What question did this study set out to answer?

To evaluate a novel retroviral vector for macrophage-specific gene expression and its therapeutic effects in ApoE-deficient mice.

December 31, 2002BloodOpen Access

Gene therapy of apolipoprotein E–deficient mice using a novel macrophage-specific retroviral vector

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Randomized trial demonstrates gene expression in macrophages improves cholesterol levels in ApoE-deficient mice, suggesting therapeutic potential.

To evaluate a novel retroviral vector for macrophage-specific gene expression and its therapeutic effects in ApoE-deficient mice.
Developed self-inactivating retroviral vectors with CD68 gene elements.
Transplanted transduced hematopoietic stem cells into ApoE-deficient mice.
Measured expression levels of ApoE and effects on hypercholesterolemia and atherosclerosis.
Macrophages expressed ApoE levels comparable to wild-type mice.
Vector-driven ApoE expression reversed hypercholesterolemia.
Atherosclerotic lesion development was significantly reduced.

Gough et al. (2002) studied this question.

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Randomized trial demonstrates gene expression in macrophages improves cholesterol levels in ApoE-deficient mice, suggesting therapeutic potential.