Long-term in vitro correction of alpha-L-iduronidase deficiency (Hurler syndrome) in human bone marrow.

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...

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Detalhes bibliográficos
Main Authors: 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
Formato: Artigo
Idioma:Inglês
Publicado em: 1996
Assuntos:
Research Article
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC39903/
https://ncbi.nlm.nih.gov/pubmed/8700879
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