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In-Depth Characterization of a Mifepristone-Regulated Expression System for AAV5-Mediated Gene Therapy in the Liver by Jolanda M. Liefhebber, Raygene Martier, Tom Van der Zon, Sonay Keskin, Angelina Huseinovic, Jacek Lubelski, Bas Blits, Harald Petry, Pavlina Konstantinova
Published 2019-06-01
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Intrastriatal Administration of AAV5-miHTT in Non-Human Primates and Rats Is Well Tolerated and Results in miHTT Transgene Expression in Key Areas of Huntington Disease Pathology by Elisabeth A. Spronck, Astrid Vallès, Margit H. Lampen, Paula S. Montenegro-Miranda, Sonay Keskin, Liesbeth Heijink, Melvin M. Evers, Harald Petry, Sander J. van Deventer, Pavlina Konstantinova, Martin de Haan
Published 2021-01-01
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Development of an AAV-Based MicroRNA Gene Therapy to Treat Machado-Joseph Disease by Raygene Martier, Marina Sogorb-Gonzalez, Janice Stricker-Shaver, Jeannette Hübener-Schmid, Sonay Keskin, Jiri Klima, Lodewijk J. Toonen, Stefan Juhas, Jana Juhasova, Zdenka Ellederova, Jan Motlik, Eva Haas, Sander van Deventer, Pavlina Konstantinova, Huu Phuc Nguyen, Melvin M. Evers
Published 2019-12-01
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