Vector Development
Research from University of Oxford provides new data on vector development
Published in Gene Therapy Weekly, March 4th, 2010
Current study results from the report, 'Long-term physiologically regulated expression of the low-density lipoprotein receptor in vivo using genomic DNA mini-gene constructs,' have been published. "Familial hypercholesterolemia (FH) is a condition caused by mutations in the low-density lipoprotein receptor (LDLR) gene. Expression of LDLR is highly regulated and excess receptor expression is cytotoxic," scientists writing in the journal Molecular Therapy report.
"To incorporate essential gene regulation into a gene therapy vector for FH, we generated vectors in which the expression of therapeutic human LDLR gene, or luciferase reporter gene, is driven by 10 kb of...
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Source: Gene Therapy Weekly (2010-03-04)
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