AAV5-miHTT Gene Therapy Demonstrates Sustained Huntingtin Lowering and Functional Improvement in Huntington Disease Mouse Models

por Spronck, Elisabeth A., Brouwers, Cynthia C., Vallès, Astrid, de Haan, Martin, Petry, Harald, van Deventer, Sander J., Konstantinova, Pavlina, Evers, Melvin M.
Publicado 2019

Therapeutic hFIX Activity Achieved after Single AAV5-hFIX Treatment in Hemophilia B Patients and NHPs with Pre-existing Anti-AAV5 NABs

por Majowicz, Anna, Nijmeijer, Bart, Lampen, Margit H., Spronck, Lisa, de Haan, Martin, Petry, Harald, van Deventer, Sander J., Meyer, Christian, Tangelder, Marco, Ferreira, Valerie
Publicado 2019

Enhanced Factor IX Activity following Administration of AAV5-R338L “Padua” Factor IX versus AAV5 WT Human Factor IX in NHPs

por Spronck, Elisabeth A., Liu, Ying Poi, Lubelski, Jacek, Ehlert, Erich, Gielen, Sander, Montenegro-Miranda, Paula, de Haan, Martin, Nijmeijer, Bart, Ferreira, Valerie, Petry, Harald, van Deventer, Sander J.
Publicado 2019

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

por Spronck, Elisabeth A., Vallès, Astrid, Lampen, Margit H., Montenegro-Miranda, Paula S., Keskin, Sonay, Heijink, Liesbeth, Evers, Melvin M., Petry, Harald, van Deventer, Sander J., Konstantinova, Pavlina, de Haan, Martin
Publicado 2021

Secreted therapeutics: monitoring durability of microRNA-based gene therapies in the central nervous system

por Sogorb-Gonzalez, Marina, Vendrell-Tornero, Carlos, Snapper, Jolanda, Stam, Anouk, Keskin, Sonay, Miniarikova, Jana, Spronck, Elisabeth A, de Haan, Martin, Nieuwland, Rienk, Konstantinova, Pavlina, van Deventer1, Sander J, Evers, Melvin M, Vallès, Astrid
Publicado 2021