CA2787827C - A scalable manufacturing platform for viral vector purification and viral vectors so purified for use in gene therapy - Google Patents
A scalable manufacturing platform for viral vector purification and viral vectors so purified for use in gene therapy Download PDFInfo
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- CA2787827C CA2787827C CA2787827A CA2787827A CA2787827C CA 2787827 C CA2787827 C CA 2787827C CA 2787827 A CA2787827 A CA 2787827A CA 2787827 A CA2787827 A CA 2787827A CA 2787827 C CA2787827 C CA 2787827C
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- aav vector
- aav
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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 |
| MX340102B (es) * | 2010-01-28 | 2016-06-24 | The Children's Hospital Of Philadelphia * | Una plataforma de fabricacion ajustable en escala, para la purificacion de vector viral y vectores virales asi purificados para el uso en terapia de genes. |
| WO2011133874A1 (en) | 2010-04-23 | 2011-10-27 | University Of Massachusetts | Multicistronic expression constructs |
| US9102949B2 (en) | 2010-04-23 | 2015-08-11 | University Of Massachusetts | CNS targeting AAV vectors and methods of use thereof |
| WO2013036118A1 (en) * | 2011-09-08 | 2013-03-14 | Uniqure Ip B.V. | Removal of contaminating viruses from aav preparations |
| ES2949648T3 (es) | 2012-12-20 | 2023-10-02 | Purdue Research Foundation | Células T que expresan un receptor antigénico quimérico como terapia contra el cáncer |
| FR3002237B1 (fr) * | 2013-02-15 | 2017-12-15 | Genethon | Methodes pour la production de particules virales aav double brin |
| CA2930872C (en) | 2013-11-26 | 2022-05-31 | University Of Florida Research Foundation, Incorporated | Adeno-associated virus vectors for treatment of glycogen storage disease |
| GB201401707D0 (en) | 2014-01-31 | 2014-03-19 | Sec Dep For Health The | Adeno-associated viral vectors |
| WO2015127128A2 (en) | 2014-02-19 | 2015-08-27 | University Of Massachusetts | Recombinant aavs having useful transcytosis properties |
| AU2015231294B2 (en) | 2014-03-18 | 2020-10-29 | University Of Massachusetts | rAAV-based compositions and methods for treating amyotrophic lateral sclerosis |
| SI3119437T1 (sl) * | 2014-03-21 | 2020-01-31 | Genzyme Corporation | Genska terapija za retinitis pigmentoza |
| JP6741590B2 (ja) | 2014-04-25 | 2020-08-19 | ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア | コレステロールレベルを低下させるためのｌｄｌｒ変異体および組成物中でのそれらの使用 |
| US10975391B2 (en) | 2014-04-25 | 2021-04-13 | University Of Massachusetts | Recombinant AAV vectors useful for reducing immunity against transgene products |
| US10689653B2 (en) | 2014-06-03 | 2020-06-23 | University Of Massachusetts | Compositions and methods for modulating dysferlin expression |
| WO2016054557A1 (en) | 2014-10-03 | 2016-04-07 | University Of Massachusetts | Novel high efficiency library-identified aav vectors |
| WO2016054554A1 (en) | 2014-10-03 | 2016-04-07 | University Of Massachusetts | Heterologous targeting peptide grafted aavs |
| CA2964272A1 (en) | 2014-10-21 | 2016-04-28 | Guangping Gao | Recombinant aav variants and uses thereof |
| WO2016083560A1 (en) * | 2014-11-28 | 2016-06-02 | Uniqure Ip B.V. | Dna impurities in a composition comprising a parvoviral virion |
| US10415044B2 (en) | 2014-12-23 | 2019-09-17 | 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 |
| EP3245218A4 (en) | 2015-01-13 | 2018-10-03 | Alfa Wassermann, Inc. | Methods of purifying adeno-associated virus (aav) and/or recombinant adeno-associated virus (raav) and gradients and flow-through buffers therefore |
| 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 |
| 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 |
| 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) |
| CN108093639B (zh) | 2015-04-16 | 2022-07-19 | 埃默里大学 | 用于肝脏中蛋白质表达的重组启动子和载体及其用途 |
| ES2856936T3 (es) | 2015-04-23 | 2021-09-28 | Univ Washington State | Suministro del gen Smad7 como un agente terapéutico |
| CA3021949C (en) | 2015-04-24 | 2023-10-17 | University Of Massachusetts | Modified aav constructs and uses thereof |
| DK3364997T5 (da) | 2015-10-22 | 2024-09-30 | Univ Massachusetts | Aspartoacylase genterapi til behandling af canavans sygdom |
| CA3002980A1 (en) | 2015-10-22 | 2017-04-27 | University Of Massachusetts | Prostate-targeting adeno-associated virus serotype vectors |
| 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 |
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