Widespread and sustained target engagement in Huntington’s disease minipigs upon intrastriatal microRNA-based gene therapy

• DOI: 10.1126/scitranslmed.abb8920
• Publication Date: 2021-04-07T19:28:55Z
• Authors: Astrid Vallès; Melvin M. Evers; Anouk Stam; Marina Sogorb-Gon...; Cynthia Brouwers; Carlos Vendrell-T...; Seyda Acar-Broekmans; Lieke Paerels; Jiri Klima; Bozena Bohuslavova; Roberta Pintauro; Valentina Fodale; Alberto Bresciani; Roman Liscak; Dusan Urgosik; Zdenek Starek; Michal Crha; Bas Blits; Harald Petry; Zdenka Ellederova; Jan Motlik; Sander van Deventer; Pavlina Konstanti...
• Keywords: Huntingtin Protein

Striatal delivery of microRNA-gene therapy results in widespread brain huntingtin protein lowering Huntington’s disease minipigs up to 1 year.