Intrathecal administration of a novel siRNA modality extends survival and improves motor function in the SOD1G93A ALS mouse model

Accessory oligos for neuronal delivery of therapeutic siRNAs for ALS

“…Fortunately, a recent publication by Place, Li, and colleagues proposes an elegant solution for this barrier. 4 These investigators developed a panel of SOD1 siRNAs that can degrade most SOD1 isoforms. They first tested these siRNAS in vitro , where they identified the top 5 performing siRNAs with potency in the low picomolar range.…”