Abstract
Pharmacological manipulation of gene expression brings new hope to the treatment of X-linked adrenoleukodystrophy ( pages 1261–1268 ).
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References
Holme, E. & Lindstedt, S. Tyrosinaemia type I and NTBC (2-(2-nitro-4-trifluoromethylbenzoyl)-1,3-cyclohexanedione. J. Inher. Metab. Dis. 21, 507– 517 (1998).
Barton, N.W. et al. Replacement therapy for inherited enzyme deficiency-marcophage-targeted glucocerebrosidase for Gaucher's disease. N. Engl. J. Med. 324, 1464–1470 (1991).
Kemp, S. et al. Gene redundancy and pharmacological gene therapy: Potential for X-linked Adrenoleukodystrophy. Nature Med. 4, 1261–1268 (1998).
Charache, S. et al. Treatment of sickle cell anemia with 5-azacytidine results in increased fetal hemoglobin production and is associated with nonrandom hypomethylation of DNA around the gamma-delta-beta-globin gene complex. Proc. Natl. Acad. Sci. USA 80, 4842– 4846 (1983).
Dover, G.J., Brusilow, S & Charache, S. Induction of fetal hemoglobin production in subjects with sickle cell anemia by oral sodium phenylbutyrate. Blood 84, 339–343 ( 1994).
Rubenstein, R.C., Egan, M.E. & Zeitlin, P.L. In vitro pharmacologic restoration of CFTR-mediated chloride transport with sodium 4-phenylbutyrate in cystic fibrosis epithelial cells containing delta F508-CFTR. J. Clin. Invest. 100, 2457–2465 (1997).
Rubenstein, R.C. & Zeitlin, P.L. A pilot clinical trial of oral sodium 4-phenylbutyrate (Buphenyl) in delta F508-homozygous cystic fibrosis patients: partial restoration of nasal epithelial CFTR function. Am. J. Respir. Crit. Care Med. 157, 484 –490 (1998).
Moser, H.W. Adrenoleukodystrophy: Phenotype, genetics, pathogenesis and therapy. Brain 120, 1485–1508 ( 1997).
Mosser, J. et al. Putative X-linked adrenoleukodystrophy gene shares unexpected homology with ABC transporters. Nature 361, 726–730 (1993).
Verleur, N., Hettema, E.H., van Roermund, C.W.T., Tabak, H.F. & Wanders, R.J.A. Transport of activated fatty acids by the peroxisomal ATP-binding-cassette transporter Pxa2 in a semi-intact yeast cell system. Eur. J. Biochem. 249, 657–661 (1997).
Acknowledgements
I thank J.M.F.G. Aerts, A.K. Groen, R.P.J. Oude Elferink and F.A. Wijburg for suggestions and M. Festen for preparation of the manuscript.
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Wanders, R. A happier sequel to Lorenzo's Oil?. Nat Med 4, 1245–1246 (1998). https://doi.org/10.1038/3225
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DOI: https://doi.org/10.1038/3225