JP2025503868A5 - - Google Patents
Info
- Publication number
- JP2025503868A5 JP2025503868A5 JP2024541218A JP2024541218A JP2025503868A5 JP 2025503868 A5 JP2025503868 A5 JP 2025503868A5 JP 2024541218 A JP2024541218 A JP 2024541218A JP 2024541218 A JP2024541218 A JP 2024541218A JP 2025503868 A5 JP2025503868 A5 JP 2025503868A5
- Authority
- JP
- Japan
- Prior art keywords
- mirna
- seq
- polynucleotide
- polynucleotide according
- sequence
- 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 (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| GB2201242.1 | 2022-01-31 | ||
| GBGB2201242.1A GB202201242D0 (en) | 2022-01-31 | 2022-01-31 | Recombinant optimized mecp2 cassettes and methods for treating rett syndrome and related disorders |
| PCT/GB2023/050207 WO2023144565A1 (en) | 2022-01-31 | 2023-01-31 | Recombinant optimized mecp2 cassettes and methods for treating rett syndrome and related disorders |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2025503868A JP2025503868A (ja) | 2025-02-06 |
| JP2025503868A5 true JP2025503868A5 (https=) | 2026-03-12 |
Family
ID=80621152
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2024541218A Pending JP2025503868A (ja) | 2022-01-31 | 2023-01-31 | レット症候群及び関連障害を治療するための、組換え最適化ｍｅｃｐ２カセット及び方法 |
Country Status (11)
| Country | Link |
|---|---|
| US (1) | US20250136651A1 (https=) |
| EP (1) | EP4473121A1 (https=) |
| JP (1) | JP2025503868A (https=) |
| KR (1) | KR20240137089A (https=) |
| CN (1) | CN118696126A (https=) |
| AU (1) | AU2023213148A1 (https=) |
| CA (1) | CA3247763A1 (https=) |
| GB (1) | GB202201242D0 (https=) |
| IL (1) | IL314321A (https=) |
| MX (1) | MX2024008599A (https=) |
| WO (1) | WO2023144565A1 (https=) |
Families Citing this family (1)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2026017965A1 (en) * | 2024-07-19 | 2026-01-22 | The University Court Of The University Of Edinburgh | Recombinant therapeutic fxn constructs and methods of treating friedreich ataxia and related conditions |
Family Cites Families (27)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US5478745A (en) | 1992-12-04 | 1995-12-26 | University Of Pittsburgh | Recombinant viral vector system |
| US6204059B1 (en) | 1994-06-30 | 2001-03-20 | University Of Pittsburgh | AAV capsid vehicles for molecular transfer |
| CA2221293C (en) | 1995-06-07 | 2012-09-18 | University Of North Carolina At Chapel Hill | Helper virus-free aav production |
| WO1998011244A2 (en) | 1996-09-11 | 1998-03-19 | The Government Of The United States Of America, Represented By The Secretary, Department Of Health And Human Services | Aav4 vector and uses thereof |
| US6156303A (en) | 1997-06-11 | 2000-12-05 | University Of Washington | Adeno-associated virus (AAV) isolates and AAV vectors derived therefrom |
| US6989264B2 (en) | 1997-09-05 | 2006-01-24 | Targeted Genetics Corporation | Methods for generating high titer helper-free preparations of released recombinant AAV vectors |
| WO1999061601A2 (en) | 1998-05-28 | 1999-12-02 | The Government Of The United States Of America, As Represented By The Secretary, Department Of Health And Human Services | Aav5 vector and uses thereof |
| ATE362542T1 (de) | 1998-11-05 | 2007-06-15 | Univ Pennsylvania | Nukleinsäuresequenzen des adeno-assoziierten virus des serotyps i, und vektoren und wirtszellen, die diese enthalten |
| AU780231B2 (en) | 1998-11-10 | 2005-03-10 | University Of North Carolina At Chapel Hill, The | Virus vectors and methods of making and administering the same |
| ATE318923T1 (de) | 2000-06-01 | 2006-03-15 | Univ North Carolina | Doppelsträngige parvovirus-vektoren |
| US6723551B2 (en) | 2001-11-09 | 2004-04-20 | The United States Of America As Represented By The Department Of Health And Human Services | Production of adeno-associated virus in insect cells |
| EP2345731B1 (en) | 2003-09-30 | 2015-10-21 | The Trustees of the University of Pennsylvania | Adeno-associated virus (AAV) clades, sequences, vectors containing same, and uses thereof |
| JP5136766B2 (ja) | 2004-12-15 | 2013-02-06 | ユニバーシティ オブ ノース カロライナ アット チャペル ヒル | キメラベクター |
| ES2751999T3 (es) | 2008-01-29 | 2020-04-02 | Applied Genetic Tech Corporation | Producción recombinante de virus adeno-asociados usando células BHK en suspensión |
| WO2010093784A2 (en) | 2009-02-11 | 2010-08-19 | The University Of North Carolina At Chapel Hill | Modified virus vectors and methods of making and using the same |
| US8609392B2 (en) | 2009-05-12 | 2013-12-17 | Transgene S.A. | Method for orthopoxvirus production and purification |
| CA2853482C (en) | 2011-10-28 | 2020-05-19 | Joshua Grieger | Cell line for production of adeno-associated virus |
| HK1220488A1 (zh) | 2013-03-15 | 2017-05-05 | The Children's Hospital Of Philadelphia | 含有填充者/填充物多核苷酸序列的载体及其使用方法 |
| ES2926774T3 (es) | 2013-03-15 | 2022-10-28 | Univ North Carolina Chapel Hill | Métodos y composiciones para vectores AAV de doble unión de glicano |
| EP3003391B1 (en) | 2013-05-31 | 2021-09-22 | The Regents of The University of California | Adeno-associated virus variants and methods of use thereof |
| HK1222673A1 (zh) | 2013-07-12 | 2017-07-07 | The Children's Hospital Of Philadelphia | Aav载体和用於抗aav(腺相关病毒)中和抗体的检测 |
| IL297919A (en) | 2013-07-22 | 2023-01-01 | Childrens Hospital Philadelphia | Variant aav and compositions, methods and uses for gene transfer to cells, organs and tissues |
| CN106232618A (zh) | 2013-10-11 | 2016-12-14 | 马萨诸塞眼科耳科诊所 | 预测祖先病毒序列的方法及其用途 |
| GB201403684D0 (en) | 2014-03-03 | 2014-04-16 | King S College London | Vector |
| SG11201802408RA (en) | 2015-11-05 | 2018-05-30 | Bamboo Therapeutics Inc | Modified friedreich ataxia genes and vectors for gene therapy |
| US11680275B2 (en) * | 2017-06-06 | 2023-06-20 | University Of Massachusetts | Self-regulating AAV vectors for safe expression of MeCP2 in rett syndrome |
| MX2023000124A (es) | 2020-06-30 | 2023-03-08 | Univ Court Univ Of Edinburgh | Sistema de expresion de transgenes. |
-
2022
- 2022-01-31 GB GBGB2201242.1A patent/GB202201242D0/en not_active Ceased
-
2023
- 2023-01-31 CA CA3247763A patent/CA3247763A1/en active Pending
- 2023-01-31 US US18/832,458 patent/US20250136651A1/en active Pending
- 2023-01-31 AU AU2023213148A patent/AU2023213148A1/en active Pending
- 2023-01-31 CN CN202380019725.2A patent/CN118696126A/zh active Pending
- 2023-01-31 IL IL314321A patent/IL314321A/en unknown
- 2023-01-31 KR KR1020247028912A patent/KR20240137089A/ko active Pending
- 2023-01-31 WO PCT/GB2023/050207 patent/WO2023144565A1/en not_active Ceased
- 2023-01-31 JP JP2024541218A patent/JP2025503868A/ja active Pending
- 2023-01-31 MX MX2024008599A patent/MX2024008599A/es unknown
- 2023-01-31 EP EP23704219.7A patent/EP4473121A1/en active Pending
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