From gene to therapy in spinal and bulbar muscular atrophy: Are we there yet? by Pennuto, M, Rinaldi, C

Beyond motor neurons: expanding the clinical spectrum in Kennedy’s disease by Manzano, R, Sorarú, G, Grunseich, C, Fratta, P, Zuccaro, E, Pennuto, M, Rinaldi, C

AR cooperates with SMAD4 to maintain skeletal muscle homeostasis by Forouhan, M, Lim, WF, Zanetti-Domingues, LC, Tynan, CJ, Roberts, TC, Malik, B, Manzano, R, Speciale, AA, Ellerington, R, Garcia-Guerra, A, Fratta, P, Sorarú, G, Greensmith, L, Pennuto, M, Wood, MJA, Rinaldi, C

Gene therapy with AR isoform 2 rescues spinal and bulbar muscular atrophy phenotype by modulating AR transcriptional activity by Lim, WF, Forouhan, M, Roberts, TC, Dabney, J, Ellerington, R, Speciale, AA, Manzano, R, Lieto, M, Sangha, G, Banerjee, S, Conceição, M, Cravo, L, Biscans, A, Roux, L, Pourshafie, N, Grunseich, C, Duguez, S, Khvorova, A, Pennuto, M, Cortes, CJ, La Spada, AR, Fischbeck, KH, Wood, MJA, Rinaldi, C