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Mark L. McCleland

Children's Hospital of Philadelphia

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skeletal actin/CMV hybrid enhancer/promoter Improved muscle-derived expression of human coagulation factor IX from a

2013
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Sustained phenotypic correction of hemophilia B dogs with a factor IX null mutation by liver-directed gene therapy

2002 Blood
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Improved muscle-derived expression of human coagulation factor IX from a skeletal actin/CMV hybrid enhancer/promoter

2000 Blood
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Role of vector in activation of T cell subsets in immune responses against the secreted transgene product factor IX.

2000 Molecular Therapy
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