JP2019523648A5 - - Google Patents
Download PDFInfo
- 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 |
|---|---|---|---|
| GB1610448.1 | 2016-06-15 | ||
| GBGB1610448.1A GB201610448D0 (en) | 2016-06-15 | 2016-06-15 | Adeno-associated viral vector system |
| GBGB1707261.2A GB201707261D0 (en) | 2017-05-05 | 2017-05-05 | Adeno-associated viral vector system |
| GB1707261.2 | 2017-05-05 | ||
| 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 (enExample) | 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)
| Country | Link |
|---|---|
| US (1) | US20190309326A1 (enExample) |
| EP (1) | EP3472328A1 (enExample) |
| JP (1) | JP2019523648A (enExample) |
| KR (1) | KR20190020745A (enExample) |
| CN (1) | CN109642242A (enExample) |
| AU (1) | AU2017286623A1 (enExample) |
| BR (1) | BR112018075855A2 (enExample) |
| CA (1) | CA3025445A1 (enExample) |
| IL (1) | IL263523A (enExample) |
| MX (1) | MX2018015629A (enExample) |
| RU (1) | RU2765826C2 (enExample) |
| SG (1) | SG11201811244SA (enExample) |
| WO (1) | WO2017216560A1 (enExample) |
Families Citing this family (14)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| EP2986635B1 (en) | 2013-04-18 | 2018-10-03 | Fondazione Telethon | Effective delivery of large genes by dual aav vectors |
| JP7240675B2 (ja) | 2017-05-05 | 2023-03-16 | ユニバーシティー オブ フロリダ リサーチ ファンデーション， インク． | オトフェルリンを発現させるための組成物および方法 |
| WO2019195726A1 (en) * | 2018-04-05 | 2019-10-10 | Oxford University Innovation Limited | Compositions and methods for the treatment of stargardt disease |
| JP2022512718A (ja) * | 2018-10-15 | 2022-02-07 | フォンダッツィオーネ・テレソン | インテインタンパク質及びその使用 |
| 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 |
| US20220267802A1 (en) * | 2019-07-15 | 2022-08-25 | President And Fellows Of Harvard College | Methods and compositions for gene delivery |
| JP7665609B2 (ja) | 2019-10-30 | 2025-04-21 | デシベル セラピューティクス インコーポレイテッド | オトフェリン二重ベクター系及びそれを含む感音難聴を治療するための組成物 |
| CA3174500A1 (en) * | 2020-04-01 | 2021-10-07 | University Of Florida Research Foundation, Incorporated | Dual aav-myo7a vectors with improved safety for the treatment of ush1b |
| JP2023537631A (ja) * | 2020-08-14 | 2023-09-04 | ケース ウエスタン リザーブ ユニバーシティ | 眼障害を処置するためのプラスミドベクターおよびナノ粒子 |
| 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 |
| WO2024257061A1 (en) * | 2023-06-16 | 2024-12-19 | 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 |
|---|---|---|---|---|
| AU2006304997B2 (en) * | 2005-10-20 | 2012-03-01 | Uniqure Ip B.V. | Improved AAV vectors produced in insect cells |
| US20090214478A1 (en) * | 2008-02-21 | 2009-08-27 | Alberto Auricchio | Method of treating ocular diseases by gene therapy |
| BR112012002628B1 (pt) * | 2009-08-07 | 2021-09-08 | Transgene Sa | Composição imunogênica, molécula de ácido nucleico, vetor, partícula viral infecciosa, micro-organismo transgênico, composição, usos da composição imunogênica e kit de partes |
| WO2013075008A1 (en) * | 2011-11-16 | 2013-05-23 | University Of Florida Research Foundation Inc. | Aav dual vector systems for gene therapy |
| EP2986635B1 (en) * | 2013-04-18 | 2018-10-03 | Fondazione Telethon | Effective delivery of large genes by dual aav vectors |
-
2017
- 2017-06-14 WO PCT/GB2017/051741 patent/WO2017216560A1/en not_active Ceased
- 2017-06-14 CN CN201780037639.9A patent/CN109642242A/zh active Pending
- 2017-06-14 AU AU2017286623A patent/AU2017286623A1/en not_active Abandoned
- 2017-06-14 KR KR1020197001281A patent/KR20190020745A/ko not_active Ceased
- 2017-06-14 US US16/309,428 patent/US20190309326A1/en not_active Abandoned
- 2017-06-14 EP EP17732175.9A patent/EP3472328A1/en not_active Withdrawn
- 2017-06-14 CA CA3025445A patent/CA3025445A1/en not_active Abandoned
- 2017-06-14 JP JP2018565324A patent/JP2019523648A/ja active Pending
- 2017-06-14 BR BR112018075855-8A patent/BR112018075855A2/pt not_active Application Discontinuation
- 2017-06-14 MX MX2018015629A patent/MX2018015629A/es unknown
- 2017-06-14 SG SG11201811244SA patent/SG11201811244SA/en unknown
- 2017-06-14 RU RU2019100525A patent/RU2765826C2/ru active
-
2018
- 2018-12-05 IL IL263523A patent/IL263523A/en unknown
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| RU2019100525A (ru) | Векторная система на основе аденоассоциированного вируса | |
| DE69429260T2 (de) | Adenovirale vektoren tierischen ursprungs und ihre verwendung bei der gentherapie | |
| JP2018520646A5 (enExample) | ||
| JP2018520997A5 (enExample) | ||
| JP2017512466A5 (enExample) | ||
| JP2020514286A5 (enExample) | ||
| JP2015508760A5 (enExample) | ||
| JP2020513814A5 (enExample) | ||
| KR20210028162A (ko) | 지대근 이영양증 2a형을 치료하기 위한 재조합 아데노 연관 바이러스 생성물 및 방법 | |
| JP2023517934A5 (enExample) | ||
| JP2013502225A5 (enExample) | ||
| Hollinger et al. | Viral vector-mediated gene therapies | |
| US20240002462A1 (en) | Treatment of neuropathy with igf-1-encoding dna constructs and hgf-encoding dna constructs | |
| JP2019524090A5 (enExample) | ||
| JP2021502978A5 (enExample) | ||
| JP2018509154A5 (enExample) | ||
| JP2017523239A5 (enExample) | ||
| IL308645A (en) | Viral vectors comprising rdh12 coding regions and methods of treating retinal dystrophies | |
| Chauhan et al. | AAV-DJ is superior to AAV9 for targeting brain and spinal cord, and de-targeting liver across multiple delivery routes in mice | |
| JP2020527167A5 (enExample) | ||
| JP2018526994A5 (enExample) | ||
| JP2022060514A (ja) | ガバペンチノイドからの干渉が減少し、ｈｇｆ異型体を発現するｄｎａコンストラクトを用いる神経病症の治療 | |
| JP2021500880A5 (enExample) | ||
| JP2020517268A5 (enExample) | ||
| US20150118187A1 (en) | Micro-organs providing sustained delivery of a therapeutic polypeptide and methods of use thereof |