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Article

Regulated high level expression of a human gamma-globin gene introduced into erythroid cells by an adeno-associated virus vector.

1 Aug 1992

Abstract excerpt

Gene therapy of severe hemoglobinopathies will require high-level expression of a transferred globin gene in erythroid cells. Distant regulatory elements flanking the beta-globin gene cluster, the locus control region, are needed for appropriate expression. We have explored the use of a human parvovirus, the adeno-associated virus (AAV), for globin gene transfer. The human A gamma-globin gene, linked to...

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PMID: 1323131
DOI: 10.1073/pnas.89.15.7257

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