Intrathecal injection of rAAV9-CBA-hABCD1 leads to partial biochemical correction in Abcd1–/– mouse spinal cord. (a–c) C26:0 levels in spinal cord, DRG, and liver after intrathecal osmotic pump (IT pump) and intrathecal bolus (IT bolus) delivery of rAAV9-CBA-hABCD1 at 1 × 1011 GC/mouse. (d–f) C24:0 levels in spinal cord, DRG, and liver after intrathecal osmotic pump and bolus delivery at 1 × 1011 GC/mouse. Data are expressed as means ± SEM. ***p < 0.001 compared with wild type; #p < 0.05, ##p < 0.01 compared with PBS control. DRG, dorsal root ganglia; GC, genome copies; PBS, phosphate-buffered saline; WT, wild type.