Gene Therapy

Researchers at University of Massachusetts, Gene Therapy Center Target Gene Therapy

Published in Gene Therapy Weekly, January 20th, 2011

2011 JAN 20 -- According to a study from the United States, "Adeno-associated virus 2 (AAV2) vectors transduce fibroblasts and mesenchymal stem cells (MSCs) inefficiently, which limits their potential widespread applicability in combinatorial gene and cell therapy. We have reported that AAV2 vectors fail to traffic efficiently to the nucleus in murine fibroblasts."

"We have also reported that site-directed mutagenesis of surface-exposed tyrosine residues on viral capsids leads to improved intracellular trafficking of the mutant vectors, and the transduction efficiency of the single tyrosine-mutant vectors is similar to 10-fold higher in human cells. In the...

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Source: Gene Therapy Weekly (2011-01-20)

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