Anti-α-synuclein ASO delivered to monoamine neurons prevents α-synuclein accumulation in a Parkinson’s disease-like mouse model and in monkeys.
AlarcĂ³n-ArĂs D, Pavia-Collado R, Miquel-Rio L, Coppola-Segovia V, FerrĂ©s-Coy A, Ruiz-Bronchal E, GalofrĂ© M, Paz V, Campa L, Revilla R, Montefeltro A, Kordower JH, Vila M, Artigas F, Bortolozzi A. EBioMedicine. 2020 Aug 15;59:102944. Pubmed
Physiological impact and disease reversion for the severe form of centronuclear myopathy linked to Dynamin
Massana Muñoz X, Kretz C, Silva-Rojas R, Ochala J, Menuet A, Romero NB, Cowling BS, Laporte J. JCI Insight. 2020 Aug 18:137899. Pubmed
Prion protein lowering is a disease-modifying therapy across prion disease stages, strains and endpoints
Minikel EV, Zhao HT, Le J, O'Moore J, Pitstick R, Graffam S, Carlson GA, Kavanaugh MP, Kriz J, Kim JB, Ma J, Wille H, Aiken J, McKenzie D, Doh-Ura K, Beck M, O'Keefe R, Stathopoulos J, Caron T, Schreiber SL, Carroll JB, Kordasiewicz HB, Cabin ...