Panelists discuss how splice modifiers work by enhancing protein production from the SMN2 gene, with risdiplam (Evrysdi) ...

Key Laboratory of Mariculture, Ocean University of China, Ministry of Education, Qingdao, China This study was carried out in accordance with the rules and approval of the respective Animal Research ...

Helmholtz Munich and the University of Sydney have identified biological mechanisms that are shared across psychiatric ...

For our high-throughput screening campaign, we utilized an NSC34 motor neuron cell line expressing an SMN2 minigene reporter ... via qPCR for dose responsiveness and to confirm the desired splicing ...