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Bioluminescent imaging of a marking transgene and correction of Fabry mice by neonatal injection of recombinant lentiviral vectors

Successful therapy for many inherited disorders could be improved if the intervention were initiated early. This is especially true for lysosomal storage disorders. Earlier intervention may allow metabolic correction to occur before lipid buildup has irreversible consequences and/or before the immun...

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Autors principals: Yoshimitsu, Makoto, Sato, Takeya, Tao, Kesheng, Walia, Jagdeep S., Rasaiah, Vanessa I., Sleep, Gillian T., Murray, Gary J., Poeppl, Armando G., Underwood, John, West, Lori, Brady, Roscoe O., Medin, Jeffrey A.
Format: Artigo
Idioma:Inglês
Publicat: National Academy of Sciences 2004
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Accés en línia:https://ncbi.nlm.nih.gov/pmc/articles/PMC534735/
https://ncbi.nlm.nih.gov/pubmed/15550536
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1073/pnas.0407572101
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