Our understanding of disease pathogenesis has been hampered by the lack of a useful disease model for ALD. Based on our research, we hypothesized that increasing the endogenous VLCFA synthesis in the central nervous system would impact the clinical phenotype of the ALD mouse. We developed the “ALD 2.0” mouse which has increased VLCFA synthesis and levels in oligodendrocytes and Schwann cells. Our results indicate that these mice develop AMN. The ALD 2.0 mouse provides a valuable model for the development of a roadmap towards the unravelling of disease pathogenesis.
