[Research Articles] Widespread and sustained target engagement in Huntingtons disease minipigs upon intrastriatal microRNA-based gene therapy

This study demonstrates widespread biodistribution, strong and durable efficiency of rAAV5-miHTT in disease-relevant regions in a large brain, and the potential of using CSF analysis to determine vector expression and efficacy in the clinic.

Source: Science Translational Medicine - April 7, 2021 Category: Biomedical Science Authors: Valles, A., Evers, M. M., Stam, A., Sogorb-Gonzalez, M., Brouwers, C., Vendrell-Tornero, C., Acar-Broekmans, S., Paerels, L., Klima, J., Bohuslavova, B., Pintauro, R., Fodale, V., Bresciani, A., Liscak, R., Urgosik, D., Starek, Z., Crha, M., Blits, B., Pe Tags: Research Articles Source Type: research