The present invention provides nucleic acid constructs, methods for identifying and validating compounds that increase the inclusion of exon 7 of SMN2 into mRNA transcribed from the SMN2 gene, compounds and pharmaceutical compositions that increase levels of SMN protein produced from the SMN2 gene, and methods for use thereof in treating of SMA.
本发明提供了核酸构建物,用于识别和验证增加SMN2
基因转录的mRNA中exon 7包含量的化合物的方法,增加从SMN2
基因产生的SMN蛋白
水平的化合物和药物组合物,以及在SMA治疗中使用它们的方法。