Generation of two human induced pluripotent stem cell lines derived from two X-linked adrenoleukodystrophy patients with ABCD1 mutations

Adrenoleukodystrophy (ALD) is an X-linked genetic disorder, characterized by demyelination in the central nervous system and adrenal insufficiency. Human induced pluripotent stem cell (hiPSC) lines derived from two Japanese male patients with ALD were generated skin fibroblasts using retroviral vectors. The hiPSC showed self-renewal pluripotency, carried either a missense or nonsense mutation ABCD1 gene. Since molecular pathogenesis caused dysfunction remains unclear, these resources provide useful tools to establish disease models develop new therapies for X-ALD.