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Novel HSC transgene approach needs validation before trials; leaves open durable lineage-specific expression in patients.
Lineage-specific and high-level expression of transgenes introduced into hematopoietic stem cells have been the holy grail of gene therapy for over a decade. Initial excitement and competitiveness to be first quickly faded into frustration and disappointment as 2 major hurdles were identified using
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Cynthia E. Dunbar (2002) studied this question.
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