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Lorenzo's oil: the sequel
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The film Lorenzo's Oil dramatically illustrated the predicament of families where there is a child with an untreatable inherited metabolic disease. The oil devised as treatment by the father of a child with X-linked adrenoleukodystrophy (X-ALD) has proved disappointing but new research (Stephen Kemp and colleagues. Nature Medicine 1998;4:1261-8. See also News and Views, pages 1245-6) brings hope of effective treatment and illustrates a new direction in gene therapy.
There are probably many redundant genes in animal and human cells, which have the capacity to bring into play alternative metabolic pathways. Chemical agents are capable of stimulating the expression of such genes and investigators at Johns Hopkins University have shown that 4-phenylbutyrate (4PBA) may be such an agent for X-ALD cells.
The defect in X-ALD is in the gene
encoding the peroxisomal membrane protein, ALDP (adrenoleukodystrophy
protein). What the Baltimore researchers have shown is that 4PBA
treatment of
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