法 |
| JP2022562030A Active JP7457833B2 (ja) | 2020-04-09 | 2021-04-09 | Ａｎｇｐｔｌ３の塩基編集および疾患の処置のためにこれを使用する方法 |
| JP2024002316A Pending JP2024038326A (ja) | 2020-04-09 | 2024-01-11 | Ｐｃｓｋ９の塩基編集および疾患の処置のためにこれを使用する方法 |
| JP2024002319A Pending JP2024038327A (ja) | 2020-04-09 | 2024-01-11 | Ａｎｇｐｔｌ３の塩基編集および疾患の処置のためにこれを使用する方法 |
Family Applications Before (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2022562027A Active JP7457832B2 (ja) | 2020-04-09 | 2021-04-09 | Ｐｃｓｋ９の塩基編集および疾患の処置のためにこれを使用する方法 |
| JP2022562030A Active JP7457833B2 (ja) | 2020-04-09 | 2021-04-09 | Ａｎｇｐｔｌ３の塩基編集および疾患の処置のためにこれを使用する方法 |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2024002319A Pending JP2024038327A (ja) | 2020-04-09 | 2024-01-11 | Ａｎｇｐｔｌ３の塩基編集および疾患の処置のためにこれを使用する方法 |
Country Status (13)
| Country | Link |
|---|---|
| US (5) | US20230158174A1 (enExample) |
| EP (3) | EP4132591A4 (enExample) |
| JP (4) | JP7457832B2 (enExample) |
| KR (2) | KR20230016176A (enExample) |
| CN (2) | CN116096883A (enExample) |
| AU (4) | AU2021252991B2 (enExample) |
| BR (2) | BR112022020428A2 (enExample) |
| CA (2) | CA3179863A1 (enExample) |
| GB (8) | GB2625902B (enExample) |
| IL (2) | IL297193A (enExample) |
| MX (1) | MX2022012683A (enExample) |
| PH (2) | PH12022553033A1 (enExample) |
| WO (3) | WO2021207711A2 (enExample) |
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| US11920148B2 (en) | 2017-02-22 | 2024-03-05 | Crispr Therapeutics Ag | Compositions and methods for gene editing |
| MX2022012683A (es) | 2020-04-09 | 2023-01-11 | Verve Therapeutics Inc | Edicion de bases de pcsk9 y metodos de uso de la misma para el tratamiento de enfermedades. |
| US11591544B2 (en) | 2020-11-25 | 2023-02-28 | Akagera Medicines, Inc. | Ionizable cationic lipids |
| CA3235827A1 (en) * | 2021-10-21 | 2023-04-27 | Wei Hsi Yeh | Genome editing compositions and methods for treatment of retinitis pigmentosa |
| JP2025507751A (ja) * | 2022-03-01 | 2025-03-21 | クリスパー・セラピューティクス・アクチェンゲゼルシャフト | アンジオポエチン様３（ａｎｇｐｔｌ３）関連状態を処置するための方法および組成物 |
| WO2023180904A1 (en) * | 2022-03-21 | 2023-09-28 | Crispr Therapeutics Ag | Methods and compositions for treating lipoprotein-related diseases |
| KR20250031230A (ko) | 2022-05-25 | 2025-03-06 | 아카제라 메디신즈, 인크. | 핵산 전달을 위한 지질 나노입자 및 이의 사용 방법 |
| EP4314267A1 (en) | 2022-06-07 | 2024-02-07 | Scribe Therapeutics Inc. | Compositions and methods for the targeting of pcsk9 |
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| EP4638748A2 (en) * | 2022-12-21 | 2025-10-29 | Intellia Therapeutics, Inc. | Compositions and methods for proprotein convertase subtilisin kexin 9 (pcsk9) editing |
| WO2024152058A1 (en) * | 2023-01-13 | 2024-07-18 | The Trustees Of The University Of Pennsylvania | Compositions and methods for the treatment of hereditary tyrosinemia type 1 |
| EP4669753A1 (en) * | 2023-02-20 | 2025-12-31 | ProQR Therapeutics II B.V. | Antisense oligonucleotides for the treatment of atherosclerotic cardiovascular disease |
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| CN118813611B (zh) * | 2023-04-19 | 2025-09-16 | 清华大学 | 一种基于rna核酶的dear核酸操纵系统及其应用 |
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| CN117587013B (zh) * | 2023-11-27 | 2024-06-14 | 湖南师范大学 | 一种血管发育异常的斑马鱼模型的构建方法 |
| CN117568313B (zh) * | 2024-01-15 | 2024-04-26 | 上海贝斯昂科生物科技有限公司 | 基因编辑组合物及其用途 |
| CN121022805A (zh) * | 2024-02-26 | 2025-11-28 | 深圳信立泰药业股份有限公司 | 用于pcsk9基因修饰或编辑的组合物及其使用方法 |
| WO2025184520A1 (en) * | 2024-02-29 | 2025-09-04 | Intellia Therapeutics, Inc. | Compositions and methods for angiopoietin like 3 (angptl3) editing |
| JP2025139413A (ja) * | 2024-03-12 | 2025-09-26 | サミー株式会社 | 遊技機 |
| WO2026020120A1 (en) * | 2024-07-19 | 2026-01-22 | University Of Massachusetts | Increasing cas9 genome editing fidelity through attenuation of guide rna watson-crick base pairing potential |
| CN120130446B (zh) * | 2025-03-10 | 2026-01-20 | 苏州系统医学研究所 | 一种制备疾病动物模型的方法及其应用 |
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