JPWO2021032068A5 - - Google Patents
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- Publication number
- JPWO2021032068A5 JPWO2021032068A5 JP2021554990A JP2021554990A JPWO2021032068A5 JP WO2021032068 A5 JPWO2021032068 A5 JP WO2021032068A5 JP 2021554990 A JP2021554990 A JP 2021554990A JP 2021554990 A JP2021554990 A JP 2021554990A JP WO2021032068 A5 JPWO2021032068 A5 JP WO2021032068A5
- Authority
- JP
- Japan
- Prior art keywords
- ptbp1
- group
- rna
- gene editing
- gene
- 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
- 108090000623 proteins and genes Proteins 0.000 claims 13
- 102000004169 proteins and genes Human genes 0.000 claims 12
- 101150052992 PTBP1 gene Proteins 0.000 claims 10
- 238000010362 genome editing Methods 0.000 claims 10
- 108091033409 CRISPR Proteins 0.000 claims 9
- 239000000203 mixture Substances 0.000 claims 8
- 210000002569 neuron Anatomy 0.000 claims 6
- 108091032973 (ribonucleotides)n+m Proteins 0.000 claims 5
- 208000012902 Nervous system disease Diseases 0.000 claims 5
- 210000004027 cell Anatomy 0.000 claims 5
- 150000001875 compounds Chemical class 0.000 claims 5
- 239000003814 drug Substances 0.000 claims 5
- 239000013604 expression vector Substances 0.000 claims 5
- 108020005004 Guide RNA Proteins 0.000 claims 4
- VYFYYTLLBUKUHU-UHFFFAOYSA-N dopamine Chemical compound NCCC1=CC=C(O)C(O)=C1 VYFYYTLLBUKUHU-UHFFFAOYSA-N 0.000 claims 4
- 229940079593 drug Drugs 0.000 claims 4
- 230000000694 effects Effects 0.000 claims 4
- 239000003112 inhibitor Substances 0.000 claims 4
- 210000004498 neuroglial cell Anatomy 0.000 claims 4
- 230000002265 prevention Effects 0.000 claims 4
- -1 small molecule compounds Chemical class 0.000 claims 4
- 238000010354 CRISPR gene editing Methods 0.000 claims 3
- 108020004414 DNA Proteins 0.000 claims 3
- 208000003098 Ganglion Cysts Diseases 0.000 claims 3
- 208000025966 Neurological disease Diseases 0.000 claims 3
- 208000005400 Synovial Cyst Diseases 0.000 claims 3
- 210000001130 astrocyte Anatomy 0.000 claims 3
- 238000000034 method Methods 0.000 claims 3
- 230000016273 neuron death Effects 0.000 claims 3
- 241000124008 Mammalia Species 0.000 claims 2
- 230000034994 death Effects 0.000 claims 2
- 230000004069 differentiation Effects 0.000 claims 2
- 229960003638 dopamine Drugs 0.000 claims 2
- 238000009472 formulation Methods 0.000 claims 2
- 230000006698 induction Effects 0.000 claims 2
- 238000004519 manufacturing process Methods 0.000 claims 2
- 239000013607 AAV vector Substances 0.000 claims 1
- 108020000948 Antisense Oligonucleotides Proteins 0.000 claims 1
- 108091023037 Aptamer Proteins 0.000 claims 1
- 108091026890 Coding region Proteins 0.000 claims 1
- 108700011259 MicroRNAs Proteins 0.000 claims 1
- 208000016285 Movement disease Diseases 0.000 claims 1
- 208000018737 Parkinson disease Diseases 0.000 claims 1
- 208000017442 Retinal disease Diseases 0.000 claims 1
- 108091027967 Small hairpin RNA Proteins 0.000 claims 1
- 108020004459 Small interfering RNA Proteins 0.000 claims 1
- 206010047571 Visual impairment Diseases 0.000 claims 1
- 239000000074 antisense oligonucleotide Substances 0.000 claims 1
- 238000012230 antisense oligonucleotides Methods 0.000 claims 1
- 238000004113 cell culture Methods 0.000 claims 1
- 210000003710 cerebral cortex Anatomy 0.000 claims 1
- 238000006243 chemical reaction Methods 0.000 claims 1
- 238000012258 culturing Methods 0.000 claims 1
- 230000007850 degeneration Effects 0.000 claims 1
- 230000002401 inhibitory effect Effects 0.000 claims 1
- 210000001259 mesencephalon Anatomy 0.000 claims 1
- 210000001577 neostriatum Anatomy 0.000 claims 1
- 230000004770 neurodegeneration Effects 0.000 claims 1
- 208000015122 neurodegenerative disease Diseases 0.000 claims 1
- 230000001737 promoting effect Effects 0.000 claims 1
- 238000012216 screening Methods 0.000 claims 1
- 210000000278 spinal cord Anatomy 0.000 claims 1
- 210000003523 substantia nigra Anatomy 0.000 claims 1
- 239000013598 vector Substances 0.000 claims 1
- 208000029257 vision disease Diseases 0.000 claims 1
- 230000004393 visual impairment Effects 0.000 claims 1
Applications Claiming Priority (9)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| CN201910760367 | 2019-08-16 | ||
| CN201910760367.6 | 2019-08-16 | ||
| CN201911046435.9 | 2019-10-30 | ||
| CN201911046435 | 2019-10-30 | ||
| PCT/CN2020/081489 WO2021031565A1 (en) | 2019-08-16 | 2020-03-26 | Treatment of Neuronal Diseases |
| CNPCT/CN2020/081489 | 2020-03-26 | ||
| CN202010740568.2 | 2020-07-28 | ||
| CN202010740568.2A CN112451669A (zh) | 2019-08-16 | 2020-07-28 | Ptbp1抑制剂在预防和/或治疗功能性神经元死亡相关的神经系统疾病中的应用 |
| PCT/CN2020/109653 WO2021032068A1 (zh) | 2019-08-16 | 2020-08-17 | Ptbp1抑制剂在预防和/或治疗功能性神经元死亡相关的神经系统疾病中的应用 |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2022543722A JP2022543722A (ja) | 2022-10-14 |
| JPWO2021032068A5 true JPWO2021032068A5 (https=) | 2023-08-01 |
| JP2022543722A5 JP2022543722A5 (https=) | 2023-08-01 |
Family
ID=74659587
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2021554990A Withdrawn JP2022543722A (ja) | 2019-08-16 | 2020-08-17 | 機能的ニューロン死に関連する神経系疾患の予防および／または治療におけるＰｔｂｐ１阻害剤の使用 |
Country Status (4)
| Country | Link |
|---|---|
| US (2) | US20220273726A1 (https=) |
| EP (1) | EP4039801A4 (https=) |
| JP (1) | JP2022543722A (https=) |
| WO (2) | WO2021032068A1 (https=) |
Families Citing this family (3)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2023150553A1 (en) * | 2022-02-01 | 2023-08-10 | University Of Rochester | Gpr17 promoter-based targeting and transduction of glial progenitor cells |
| WO2022246204A2 (en) * | 2021-05-21 | 2022-11-24 | Ionis Pharmaceuticals, Inc. | Compounds for reducing ptbp1 expression |
| US20250161495A1 (en) * | 2022-02-01 | 2025-05-22 | University Of Rochester | Methods of generating a population of neurons from human glial progenitor cells and genetic constructs for carrying out such methods |
Family Cites Families (22)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US4797368A (en) | 1985-03-15 | 1989-01-10 | The United States Of America As Represented By The Department Of Health And Human Services | Adeno-associated virus as eukaryotic expression vector |
| US5139941A (en) | 1985-10-31 | 1992-08-18 | University Of Florida Research Foundation, Inc. | AAV transduction vectors |
| WO1991018088A1 (en) | 1990-05-23 | 1991-11-28 | The United States Of America, Represented By The Secretary, United States Department Of Commerce | Adeno-associated virus (aav)-based eucaryotic vectors |
| US5173414A (en) | 1990-10-30 | 1992-12-22 | Applied Immune Sciences, Inc. | Production of recombinant adeno-associated virus vectors |
| US5252479A (en) | 1991-11-08 | 1993-10-12 | Research Corporation Technologies, Inc. | Safe vector for gene therapy |
| US6995006B2 (en) | 1997-09-05 | 2006-02-07 | Targeted Genetics Corporation | Methods for generating high titer helper-free preparations of released recombinant AAV vectors |
| US6566118B1 (en) | 1997-09-05 | 2003-05-20 | Targeted Genetics Corporation | Methods for generating high titer helper-free preparations of released recombinant AAV vectors |
| PT1916258E (pt) | 1999-08-09 | 2014-07-29 | Genzyme Corp | Aumento da expressão de uma sequência nucleotídica heteróloga de cadeia simples a partir de vectores virais recombinantes por concepção da sequência de maneira que esta forme pares de bases intracadeia |
| KR20090071603A (ko) * | 2006-09-19 | 2009-07-01 | 노파르티스 아게 | Ｒａｆ 억제제에 대한 표적 조절, 효능, 진단 및/또는 예후의 바이오마커 |
| EP2104734A2 (en) * | 2006-12-08 | 2009-09-30 | Asuragen, INC. | Mir-20 regulated genes and pathways as targets for therapeutic intervention |
| US20100144838A1 (en) * | 2008-12-01 | 2010-06-10 | Beck William T | Methods for Identifying Modulators of Pyrimidine Tract Binding Protein |
| CN102858985A (zh) * | 2009-07-24 | 2013-01-02 | 西格马-奥尔德里奇有限责任公司 | 基因组编辑方法 |
| US20130209463A1 (en) * | 2010-06-30 | 2013-08-15 | Compugen Ltd. | Polypeptides and uses thereof as a drug for treatment of multiple sclerosis, rheumatoid arthritis and other autoimmune disorders |
| WO2014071157A1 (en) * | 2012-11-01 | 2014-05-08 | The Regents Of The University Of California | Methods for engineering non-neuronal cells into neurons and using newly engineered neurons to treat neurodegenerative diseases |
| WO2017003466A1 (en) * | 2015-06-30 | 2017-01-05 | University Of South Florida | Conversion of non-neuronal cells into neurons |
| CN105950557B (zh) * | 2016-04-22 | 2019-10-25 | 中国科学院生物物理研究所 | 一种控制人神经细胞重编程的信号通路及其应用 |
| WO2018048877A1 (en) * | 2016-09-06 | 2018-03-15 | The University Of North Carolina At Chapel Hill | Generation of neurons by reprogramming of oligodendrocytes and oligodendrocyte precursor cells |
| WO2018112446A2 (en) * | 2016-12-18 | 2018-06-21 | Selonterra, Inc. | Use of apoe4 motif-mediated genes for diagnosis and treatment of alzheimer's disease |
| CA3096691A1 (en) * | 2018-04-11 | 2019-10-17 | The Regents Of The University Of California | Reprogramming of non-neuronal cells into neurons and methods and compositions to treat neurodegenerative diseases and disorders |
| WO2020113338A1 (en) * | 2018-12-05 | 2020-06-11 | Adaerata, Limited Partnership | Method of reducing neuronal microtubule binding protein tau (tau) levels |
| CN109913420A (zh) * | 2019-03-07 | 2019-06-21 | 北京师范大学 | Cdc20共表达基因网络作为胶质瘤治疗靶点的应用 |
| CN109777877B (zh) * | 2019-03-19 | 2022-01-04 | 宁波市第一医院 | 基于ptbp1甲基化辅助诊断脑动脉瘤的检测试剂盒及其应用 |
-
2020
- 2020-08-17 JP JP2021554990A patent/JP2022543722A/ja not_active Withdrawn
- 2020-08-17 EP EP20855397.4A patent/EP4039801A4/en not_active Withdrawn
- 2020-08-17 US US17/627,052 patent/US20220273726A1/en not_active Abandoned
- 2020-08-17 WO PCT/CN2020/109653 patent/WO2021032068A1/zh not_active Ceased
- 2020-08-17 WO PCT/CN2020/109655 patent/WO2021032069A1/en not_active Ceased
-
2025
- 2025-07-18 US US19/273,720 patent/US20250345365A1/en active Pending
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