Published in
American Association for the Advancement of Science, Science Translational Medicine, 588(13), 2021
DOI: 10.1126/scitranslmed.abb8920
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Widespread and sustained target engagement in Huntington’s disease minipigs upon intrastriatal microRNA-based gene therapy
Journal article published in 2021 by
Astrid Vallès 
,
Melvin M. Evers ,
Anouk Stam,
Marina Sogorb-Gonzalez ,
Cynthia Brouwers ,
Carlos Vendrell-Tornero,
Seyda Acar-Broekmans,
Lieke Paerels ,
Jiri Klima ,
Bozena Bohuslavova,
Roberta Pintauro ,
Valentina Fodale,
Alberto Bresciani ,
Roman Liscak,
Dusan Urgosik
and other authors.
,
Melvin M. Evers ,
Anouk Stam,
Marina Sogorb-Gonzalez ,
Cynthia Brouwers ,
Carlos Vendrell-Tornero,
Seyda Acar-Broekmans,
Lieke Paerels ,
Jiri Klima ,
Bozena Bohuslavova,
Roberta Pintauro ,
Valentina Fodale,
Alberto Bresciani ,
Roman Liscak,
Dusan Urgosik
and other authors.
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Abstract
Striatal delivery of microRNA-gene therapy results in widespread brain huntingtin protein lowering in Huntington’s disease minipigs up to 1 year.