Widespread and sustained target engagement in Huntington’s disease minipigs upon intrastriatal microRNA-based gene therapyAstrid Vallès, Pavlina Konstantinova, Seyda Acar-Broekmans et al.|Science Translational Medicine|2021Cited by 51
AAV5-miHTT-mediated huntingtin lowering improves brain health in a Huntington’s disease mouse modelSarah B. Thomson, Blair R. Leavitt, Anouk Stam et al.|Brain|2022Cited by 25
I05 Sustained mutant huntingtin lowering in the brain and cerebrospinal fluid of huntington disease minipigs mediated by AAV5-MIHTT gene therapyAstrid Valls, Pavlina Konstantinova, Cynthia Brouwers et al.|Unknown|2018Cited by 3