Since the overexpression of Abcd2/Aldrp resulted in complete correction of VLCFA β-oxidation in X-ALD fibroblasts [38] and the prevention of the VLCFA accumulation, axonal degeneration and clinical symptoms in Abcd1 knockout mice [34], Abcd2/Aldrp is an attractive candidate gene for pharmacological gene therapy. This evidence concerns the gene ABCD2 and X-linked adrenoleukodystrophy.