The present invention provides methods for treating neurological diseases and disorders. Compounds that replace or enhance the function of SMN or alleviate or reduce a phenotype of cells with low SMN protein levels are provided. Screening methods and kits for identifying such compounds also are provided.
本发明提供了治疗神经系统疾病和失调的方法。本发明提供了替代或增强 SMN 功能或减轻或减少 SMN 蛋白
水平低的细胞表型的化合物。本发明还提供了用于鉴定此类化合物的筛选方法和试剂盒。