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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Argitaratua izan da:	Proc Natl Acad Sci U S A
Egile Nagusiak:	Miller, J L, Donahue, R E, Sellers, S E, Samulski, R J, Young, N S, Nienhuis, A W
Formatua:	Artigo
Hizkuntza:	Inglês
Argitaratua:	National Academy of Sciences 1994
Gaiak:	Research Article
Sarrera elektronikoa:	https://ncbi.nlm.nih.govhttps://pmc.ncbi.nlm.nih.gov/articles/PMC44982/ https://ncbi.nlm.nih.govhttps://pubmed.ncbi.nlm.nih.gov/7524085/ https://ncbi.nlm.nih.govhttps://doi.org/10.1073/pnas.91.21.10183
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