Cell-mediated cytotoxicity surrounding the brain in spinal muscular atrophy

We here longitudinally investigated how SMA and nusinersen shaped local immune responses in the cerebrospinal fluid (CSF) using single cell RNA-sequencing and single cell TCR-sequencing. Overall design: Single-cell RNA and TCR sequencing data of 23 individual CSF cell samples from 7 individual SMA patients that were longitudinally (baseline: n = 7; 2 months: n = 2; 6 months: n = 6; 10 months: n = 6) and 2 additional SMA patients at 10 months post_treatment.