Gene therapy rescues disease phenotype in a spinal muscular atrophy with respiratory distress type 1 (SMARD1) mouse model
基因疗法可挽救脊髓性肌萎缩症伴呼吸窘迫1型(SMARD1)小鼠模型的疾病表型
期刊:Science Advances
影响因子:12.5
doi:10.1126/sciadv.1500078
Nizzardo, Monica; Simone, Chiara; Rizzo, Federica; Salani, Sabrina; Dametti, Sara; Rinchetti, Paola; Del Bo, Roberto; Foust, Kevin; Kaspar, Brian K; Bresolin, Nereo; Comi, Giacomo P; Corti, Stefania