JP2022540446A5 - - Google Patents
Info
- Publication number
- JP2022540446A5 JP2022540446A5 JP2022500898A JP2022500898A JP2022540446A5 JP 2022540446 A5 JP2022540446 A5 JP 2022540446A5 JP 2022500898 A JP2022500898 A JP 2022500898A JP 2022500898 A JP2022500898 A JP 2022500898A JP 2022540446 A5 JP2022540446 A5 JP 2022540446A5
- Authority
- JP
- Japan
- Prior art keywords
- rna
- composition according
- rhodopsin
- protein
- pathogenic
- 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
Applications Claiming Priority (5)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201962872604P | 2019-07-10 | 2019-07-10 | |
| US62/872,604 | 2019-07-10 | ||
| US202062968819P | 2020-01-31 | 2020-01-31 | |
| US62/968,819 | 2020-01-31 | ||
| PCT/US2020/041634 WO2021007529A1 (en) | 2019-07-10 | 2020-07-10 | Rna-targeting knockdown and replacement compositions and methods for use |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2022540446A JP2022540446A (ja) | 2022-09-15 |
| JP2022540446A5 true JP2022540446A5 (https=) | 2023-07-18 |
Family
ID=71995058
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2022500898A Pending JP2022540446A (ja) | 2019-07-10 | 2020-07-10 | Ｒｎａ標的ノックダウン及び置換の組成物及び使用方法 |
Country Status (7)
| Country | Link |
|---|---|
| US (1) | US20210009987A1 (https=) |
| EP (1) | EP3997227A1 (https=) |
| JP (1) | JP2022540446A (https=) |
| CN (1) | CN114450031A (https=) |
| AU (1) | AU2020310201A1 (https=) |
| CA (1) | CA3145309A1 (https=) |
| WO (1) | WO2021007529A1 (https=) |
Families Citing this family (17)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2011160052A2 (en) | 2010-06-18 | 2011-12-22 | The University Of North Carolina At Chapel Hill | Methods and compositions for sequence specific rna endonucleases |
| EP3901262A1 (en) * | 2020-04-20 | 2021-10-27 | Universität Regensburg | Compositions for use in treating autosomal dominant best1-related retinopathies |
| EP4216964A4 (en) * | 2020-09-24 | 2025-02-12 | Ionis Pharmaceuticals, Inc. | Compounds and methods for reducing apoe expression |
| EP4291654A2 (en) | 2021-02-12 | 2023-12-20 | Alnylam Pharmaceuticals, Inc. | Superoxide dismutase 1 (sod1) irna compositions and methods of use thereof for treating or preventing superoxide dismutase 1- (sod1-) associated neurodegenerative diseases |
| WO2022221278A1 (en) * | 2021-04-12 | 2022-10-20 | Locanabio, Inc. | Compositions and methods comprising hybrid promoters |
| WO2022241059A2 (en) * | 2021-05-11 | 2022-11-17 | Mammoth Biosciences, Inc. | Effector proteins and methods of use |
| WO2023015153A2 (en) * | 2021-08-02 | 2023-02-09 | President And Fellows Of Harvard College | ANTISENSE OLIGONUCLEOTIDE TARGETING APOE ε4 AND USES THEREOF |
| EP4419688A2 (en) * | 2021-10-21 | 2024-08-28 | Prime Medicine, Inc. | Genome editing compositions and methods for treatment of usher syndrome type 3 |
| US12173368B2 (en) * | 2022-03-11 | 2024-12-24 | New England Biolabs, Inc. | Compositions and analysis of dephosphorylated oligoribonucleotides |
| EP4493702A4 (en) * | 2022-03-18 | 2026-04-01 | Univ Florida | METHODS AND COMPOSITIONS FOR TREATING TNNT2-RELATED CARDIOMYOPATHY WITH A VIRAL VECTOR |
| WO2023215761A1 (en) * | 2022-05-03 | 2023-11-09 | Tacit Therapeutics, Inc. | Localization of trans-splicing nucleic acid molecules to and within the cellular nucleus |
| WO2024196955A2 (en) * | 2023-03-20 | 2024-09-26 | Duke University | Apoe-targeted microglia-specific gene therapy for neurological diseases |
| WO2025043118A1 (en) * | 2023-08-23 | 2025-02-27 | Ohio State Innovation Foundation | Making tiny rnas inside the cell |
| WO2025043278A1 (en) * | 2023-08-25 | 2025-03-06 | PYC Therapeutics Limited | Gene-mediated conditions |
| WO2025159427A1 (ko) * | 2024-01-26 | 2025-07-31 | 서울대학교산학협력단 | 신규한 apoe 안티센스 올리고뉴클레오타이드 및 이의 용도 |
| WO2025217591A1 (en) * | 2024-04-12 | 2025-10-16 | Strand Therapeutics Inc. | Human-derived synthetic regulators and uses thereof |
| WO2026010243A1 (ko) * | 2024-07-03 | 2026-01-08 | 주식회사 아바타테라퓨틱스 | Crispr/cas 시스템을 사용하는 best1 유전자 편집 방법 |
Family Cites Families (14)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| ES2714007T3 (es) | 2007-04-09 | 2019-05-24 | Univ Florida | Composiciones de vectores rAAV que tienen proteínas de la cápside modificadas en tirosina y métodos para su uso |
| EP2058401A1 (en) * | 2007-10-05 | 2009-05-13 | Genethon | Widespread gene delivery to motor neurons using peripheral injection of AAV vectors |
| CN101895633A (zh) | 2010-07-14 | 2010-11-24 | 中兴通讯股份有限公司 | 一种移动终端及其解锁方法 |
| WO2012068627A1 (en) | 2010-11-24 | 2012-05-31 | The University Of Western Australia | Peptides for the specific binding of rna targets |
| US9951349B2 (en) * | 2011-09-27 | 2018-04-24 | Yale University | Compositions and methods for transient expression of recombinant RNA |
| DK2784157T3 (da) | 2011-10-21 | 2019-10-21 | Univ Kyushu Nat Univ Corp | Designfremgangsmåde til et rna-bindende protein under anvendelse af ppr-motiv og anvendelse deraf |
| US9850269B2 (en) * | 2014-04-25 | 2017-12-26 | Translate Bio, Inc. | Methods for purification of messenger RNA |
| US10330674B2 (en) | 2015-01-13 | 2019-06-25 | Massachusetts Institute Of Technology | Pumilio domain-based modular protein architecture for RNA binding |
| EP3858992A1 (en) * | 2015-03-13 | 2021-08-04 | The Jackson Laboratory | A three-component crispr/cas complex system and uses thereof |
| EP3289080B1 (en) * | 2015-04-30 | 2021-08-25 | The Trustees of Columbia University in the City of New York | Gene therapy for autosomal dominant diseases |
| US10876101B2 (en) | 2017-03-28 | 2020-12-29 | Locanabio, Inc. | CRISPR-associated (Cas) protein |
| CN108690861A (zh) * | 2017-04-10 | 2018-10-23 | 肖晓莺 | 用于早期乳腺癌症诊断的微小核糖核酸 |
| US10392616B2 (en) | 2017-06-30 | 2019-08-27 | Arbor Biotechnologies, Inc. | CRISPR RNA targeting enzymes and systems and uses thereof |
| US10476825B2 (en) | 2017-08-22 | 2019-11-12 | Salk Institue for Biological Studies | RNA targeting methods and compositions |
-
2020
- 2020-07-10 EP EP20753516.2A patent/EP3997227A1/en not_active Withdrawn
- 2020-07-10 JP JP2022500898A patent/JP2022540446A/ja active Pending
- 2020-07-10 AU AU2020310201A patent/AU2020310201A1/en not_active Abandoned
- 2020-07-10 CA CA3145309A patent/CA3145309A1/en active Pending
- 2020-07-10 US US16/926,205 patent/US20210009987A1/en not_active Abandoned
- 2020-07-10 CN CN202080061471.7A patent/CN114450031A/zh active Pending
- 2020-07-10 WO PCT/US2020/041634 patent/WO2021007529A1/en not_active Ceased
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