Article
Construction of minicircle DNA vectors capable of correcting familial hypercholesterolemia phenotype in a LDLR-deficient mouse model.
Gene therapy - 1 Aug 2016
Hou X, Jiao R, Guo X, Wang T, Chen P, Wang D, Chen Y, He C-Y, Chen Z-Y
Abstract excerpt
Familial hypercholesterolemia (FH) caused by defect in low-density lipoprotein receptor (LDLR) is a life-threatening disease with poor response to conventional treatments. Earlier gene therapy studies have generated promising results, but further development is hampered because the cells harboring the viral vectors were eliminated by host immune system soon after delivery, whereas the nonviral vectors were too...
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