Efficiency of Different Serotypes of AAV Vectors in Human Stem Cells for Ex Vivo Gene Therapy

INTRODUCTION: Viral vectors are commonly used in ex vivo approaches for stem cell therapy modalities; however, the potential for insertional mutagenesis by random DNA integration limits the application of retro-and lenti-viral vectors for stem cell therapy in vivo. Recombinant adeno-associated viral (rAAV) vectors exist as extrachromosomal episomes rather than as DNA integrated genomes, which ensures that AAV transduction remains innocuous [1]. Each serotype of AAV has a unique cellular transduction characteristic due to its unique tropism, which results in low-efficiency transduction of AAV vectors in rat bone marrow cells and mouse muscle-derived stem cells (MDSCs) as observed in our previous studies. The purpose of this study is to test whether high-efficiency transduction human stem cells, isolated from bone marrow and muscle, can be achieved by the use of an appropriate AAV vector serotype carrying the GFP reporter gene.