AAV5-miHTT Gene Therapy Demonstrates Sustained Huntingtin Lowering and Functional Improvement in Huntington Disease Mouse Models
Spronck, Elisabeth A., Brouwers, Cynthia C., Vallès, Astrid, de Haan, Martin, Petry, Harald, van Deventer, Sander J., Konstantinova, Pavlina, Evers, Melvin M.
Published in Molecular therapy. Methods & clinical development (14.06.2019)
Published in Molecular therapy. Methods & clinical development (14.06.2019)
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Enhanced Factor IX Activity following Administration of AAV5-R338L “Padua” Factor IX versus AAV5 WT Human Factor IX in NHPs
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.
Published in Molecular therapy. Methods & clinical development (13.12.2019)
Published in Molecular therapy. Methods & clinical development (13.12.2019)
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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
Spronck, Elisabeth A, Vallès, Astrid, Lampen, Margit H, Montenegro-Miranda, Paula S, Keskin, Sonay, Heijink, Liesbeth, Evers, Melvin M, Petry, Harald, Deventer, Sander J van, Konstantinova, Pavlina, Haan, Martin de
Published in Brain sciences (20.01.2021)
Published in Brain sciences (20.01.2021)
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Secreted therapeutics: monitoring durability of microRNA-based gene therapies in the central nervous system
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
Published in Brain communications (01.01.2021)
Published in Brain communications (01.01.2021)
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Preclinical efficacy and safety of adeno-associated virus 5 alpha-galactosidase A gene therapy for Fabry disease
Liefhebber, Jolanda M.P., Brasser, Giso, Spronck, Elisabeth A., Ottenhoff, Roelof, Paerels, Lieke, Ferraz, Maria J., Schwarz, Lukas K., Efthymiopoulou, Nikoleta, Kuo, Chi-Lin, Montenegro-Miranda, Paula S., Evers, Melvin M., Aerts, Johannes M.F.G., Liu, Ying Poi
Published in Molecular therapy. Methods & clinical development (01.11.2024)
Published in Molecular therapy. Methods & clinical development (01.11.2024)
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