Functional Intercellular Transmission of miHTT via Extracellular Vesicles: An In Vitro Proof-of-Mechanism Study

por Morais, Roberto D. V. S., Sogorb-González, Marina, Bar, Citlali, Timmer, Nikki C., Van der Bent, M. Leontien, Wartel, Morgane, Vallès, Astrid
Publicado 2022

AAV5-miHTT Lowers Huntingtin mRNA and Protein without Off-Target Effects in Patient-Derived Neuronal Cultures and Astrocytes

por Keskin, Sonay, Brouwers, Cynthia C., Sogorb-Gonzalez, Marina, Martier, Raygene, Depla, Josse A., Vallès, Astrid, van Deventer, Sander J., Konstantinova, Pavlina, Evers, Melvin M.
Publicado 2019

Cerebral Organoids: A Human Model for AAV Capsid Selection and Therapeutic Transgene Efficacy in the Brain

por Depla, Josse A., Sogorb-Gonzalez, Marina, Mulder, Lance A., Heine, Vivi M., Konstantinova, Pavlina, van Deventer, Sander J., Wolthers, Katja C., Pajkrt, Dasja, Sridhar, Adithya, Evers, Melvin M.
Publicado 2020

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

Development of an AAV-Based MicroRNA Gene Therapy to Treat Machado-Joseph Disease

por Martier, Raygene, Sogorb-Gonzalez, Marina, Stricker-Shaver, Janice, Hübener-Schmid, Jeannette, Keskin, Sonay, Klima, Jiri, Toonen, Lodewijk J., Juhas, Stefan, Juhasova, Jana, Ellederova, Zdenka, Motlik, Jan, Haas, Eva, van Deventer, Sander, Konstantinova, Pavlina, Nguyen, Huu Phuc, Evers, Melvin M.
Publicado 2019