JPWO2022028472A5 - - Google Patents
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- Publication number
- JPWO2022028472A5 JPWO2022028472A5 JP2023507927A JP2023507927A JPWO2022028472A5 JP WO2022028472 A5 JPWO2022028472 A5 JP WO2022028472A5 JP 2023507927 A JP2023507927 A JP 2023507927A JP 2023507927 A JP2023507927 A JP 2023507927A JP WO2022028472 A5 JPWO2022028472 A5 JP WO2022028472A5
- Authority
- JP
- Japan
- Prior art keywords
- seq
- nucleic acid
- acid sequence
- hsa
- mir
- 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
- 150000007523 nucleic acids Chemical group 0.000 claims description 255
- 108091028043 Nucleic acid sequence Proteins 0.000 claims description 139
- 108020004707 nucleic acids Proteins 0.000 claims description 106
- 102000039446 nucleic acids Human genes 0.000 claims description 106
- 239000003623 enhancer Substances 0.000 claims description 50
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 claims description 36
- 239000013608 rAAV vector Substances 0.000 claims description 36
- 239000013598 vector Substances 0.000 claims description 33
- 101000575685 Homo sapiens Synembryn-B Proteins 0.000 claims description 30
- 102100026014 Synembryn-B Human genes 0.000 claims description 30
- 239000002679 microRNA Substances 0.000 claims description 28
- 108700011259 MicroRNAs Proteins 0.000 claims description 26
- 108091068990 Homo sapiens miR-133a-1 stem-loop Proteins 0.000 claims description 23
- 102100021947 Survival motor neuron protein Human genes 0.000 claims description 20
- 201000010099 disease Diseases 0.000 claims description 18
- 208000035475 disorder Diseases 0.000 claims description 18
- 108091069016 Homo sapiens miR-122 stem-loop Proteins 0.000 claims description 17
- 108091067643 Homo sapiens miR-208a stem-loop Proteins 0.000 claims description 17
- 108091086543 Homo sapiens miR-208b stem-loop Proteins 0.000 claims description 17
- 108091007780 MiR-122 Proteins 0.000 claims description 17
- 125000003275 alpha amino acid group Chemical group 0.000 claims description 12
- 210000004185 liver Anatomy 0.000 claims description 12
- 239000008194 pharmaceutical composition Substances 0.000 claims description 11
- 239000002245 particle Substances 0.000 claims description 10
- 108091092234 Homo sapiens miR-488 stem-loop Proteins 0.000 claims description 9
- 241001655883 Adeno-associated virus - 1 Species 0.000 claims description 8
- 241000702423 Adeno-associated virus - 2 Species 0.000 claims description 8
- 241000202702 Adeno-associated virus - 3 Species 0.000 claims description 8
- 241000580270 Adeno-associated virus - 4 Species 0.000 claims description 8
- 241001634120 Adeno-associated virus - 5 Species 0.000 claims description 8
- 241000972680 Adeno-associated virus - 6 Species 0.000 claims description 8
- 241001164823 Adeno-associated virus - 7 Species 0.000 claims description 8
- 241001164825 Adeno-associated virus - 8 Species 0.000 claims description 8
- 241000649045 Adeno-associated virus 10 Species 0.000 claims description 8
- 241000649046 Adeno-associated virus 11 Species 0.000 claims description 8
- 241000649047 Adeno-associated virus 12 Species 0.000 claims description 8
- 241000300529 Adeno-associated virus 13 Species 0.000 claims description 8
- 239000013603 viral vector Substances 0.000 claims description 8
- 239000013607 AAV vector Substances 0.000 claims description 6
- 208000002320 spinal muscular atrophy Diseases 0.000 claims description 6
- 230000000747 cardiac effect Effects 0.000 claims description 5
- 108090000565 Capsid Proteins Proteins 0.000 claims description 4
- 102100023321 Ceruloplasmin Human genes 0.000 claims description 4
- 239000002773 nucleotide Substances 0.000 claims description 4
- 125000003729 nucleotide group Chemical group 0.000 claims description 4
- 230000003612 virological effect Effects 0.000 claims description 4
- 208000032225 Proximal spinal muscular atrophy type 1 Diseases 0.000 claims description 2
- 208000033522 Proximal spinal muscular atrophy type 2 Diseases 0.000 claims description 2
- 208000033526 Proximal spinal muscular atrophy type 3 Diseases 0.000 claims description 2
- 208000033550 Proximal spinal muscular atrophy type 4 Diseases 0.000 claims description 2
- 101150081851 SMN1 gene Proteins 0.000 claims description 2
- 108700019146 Transgenes Proteins 0.000 claims description 2
- 230000002950 deficient Effects 0.000 claims description 2
- 230000002708 enhancing effect Effects 0.000 claims description 2
- 238000012224 gene deletion Methods 0.000 claims description 2
- 108091070501 miRNA Proteins 0.000 claims description 2
- 230000035772 mutation Effects 0.000 claims description 2
- 108090000623 proteins and genes Proteins 0.000 claims description 2
- 102000004169 proteins and genes Human genes 0.000 claims description 2
- 208000032471 type 1 spinal muscular atrophy Diseases 0.000 claims description 2
- 208000032521 type II spinal muscular atrophy Diseases 0.000 claims description 2
- 208000032527 type III spinal muscular atrophy Diseases 0.000 claims description 2
- 208000005606 type IV spinal muscular atrophy Diseases 0.000 claims description 2
- 201000006913 intermediate spinal muscular atrophy Diseases 0.000 claims 1
- 238000002560 therapeutic procedure Methods 0.000 claims 1
- 238000000034 method Methods 0.000 description 11
- 238000012986 modification Methods 0.000 description 1
- 230000004048 modification Effects 0.000 description 1
- 239000000546 pharmaceutical excipient Substances 0.000 description 1
Applications Claiming Priority (5)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| CN2020107173 | 2020-08-05 | ||
| CNPCT/CN2020/107173 | 2020-08-05 | ||
| CNPCT/CN2020/138056 | 2020-12-21 | ||
| CN2020138056 | 2020-12-21 | ||
| PCT/CN2021/110521 WO2022028472A1 (en) | 2020-08-05 | 2021-08-04 | Nucleic acid constructs and uses thereof for treating spinal muscular atrophy |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2023537358A JP2023537358A (ja) | 2023-08-31 |
| JPWO2022028472A5 true JPWO2022028472A5 (enExample) | 2024-08-13 |
Family
ID=80120030
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2023507927A Pending JP2023537358A (ja) | 2020-08-05 | 2021-08-04 | 核酸構築物及び脊髄性筋委縮症治療のためのその使用 |
Country Status (9)
| Country | Link |
|---|---|
| US (1) | US20230293730A1 (enExample) |
| EP (1) | EP4192518A4 (enExample) |
| JP (1) | JP2023537358A (enExample) |
| KR (1) | KR20230044522A (enExample) |
| CN (1) | CN116685329B (enExample) |
| AU (1) | AU2021323289A1 (enExample) |
| CA (1) | CA3190601A1 (enExample) |
| IL (1) | IL300277A (enExample) |
| WO (1) | WO2022028472A1 (enExample) |
Families Citing this family (5)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US20230151364A1 (en) * | 2020-04-20 | 2023-05-18 | Regents Of The University Of Minnesota | Compositions and methods for increasing sodium current in cardiac cells |
| WO2023219394A1 (ko) * | 2022-05-10 | 2023-11-16 | 서울대학교산학협력단 | 인간 smn1 단백질 변이체 및 이의 용도 |
| EP4577662A1 (en) * | 2022-08-24 | 2025-07-02 | Beacon Therapeutics (USA), Inc. | Methods and compositions for the production of recombinant adeno-associated virus (raav) vectors |
| CN121175424A (zh) * | 2023-05-12 | 2025-12-19 | 上海金珂博生物技术有限公司 | 一种携带smn基因表达框的病毒载体及其用途 |
| CN118685413B (zh) * | 2024-08-28 | 2025-02-07 | 杭州嘉因生物科技有限公司 | 下调内源性smn的诱导型稳定细胞株的构建及其应用 |
Family Cites Families (10)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CN101341259B (zh) * | 2005-08-01 | 2011-12-21 | 俄亥俄州立大学研究基金会 | 用于乳腺癌的诊断、预后和治疗的基于MicroRNA的方法和组合物 |
| CN101787373B (zh) * | 2009-01-23 | 2013-06-19 | 中国人民解放军第二军医大学东方肝胆外科医院 | 一种携带外源基因在包装细胞中高效生产的重组病毒载体、其构建方法及其用途 |
| DK2826860T3 (en) * | 2010-04-23 | 2018-12-03 | Univ Massachusetts | CNS targeting AAV vectors and methods for their use |
| FR3004463A1 (fr) * | 2013-04-11 | 2014-10-17 | Genethon | Systeme d'expression pour une therapie genique selective |
| CN110392582A (zh) * | 2017-02-08 | 2019-10-29 | 弗里德里克·米谢尔生物医学研究所 | 用于使基因在视网膜神经节细胞中特异性表达的启动子SynP88 |
| JOP20190200A1 (ar) * | 2017-02-28 | 2019-08-27 | Univ Pennsylvania | تركيبات نافعة في معالجة ضمور العضل النخاعي |
| CN108795946B (zh) * | 2018-06-28 | 2022-03-04 | 北京锦篮基因科技有限公司 | 携带设计smn1基因表达框的重组腺相关病毒及应用 |
| CN111088284B (zh) * | 2019-05-31 | 2022-02-01 | 北京京谱迪基因科技有限公司 | 携带截短抗肌萎缩蛋白基因表达框的aav载体、制备方法及应用 |
| CN110616262A (zh) * | 2019-08-14 | 2019-12-27 | 深圳市人民医院 | 外泌体中microRNA在评估脑胶质瘤放疗效果中的用途 |
| CN113755524B (zh) * | 2020-06-02 | 2023-11-03 | 舒泰神（北京）生物制药股份有限公司 | 用于治疗脊髓性肌萎缩的腺相关病毒载体及其用途 |
-
2021
- 2021-08-04 EP EP21852149.0A patent/EP4192518A4/en active Pending
- 2021-08-04 JP JP2023507927A patent/JP2023537358A/ja active Pending
- 2021-08-04 CN CN202180067807.5A patent/CN116685329B/zh active Active
- 2021-08-04 US US18/019,449 patent/US20230293730A1/en active Pending
- 2021-08-04 CA CA3190601A patent/CA3190601A1/en active Pending
- 2021-08-04 WO PCT/CN2021/110521 patent/WO2022028472A1/en not_active Ceased
- 2021-08-04 AU AU2021323289A patent/AU2021323289A1/en active Pending
- 2021-08-04 KR KR1020237007688A patent/KR20230044522A/ko active Pending
- 2021-08-04 IL IL300277A patent/IL300277A/en unknown
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