Retroviral-mediated transfer of the human glucocerebrosidase gene into cultured Gaucher bone marrow.

Gaucher disease, a lysosomal glycolipid storage disorder, results from the genetic deficiency of an acidic glucosidase, glucocerebrosidase (GC). The beneficial effects of allogeneic bone marrow transplantation (BMT) for Gaucher disease suggest that GC gene transduction and the transplantation of aut...

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Pubblicato in:	J Clin Invest
Autori principali:	Nolta, J A, Yu, X J, Bahner, I, Kohn, D B
Natura:	Artigo
Lingua:	Inglês
Pubblicazione:	American Society for Clinical Investigation 1992
Soggetti:	Research Article
Accesso online:	https://ncbi.nlm.nih.govhttps://pmc.ncbi.nlm.nih.gov/articles/PMC443108/ https://ncbi.nlm.nih.govhttps://pubmed.ncbi.nlm.nih.gov/1379609/ https://ncbi.nlm.nih.govhttps://doi.org/10.1172/JCI115868
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