Widespread and sustained target engagement in Huntington’s disease minipigs upon intrastriatal microRNA-based gene therapyAstrid Vallès, Pavlina Konstantinova, Jiřı́ Klı́ma et al.|Science Translational Medicine|2021Cited by 51
Analysis of mutant and total huntingtin expression in Huntington’s disease murine modelsValentina Fodale, Alberto Bresciani, Roberta Pintauro et al.|Scientific Reports|2020Cited by 33
Quantifying Huntingtin Protein in Human Cerebrospinal Fluid Using a Novel Polyglutamine Length-Independent AssayValentina Fodale, Alberto Bresciani, Roberta Pintauro et al.|Journal of Huntington s Disease|2022Cited by 9
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