Development of a rabbit model for adrenoleukodystrophy: A pilot study on gene therapy using rAAV9.

X-linked adrenoleukodystrophy (X-ALD) is a common peroxisomal disorder caused by mutations in the gene, leading to the accumulation of very long-chain fatty acids (VLCFAs). This progressive neurodegenerative disease manifests in three primary forms: childhood-acquired cerebral demyelination (CALD), adult myelopathy (AMN), and primary adrenal cortical insufficiency. Bone marrow transplantation effectively halts disease progression only in the early stages of CALD.