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

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

Alternative splicing is a process that enables a messenger RNA (mRNA) to direct synthesis of different protein variants (isoforms) that may have different cellular functions or properties.

who had one or two copies of the SMN2 backup gene, or two copies of the SMN2 backup gene, respectively. Breaking barriers to CAR T-cell therapy: The vision for expa... Live from ASCO 2025 ...

who have either been diagnosed with SMA type 1 - the most severe type - or have up to 3 copies of another gene known as SMN2. Around 550-600 babies are born with the disease in Europe each year ...

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 ...