Selection of transduced CD34+ progenitors and enzymatic correction of cells from Gaucher patients, with bicistronic vectors.

The gene transfer efficiency of human hematopoietic stem cells is still inadequate for efficient gene therapy of most disorders. To overcome this problem, a selectable retroviral vector system for gene therapy has been developed for gene therapy of Gaucher disease. We constructed a bicistronic retro...

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Pubblicato in:	Proc Natl Acad Sci U S A
Autori principali:	Migita, M, Medin, J A, Pawliuk, R, Jacobson, S, Nagle, J W, Anderson, S, Amiri, M, Humphries, R K, Karlsson, S
Natura:	Artigo
Lingua:	Inglês
Pubblicazione:	National Academy of Sciences 1995
Soggetti:	Research Article
Accesso online:	https://ncbi.nlm.nih.govhttps://pmc.ncbi.nlm.nih.gov/articles/PMC40299/ https://ncbi.nlm.nih.govhttps://pubmed.ncbi.nlm.nih.gov/8618847/ https://ncbi.nlm.nih.govhttps://doi.org/10.1073/pnas.92.26.12075
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