ES2680915T3 - Plataforma de fabricación escalable para la purificación de vectores virales y vectores virales purificados de este modo para su utilización en terapia génica - Google Patents
Plataforma de fabricación escalable para la purificación de vectores virales y vectores virales purificados de este modo para su utilización en terapia génica Download PDFInfo
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| US9217155B2 (en) | 2008-05-28 | 2015-12-22 | University Of Massachusetts | Isolation of novel AAV'S and uses thereof |
| WO2010138263A2 (en) | 2009-05-28 | 2010-12-02 | University Of Massachusetts | Novel aav 's and uses thereof |
| JP2013517798A (ja) * | 2010-01-28 | 2013-05-20 | ザ チルドレンズ ホスピタル オブ フィラデルフィア | ウイルスベクター精製における拡張可能な製造プラットフォームおよび遺伝子治療における使用のための高純度ウイルスベクター |
| WO2011133890A1 (en) | 2010-04-23 | 2011-10-27 | University Of Massachusetts | Cns targeting aav vectors and methods of use thereof |
| WO2011133874A1 (en) | 2010-04-23 | 2011-10-27 | University Of Massachusetts | Multicistronic expression constructs |
| JP6198278B2 (ja) * | 2011-09-08 | 2017-09-20 | ユニキュアー アイピー ビー．ブイ． | Ａａｖ調製物からの混入ウイルスの除去 |
| EP4282419A1 (en) | 2012-12-20 | 2023-11-29 | Purdue Research Foundation | Chimeric antigen receptor-expressing t cells as anti-cancer therapeutics |
| FR3002237B1 (fr) * | 2013-02-15 | 2017-12-15 | Genethon | Methodes pour la production de particules virales aav double brin |
| ES2690643T3 (es) | 2013-11-26 | 2018-11-21 | The United States Of America, As Represented By The Secretary, Department Of Health And Human Services | Vectores virales adeno-asociados para el tratamiento de enfermedad de almacenamiento de glucógeno |
| GB201401707D0 (en) | 2014-01-31 | 2014-03-19 | Sec Dep For Health The | Adeno-associated viral vectors |
| US10072251B2 (en) | 2014-02-19 | 2018-09-11 | University Of Massachusetts | Recombinant AAVS having useful transcytosis properties |
| US10280418B2 (en) | 2014-03-18 | 2019-05-07 | Univeristy Of Massachusetts | RAAV-based compositions and methods for treating amyotrophic lateral sclerosis |
| CN115252823A (zh) * | 2014-03-21 | 2022-11-01 | 建新公司 | 用于色素性视网膜炎的基因疗法 |
| WO2015164786A1 (en) | 2014-04-25 | 2015-10-29 | University Of Massachusetts | Recombinant aav vectors useful for reducing immunity against transgene products |
| US11555059B2 (en) | 2014-04-25 | 2023-01-17 | The Trustees Of The University Of Pennsylvania | LDLR variants and their use in compositions for reducing cholesterol levels |
| WO2015187825A2 (en) | 2014-06-03 | 2015-12-10 | University Of Massachusetts | Compositions and methods for modulating dysferlin expression |
| US10370432B2 (en) | 2014-10-03 | 2019-08-06 | University Of Massachusetts | Heterologous targeting peptide grafted AAVS |
| JP6842410B2 (ja) | 2014-10-03 | 2021-03-17 | ユニバーシティ オブ マサチューセッツ | 新規の高効率ライブラリーにより同定されるａａｖベクター |
| AU2015335923B2 (en) | 2014-10-21 | 2021-04-29 | University Of Massachusetts | Recombinant AAV variants and uses thereof |
| EP3591070B1 (en) * | 2014-11-28 | 2022-12-21 | uniQure IP B.V. | Dna impurities in a composition comprising a parvoviral virion |
| CA2972038C (en) | 2014-12-23 | 2023-05-16 | The United States Of America, As Represented By The Secretary, Department Of Health And Human Services | Adeno-associated virus vectors encoding modified g6pc and uses thereof |
| US9862936B2 (en) | 2015-01-13 | 2018-01-09 | Alfa Wassermann, Inc. | Methods of purifying adeno-associated virus (AAV) and/or recombinant adeno-associated virus (rAAV) and gradients and flow-through buffers therefore |
| EP3054007A1 (en) | 2015-02-09 | 2016-08-10 | Institut National De La Sante Et De La Recherche Medicale (Inserm) | Recombinant adeno-associated virus particle purification comprising an immuno-affinity purification step |
| EP3054006A1 (en) | 2015-02-09 | 2016-08-10 | Institut National De La Sante Et De La Recherche Medicale (Inserm) | Recombinant adeno-associated virus particle purification with multiple-step anion exchange chromatography |
| US10584321B2 (en) | 2015-02-13 | 2020-03-10 | University Of Massachusetts | Compositions and methods for transient delivery of nucleases |
| EP3262162A4 (en) * | 2015-02-23 | 2018-08-08 | Voyager Therapeutics, Inc. | Regulatable expression using adeno-associated virus (aav) |
| EP3283126B1 (en) | 2015-04-16 | 2019-11-06 | Emory University | Recombinant promoters and vectors for protein expression in liver and use thereof |
| ES2856936T3 (es) | 2015-04-23 | 2021-09-28 | Univ Washington State | Suministro del gen Smad7 como un agente terapéutico |
| WO2016172008A1 (en) | 2015-04-24 | 2016-10-27 | University Of Massachusetts | Modified aav constructions and uses thereof |
| CA3002980A1 (en) | 2015-10-22 | 2017-04-27 | University Of Massachusetts | Prostate-targeting adeno-associated virus serotype vectors |
| ES2978086T3 (es) | 2015-10-22 | 2024-09-05 | Univ Massachusetts | Terapia génica con aspartoacilasa en el tratamiento de enfermedad de Canavan |
| CN105420275A (zh) * | 2015-11-27 | 2016-03-23 | 中国科学院苏州生物医学工程技术研究所 | 制备外源功能基因定点整合的人神经干细胞的方法 |
| ES2918998T3 (es) | 2015-12-11 | 2022-07-21 | Univ Pennsylvania | Método de purificación escalable para AAVrh10 |
| WO2017100674A1 (en) | 2015-12-11 | 2017-06-15 | The Trustees Of The University Of Pennsylvania | Scalable purification method for aav1 |
| US11015174B2 (en) * | 2015-12-11 | 2021-05-25 | The Trustees Of The University Of Pennsylvania | Scalable purification method for AAV8 |
| US11098286B2 (en) * | 2015-12-11 | 2021-08-24 | The Trustees Of The University Of Pennsylvania | Scalable purification method for AAV9 |
| US10889832B2 (en) * | 2015-12-11 | 2021-01-12 | The Trustees Of The University Of Pennsylvania | Gene therapy for treating familial hypercholesterolemia |
| JP7128741B2 (ja) | 2015-12-18 | 2022-08-31 | サンガモ セラピューティクス， インコーポレイテッド | Ｔ細胞受容体の標的化破壊 |
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