Skip to main content

Thank you for visiting nature.com. You are using a browser version with limited support for CSS. To obtain the best experience, we recommend you use a more up to date browser (or turn off compatibility mode in Internet Explorer). In the meantime, to ensure continued support, we are displaying the site without styles and JavaScript.

  • News & Views
  • Published:

A happier sequel to Lorenzo's Oil?

Nature Medicine volume 4, pages 1245–1246 (1998)Cite this article

Abstract

Pharmacological manipulation of gene expression brings new hope to the treatment of X-linked adrenoleukodystrophy ( pages 1261–1268 ).

This is a preview of subscription content, access via your institution

Access options

Buy this article

  • Purchase on Springer Link
  • Instant access to full article PDF
Buy now

Prices may be subject to local taxes which are calculated during checkout

References

  1. 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).

    Article  CAS  Google Scholar 

  2. 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).

    Article  CAS  Google Scholar 

  3. Kemp, S. et al. Gene redundancy and pharmacological gene therapy: Potential for X-linked Adrenoleukodystrophy. Nature Med. 4, 1261–1268 (1998).

    Article  CAS  Google Scholar 

  4. 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).

    Article  CAS  Google Scholar 

  5. 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).

    CAS  PubMed  Google Scholar 

  6. 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).

    Article  CAS  Google Scholar 

  7. 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).

    Article  CAS  Google Scholar 

  8. Moser, H.W. Adrenoleukodystrophy: Phenotype, genetics, pathogenesis and therapy. Brain 120, 1485–1508 ( 1997).

    Article  Google Scholar 

  9. Mosser, J. et al. Putative X-linked adrenoleukodystrophy gene shares unexpected homology with ABC transporters. Nature 361, 726–730 (1993).

    Article  CAS  Google Scholar 

  10. 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).

    Article  CAS  Google Scholar 

Download references

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.

Author information

Authors and Affiliations

  1. Academic Medical Centre Department of Pediatrics, Univ. of Amsterdam, 1100 DE Amsterdam, The Netherlands

    Ronald J. A. Wanders

Authors
  1. Ronald J. A. Wanders
    View author publications

    You can also search for this author in PubMed Google Scholar

Rights and permissions

Reprints and permissions

About this article

Cite this article

Wanders, R. A happier sequel to Lorenzo's Oil?. Nat Med 4, 1245–1246 (1998). https://doi.org/10.1038/3225

Download citation

  • Issue Date:

  • DOI: https://doi.org/10.1038/3225

Search

Advanced search

Quick links

Nature Briefing

Sign up for the Nature Briefing newsletter — what matters in science, free to your inbox daily.

Get the most important science stories of the day, free in your inbox. Sign up for Nature Briefing