Article
Long-term in vitro correction of alpha-L-iduronidase deficiency (Hurler syndrome) in human bone marrow.
Proceedings of the National Academy of Sciences of the United States of America - 5 Mar 1996
Fairbairn L J, Lashford L S, Spooncer E, McDermott R H, Lebens G, Arrand J E, Arrand J R, Bellantuono I, Holt R, Hatton C E, Cooper A, Besley G T, Wraith J E, Anson D S, Hopwood J J, Dexter T M
Abstract excerpt
Allogeneic bone marrow transplantation is the most effective treatment for Hurler syndrome but, since this therapy is not available to all patients, we have considered an alternative approach based on transfer and expression of the normal gene in autologous bone marrow. A retroviral vector carrying the full-length cDNA for alpha-L-iduronidase has been constructed and used to transduce bone marrow from patients...
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