Selective in vivo targeting of human liver tumors by optimized AAV3 vectors in a murine xenograft model.

ABSTRACT: Current challenges for recombinant adeno-associated virus (rAAV) vector-based cancer treatment include the low efficiency and the lack of specificity in vivo. rAAV serotype 3 (rAAV3) vectors have previously been shown to be ineffective in normal mouse tissues following systemic administration. In the present study, we report that rAAV3 vectors can efficiently target and transduce various human liver cancer cells in vivo. Elimination of specific surface-exposed serine and threonine residues on rAAV3 capsids results in further augmentation in the transduction efficiency of these vectors, without any change in the viral tropism and cellular receptor interactions. In addition, we have identified a potential chemotherapy drug, shikonin, as a multifunctional compound to inhibit liver tumor growt

SUBMITTER: Ling C 

PROVIDER: S-EPMC4270149 | biostudies-literature | 2014 Dec

REPOSITORIES: biostudies-literature