865. Focus on Specificity: Creating Efficient CML-Specific Recombinant Adeno-Associated Viral (AAV) Vectors Using a Random Peptide Library

Introduction: Most of the viral vectors applied in current gene therapeutic studies exhibit a broad tropism. In line with this, recombinant adeno-associated virus (rAAV) vectors have been used to transduce a variety of cell and tissue types. However, standard rAAV-2-based vectors lack the specificity and selectivity required for transduction of cells in vivo, as necessary for gene therapeutic approaches for diseases like chronic myelogenous leukemia (CML). The recently published method of a random AAV peptide library (Muller et al., Nat. Biotechnol. 21, 2003) now allows the generation of target cell-specific rAAV capsid-mutants. To obtain a highly specific and efficient rAAV vector for the transduction of CML cells, we performed a CML-specific in vitro selection on the CML cell line K562 using this random AAV peptide library.