Table of Contents
Washington University Experience | MYELIN (NON-IMMUNE MEDIATED) | Adrenoleukodystrophy (ALD, AMN) | 1G0 Denouement Case 1
1G0 Denouement The current thinking is that damage, particularly lipid peroxidation in ALD, is the result of oxidative stress. Several mouse models have been developed, particularly of the adrenomyeloneuropathy pattern of disease. ALD progression can be halted by allogeneic hematopoietic cell transplantation. A lentiviral vector encoding wild-type ABCD1 re-infused into ALD patients after myeloablative treatment has been able to inhibit progression of myelinopathy. The clinical pattern of this patient’s disease, the presence of a number of patients in his family with a pattern suggestive of adrenomyeloneuropathy, and presence of posterior column spinal cord degeneration in the current patient may reflect a family whose members seemingly express both disease variants, which has been reported previously.
