Elucidating the Pathogenic Mechanism of Spinal Muscular Atrophy Through the Investigation of UTS2

We elucidated the role of the <i>UTS2</i> gene in an SMA cell model, emphasizing its dysregulation and identifying potential therapeutic targets. Urantide, a UTS2 inhibitor, had significant biological effects on the SMA cell model, indicating that it is a promising therapeutic strategy for SMA. These findings provide valuable insights for advancing drug development and clinical treatment of SMA.