JP2020511470A5 - - Google Patents
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- Publication number
- JP2020511470A5 JP2020511470A5 JP2019550825A JP2019550825A JP2020511470A5 JP 2020511470 A5 JP2020511470 A5 JP 2020511470A5 JP 2019550825 A JP2019550825 A JP 2019550825A JP 2019550825 A JP2019550825 A JP 2019550825A JP 2020511470 A5 JP2020511470 A5 JP 2020511470A5
- Authority
- JP
- Japan
- Prior art keywords
- disease
- lysosomal transmembrane
- disorder
- transmembrane protein
- lysosomal
- 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
Priority Applications (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2023005909A JP2023052475A (ja) | 2017-03-15 | 2023-01-18 | リソソーム障害を治療する方法 |
| JP2025000101A JP2025039642A (ja) | 2017-03-15 | 2025-01-06 | リソソーム障害を治療する方法 |
Applications Claiming Priority (5)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201762471741P | 2017-03-15 | 2017-03-15 | |
| US62/471,741 | 2017-03-15 | ||
| US201762507713P | 2017-05-17 | 2017-05-17 | |
| US62/507,713 | 2017-05-17 | ||
| PCT/US2018/022598 WO2018170239A1 (en) | 2017-03-15 | 2018-03-15 | Methods of treating lysosomal disorders |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2023005909A Division JP2023052475A (ja) | 2017-03-15 | 2023-01-18 | リソソーム障害を治療する方法 |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2020511470A JP2020511470A (ja) | 2020-04-16 |
| JP2020511470A5 true JP2020511470A5 (https=) | 2021-04-15 |
| JP7233375B2 JP7233375B2 (ja) | 2023-03-06 |
Family
ID=63523990
Family Applications (3)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2019550825A Active JP7233375B2 (ja) | 2017-03-15 | 2018-03-15 | リソソーム障害を治療する方法 |
| JP2023005909A Pending JP2023052475A (ja) | 2017-03-15 | 2023-01-18 | リソソーム障害を治療する方法 |
| JP2025000101A Pending JP2025039642A (ja) | 2017-03-15 | 2025-01-06 | リソソーム障害を治療する方法 |
Family Applications After (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2023005909A Pending JP2023052475A (ja) | 2017-03-15 | 2023-01-18 | リソソーム障害を治療する方法 |
| JP2025000101A Pending JP2025039642A (ja) | 2017-03-15 | 2025-01-06 | リソソーム障害を治療する方法 |
Country Status (8)
| Country | Link |
|---|---|
| US (2) | US11806367B2 (https=) |
| EP (1) | EP3596111A4 (https=) |
| JP (3) | JP7233375B2 (https=) |
| CN (1) | CN110418800A (https=) |
| AU (2) | AU2018236353B2 (https=) |
| CA (1) | CA3056617A1 (https=) |
| IL (1) | IL269082A (https=) |
| WO (1) | WO2018170239A1 (https=) |
Families Citing this family (12)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CA3056617A1 (en) | 2017-03-15 | 2018-09-20 | The Regents Of The University Of California | Methods of treating lysosomal disorders |
| KR102918544B1 (ko) | 2018-07-12 | 2026-01-27 | 로켓 파마슈티컬스, 리미티드 | 다논병을 치료하기 위한 유전자 요법 벡터 |
| CN109880849B (zh) * | 2019-01-03 | 2023-04-11 | 四川大学 | 靶向肿瘤相关巨噬细胞的GHOST-shRNA表达载体复合物及其应用 |
| WO2020160458A1 (en) | 2019-02-01 | 2020-08-06 | Avrobio, Inc. | Compositions and methods for treating neurocognitive disorders |
| WO2020179931A1 (ja) * | 2019-03-07 | 2020-09-10 | 国立大学法人 東京医科歯科大学 | ハイスループット遺伝子編集技術 |
| US12319928B2 (en) * | 2019-03-21 | 2025-06-03 | The Regents Of The University Of Colorado, A Body Corporate | Methods of treating Danon disease |
| WO2020210218A1 (en) * | 2019-04-12 | 2020-10-15 | The Regents Of The University Of California | Methods for treating inherited eye defects |
| WO2021073607A1 (en) * | 2019-10-17 | 2021-04-22 | Westlake Therapeutics (Hangzhou) Co. Limited | Engineering red blood cells for treatment of lysosomal storage diseases |
| GB202003536D0 (en) * | 2020-03-11 | 2020-04-29 | Ucl Business Ltd | Gene therapy |
| US12173304B2 (en) * | 2020-07-08 | 2024-12-24 | Neuracle Genetics Inc. | Intron fragments |
| CN114934066B (zh) * | 2022-04-28 | 2024-10-18 | 深圳临研医学有限公司 | 石骨症的基因编辑体系及其应用 |
| CN118388623B (zh) * | 2024-06-28 | 2024-10-08 | 首都医科大学 | Sialin蛋白在调控细胞线粒体功能中的应用 |
Family Cites Families (9)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| EP3196301B1 (en) | 2012-07-11 | 2018-10-17 | Sangamo Therapeutics, Inc. | Methods and compositions for the treatment of monogenic diseases |
| JP2015529466A (ja) | 2012-09-14 | 2015-10-08 | ザ リージェンツ オブ ザ ユニバーシティ オブ カリフォルニア | 鎌状赤血球症の幹細胞遺伝子治療のためのレンチウイルスベクター |
| HK1208371A1 (en) | 2012-10-09 | 2016-03-04 | 桑比欧公司 | Methods and compositions for treatment of retinal degeneration |
| US10155794B2 (en) | 2013-07-16 | 2018-12-18 | The Trustees Of The University Of Pennsylvania | Compositions and methods for treatment of disorders related to CEP290 |
| CN106459995B (zh) | 2013-11-07 | 2020-02-21 | 爱迪塔斯医药有限公司 | 使用统治型gRNA的CRISPR相关方法和组合物 |
| PE20180772A1 (es) * | 2015-07-01 | 2018-05-07 | Univ California | Vectores retrovirales que contienen un promotor de ubiquitina c humana de orientacion inversa |
| US10670862B2 (en) | 2015-07-02 | 2020-06-02 | Microsoft Technology Licensing, Llc | Diffractive optical elements with asymmetric profiles |
| JP7089282B2 (ja) * | 2015-07-20 | 2022-06-22 | アンジオクライン・バイオサイエンス・インコーポレイテッド | 幹細胞移植のための方法および組成物 |
| CA3056617A1 (en) | 2017-03-15 | 2018-09-20 | The Regents Of The University Of California | Methods of treating lysosomal disorders |
-
2018
- 2018-03-15 CA CA3056617A patent/CA3056617A1/en active Pending
- 2018-03-15 AU AU2018236353A patent/AU2018236353B2/en active Active
- 2018-03-15 JP JP2019550825A patent/JP7233375B2/ja active Active
- 2018-03-15 US US16/493,573 patent/US11806367B2/en active Active
- 2018-03-15 WO PCT/US2018/022598 patent/WO2018170239A1/en not_active Ceased
- 2018-03-15 CN CN201880018325.9A patent/CN110418800A/zh active Pending
- 2018-03-15 EP EP18766923.9A patent/EP3596111A4/en active Pending
-
2019
- 2019-09-02 IL IL26908219A patent/IL269082A/en unknown
-
2023
- 2023-01-18 JP JP2023005909A patent/JP2023052475A/ja active Pending
- 2023-06-27 US US18/342,642 patent/US20240009247A1/en active Pending
-
2024
- 2024-03-20 AU AU2024201797A patent/AU2024201797A1/en active Pending
-
2025
- 2025-01-06 JP JP2025000101A patent/JP2025039642A/ja active Pending
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