Article
Functional requirements for phenotypic correction of murine beta-thalassemia: implications for human gene therapy.
Blood - 15 May 2001
Persons D A, Allay E R, Sabatino D E, Kelly P, Bodine D M, Nienhuis A W
Abstract excerpt
As initial human gene therapy trials for beta-thalassemia are contemplated, 2 critical questions important to trial design and planning have emerged. First, what proportion of genetically corrected hematopoietic stem cells (HSCs) will be needed to achieve a therapeutic benefit? Second, what level of expression of a transferred globin gene will be required to improve beta-thalassemic erythropoiesis? These...
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