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

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

Potent and sustained huntingtin lowering via AAV5 encoding miRNA preserves striatal volume and cognitive function in a humanized mouse model of Huntington disease

por Caron, Nicholas S, Southwell, Amber L, Brouwers, Cynthia C, Cengio, Louisa Dal, Xie, Yuanyun, Black, Hailey Findlay, Anderson, Lisa M, Ko, Seunghyun, Zhu, Xiang, van Deventer, Sander J, Evers, Melvin M, Konstantinova, Pavlina, Hayden, Michael R
Publicado 2020