JP2019523648A5 - - Google Patents
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- Publication number
- JP2019523648A5 JP2019523648A5 JP2018565324A JP2018565324A JP2019523648A5 JP 2019523648 A5 JP2019523648 A5 JP 2019523648A5 JP 2018565324 A JP2018565324 A JP 2018565324A JP 2018565324 A JP2018565324 A JP 2018565324A JP 2019523648 A5 JP2019523648 A5 JP 2019523648A5
- Authority
- JP
- Japan
- Prior art keywords
- nucleic acid
- acid sequence
- aav vector
- seq
- nucleotides
- 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 |
|---|---|---|---|
| GBGB1610448.1A GB201610448D0 (en) | 2016-06-15 | 2016-06-15 | Adeno-associated viral vector system |
| GB1610448.1 | 2016-06-15 | ||
| GB1707261.2 | 2017-05-05 | ||
| GBGB1707261.2A GB201707261D0 (en) | 2017-05-05 | 2017-05-05 | Adeno-associated viral vector system |
| PCT/GB2017/051741 WO2017216560A1 (en) | 2016-06-15 | 2017-06-14 | Dual overlapping adeno-associated viral vector system for expressing abc4a |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2019523648A JP2019523648A (ja) | 2019-08-29 |
| JP2019523648A5 true JP2019523648A5 (cg-RX-API-DMAC10.html) | 2020-07-27 |
Family
ID=59101505
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2018565324A Pending JP2019523648A (ja) | 2016-06-15 | 2017-06-14 | Ａｂｃ４ａを発現させるためのデュアル重複アデノ随伴ウイルスベクターシステム |
Country Status (13)
Families Citing this family (14)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| DK2986635T3 (en) | 2013-04-18 | 2019-01-28 | Fond Telethon | EFFECTIVE DELIVERY OF BIG GENES THROUGH DUAL-AAV VECTORS |
| US12359221B2 (en) | 2017-05-05 | 2025-07-15 | The Regents Of The University Of California | Compositions and methods for expressing otoferlin |
| US20210147870A1 (en) * | 2018-04-05 | 2021-05-20 | Nightstarx Limited | Compositions and methods for the treatment of stargardt disease |
| CA3116606A1 (en) * | 2018-10-15 | 2020-04-23 | Fondazione Telethon | Intein proteins and uses thereof |
| GB201817469D0 (en) * | 2018-10-26 | 2018-12-12 | Univ Oxford Innovation Ltd | Gene therapy for retinal disease |
| WO2020093018A1 (en) | 2018-11-01 | 2020-05-07 | University Of Florida Research Foundation, Incorporated | A codon optimized otoferlin aav dual vector gene therapy |
| EP3921032A4 (en) | 2019-02-08 | 2022-11-30 | Decibel Therapeutics, Inc. | COMPOSITIONS AND METHODS FOR TREATMENT OF SENSORINEURAL HEARING LOSS USING OTOFERLIN DOUBLE VECTOR SYSTEMS |
| WO2021015997A1 (en) * | 2019-07-15 | 2021-01-28 | President And Fellows Of Harvard College | Methods and compositions for gene delivery |
| TWI896570B (zh) | 2019-10-30 | 2025-09-11 | 美商迪賽博治療公司 | 使用耳畸蛋白雙載體系統治療感覺神經性聽力損失之組合物及方法 |
| IL295741A (en) * | 2020-04-01 | 2022-10-01 | Univ Florida | Dual aav-myo7a vectors with improved safety for the treatment of ush1b |
| US20230338580A1 (en) * | 2020-08-14 | 2023-10-26 | Case Western Reserve University | Plasmid vectors and nanoparticles for treating ocular disorders |
| US20240425877A1 (en) * | 2021-05-07 | 2024-12-26 | Ucl Business Ltd | Abca4 genome editing |
| WO2023160454A1 (zh) * | 2022-02-25 | 2023-08-31 | 北京中因科技有限公司 | 表达盒组合及其应用 |
| EP4728080A1 (en) * | 2023-06-16 | 2026-04-22 | Arkasubhra Ghosh | A hybrid dual aav vector system with splice enhancer elements for expression of large genes |
Family Cites Families (5)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| PT3272872T (pt) * | 2005-10-20 | 2020-06-26 | Uniqure Ip Bv | Vetores aav melhorados produzidos em células de insetos |
| US20090214478A1 (en) * | 2008-02-21 | 2009-08-27 | Alberto Auricchio | Method of treating ocular diseases by gene therapy |
| CA2770075C (en) * | 2009-08-07 | 2021-08-24 | Perrine Martin | Composition for treating hbv infection |
| WO2013075008A1 (en) * | 2011-11-16 | 2013-05-23 | University Of Florida Research Foundation Inc. | Aav dual vector systems for gene therapy |
| DK2986635T3 (en) * | 2013-04-18 | 2019-01-28 | Fond Telethon | EFFECTIVE DELIVERY OF BIG GENES THROUGH DUAL-AAV VECTORS |
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2017
- 2017-06-14 AU AU2017286623A patent/AU2017286623A1/en not_active Abandoned
- 2017-06-14 WO PCT/GB2017/051741 patent/WO2017216560A1/en not_active Ceased
- 2017-06-14 US US16/309,428 patent/US20190309326A1/en not_active Abandoned
- 2017-06-14 CA CA3025445A patent/CA3025445A1/en not_active Abandoned
- 2017-06-14 RU RU2019100525A patent/RU2765826C2/ru active
- 2017-06-14 SG SG11201811244SA patent/SG11201811244SA/en unknown
- 2017-06-14 KR KR1020197001281A patent/KR20190020745A/ko not_active Ceased
- 2017-06-14 MX MX2018015629A patent/MX2018015629A/es unknown
- 2017-06-14 BR BR112018075855-8A patent/BR112018075855A2/pt not_active Application Discontinuation
- 2017-06-14 JP JP2018565324A patent/JP2019523648A/ja active Pending
- 2017-06-14 CN CN201780037639.9A patent/CN109642242A/zh active Pending
- 2017-06-14 EP EP17732175.9A patent/EP3472328A1/en not_active Withdrawn
-
2018
- 2018-12-05 IL IL263523A patent/IL263523A/en unknown