Recombinant AAV Vector with MITF-M Promoter for Melanoma Gene Therapy

We have developed the recombinant adeno-associated virus (AAV) carrying the EGFP gene under the control of the microphtalmia-associated transcription factor-M (MITF-M) promoter region for melanoma-specific expression. MITF-M distal enhancer (MDE) region enhances the specific expression of the reporter gene specifically in cultured melanoma cells. Expression of EGFP protein was very high in AAV-CMV-EGFP infected cells but relatively low in cells infected with AAV-Mitf(Enh/Pro)-EGFP. After an in vitro infection by a recombinant AAV carrying the EGFP gene under the control of human MITF-M promoter, the reporter gene was expressed in MITF-M producing melanoma cell lines (SK-28 and G361), but not in MITF-M non-producing cell lines (HaCat). These results suggest that the utilization of the MITF-M promoter in a recombinant AAV vector could provide benefits in gene therapy applications.