Figure 2.
Distribution of ABCD1 protein after intrathecal delivery of rAAV9-CBA-hABCD1. (a) Representative Western blot images show ABCD1 expression in various organs of Abcd1–/– mice after intrathecal osmotic pump (IT pump) and intrathecal bolus (IT bolus) delivery at various vector doses and (b) quantification of protein intensity after normalization to β-actin. (c) Representative Western blot images show rAAV9-CBA-hABCD1 expression in various organs after intravenous (IV) injection in spinal cord and heart tissue and (d) protein intensity comparison between IT pump and IV injection. Results are expressed as means ± SEM of the various animals: *p < 0.05, ***p < 0.001. ABCD1, peroxisomal ATP-binding cassette transporter subfamily D member 1; GC, genome copies; DRG, dorsal root ganglia; PBS, phosphate-buffered saline; SC, spinal cord; SN, sciatic nerve; WT, wild type.
