Table 1.

Overview of studies

Vector	Delivery route	Dose	Purpose	Method of analysis
Self-complementary rAAV9-CBA-GFP	Intrathecal pump	1.5 × 1011 GC/mouse 3 × 1011 GC/mouse	Distribution of GFP across compartments and cell types of the nervous system	Immunofluorescence staining
Single-strand rAAV9-CBA-hABCD1	Intrathecal pump Intrathecal bolus	1 × 1011 GC/mouse	Distribution of ABCD1 in nervous system and peripheral organs Determination of the functionality of rAAV9-CBA-hABCD1	Western blot Lipid analysis by ESI-MS
Single-strand rAAV9-CBA-hABCD1-HA	Intrathecal pump Intrathecal bolus	2 × 1011 GC/mouse	Distribution of ABCD1-HA in nervous system and peripheral organs as well as identifying cell types targeted	Immunofluorescence staining

ABCD1, peroxisomal ATP-binding cassette transporter subfamily D member 1; ESI-MS, electrospray ionization-mass spectrometry; GC, genome copies; GFP, green fluorescent protein; HA, hemagglutinin.