Adeno-Associated Virus (AAV) Vectors

Research in the Gao Lab primarily involves the discovery, development and use of adeno-associated virus vectors for gene therapy of genetic diseases and the study of miRNA functions in mammals. The lab works on isolation, characterization and vectorology of novel AAV vectors from primate tissues, molecular mechanisms of AAV evolution and diversity, and molecular interactions between endogenous AAV, AAV vector, host genomes and innate RNAi defense pathways. We also develop novel strategies for rAAV gene therapy of an inherited neurodegenerative disease, Canavan Disease, using novel AAVs that can cross the blood-brain-barrier for efficient CNS gene delivery and endogenous miRNA-mediated posttranscriptional de-targeting. Another area of research interest in the lab is to explore AAV vectors for delivery of denovo synthesized biological miRNA antagonists or over expression of microRNAs to elucidate micro RNA functions in adult mammals.