Widespread and sustained target engagement in Huntington’s disease minipigs upon intrastriatal microRNA-based gene therapyAstrid Vallès, Pavlina Konstantinova, Melvin M. Evers et al.|Science Translational Medicine|2021Cited by 51
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
Gene Supplementation of MYO7A or activation of Myo7b for treatment of Usher syndrome 1BDavid Manuel Mittas, Elvir Bećirović, Klara Sonnie Hinrichsmeyer et al.|bioRxiv (Cold Spring Harbor Laboratory)|2026Cited by 0