Postsymptomatic restoration of SMN rescues the disease phenotype in a mouse model of severe spinal muscular atrophy
在严重脊髓性肌萎缩症小鼠模型中,SMN 的症状后恢复可挽救疾病表型
期刊:Journal of Clinical Investigation
影响因子:13.3
doi:10.1172/JCI57291
Cathleen M Lutz, Shingo Kariya, Sunita Patruni, Melissa A Osborne, Don Liu, Christopher E Henderson, Darrick K Li, Livio Pellizzoni, José Rojas, David M Valenzuela, Andrew J Murphy, Margaret L Winberg, Umrao R Monani