Targeting the 5' untranslated region of SMN2 as a therapeutic strategy for spinal muscular atrophy by Winkelsas, AM, Grunseich, C, Harmison, GG, Chwalenia, K, Rinaldi, C, Hammond, SM, Johnson, K, Bowerman, M, Arya, S, Talbot, K, Wood, MJ, Fischbeck, KH

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