Recombinant adeno-associated virus (rAAV)-mediated expression of a human gamma-globin gene in human progenitor-derived erythroid cells.

Effective gene therapy for the severe hemoglobin (Hb) disorders, sickle-cell anemia and thalassemia, will require an efficient method to transfer, integrate, and express a globin gene in primary erythroid cells. To evaluate recombinant adeno-associated virus (rAAV) for this purpose, we constructed a...

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Bibliographische Detailangaben
Hauptverfasser: Miller, J L, Donahue, R E, Sellers, S E, Samulski, R J, Young, N S, Nienhuis, A W
Format: Artigo
Sprache:Inglês
Veröffentlicht: 1994
Schlagworte:
Research Article
Online Zugang:https://ncbi.nlm.nih.gov/pmc/articles/PMC44982/
https://ncbi.nlm.nih.gov/pubmed/7524085
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