The present invention provides a pharmaceutical composition which causes skipping of the 53rd exon in the human dystrophin gene with a high efficiency.
The present invention provides an oligomer which efficiently enables to cause skipping of the 53rd exon in the human dystrophin gene.
本发明提供了一种药物组合物,其高效地导致人类肌营养不良症
基因中的第53外显子跳跃。本发明提供了一种寡核苷酸,其能够高效地导致人类肌营养不良症
基因中的第53外显子跳跃。