Kay, Chris; Collins, Jennifer A.; Caron, Nicholas S.; Agostinho, Luciana de Andrade; Findlay-Black, Hailey; Casal, Lorenzo; Sumathipala, Dulika; Dissanayake, Vajira H. W.; Cornejo-Olivas, Mario; Baine, Fiona; Krause, Amanda; Greenberg, Jacquie L.; Paiva, Carmen Lucia Antao; Squitieri, Ferdinando; Hayden, Michael R.
(Elsevier, 2019)
Huntington disease (HD) is a fatal neurodegenerative disorder caused by a gain-of-function mutation in HTT. Suppression of mutant HTT has emerged as a leading therapeutic strategy for HD, with allele-selective approaches ...
