The molecular genetics of the AAV life cycle
The molecular genetics of the AAV life cycle and the use of the virus as a potential vector in human gene therapy are the areas of emphasis in the Berns laboratory. Current projects include AAV as a vector for treatment of retinal neovascularization and as a vector to inhibit vascular intimal proliferation in renal transplants. AAV is of interest as a gene therapy vector because of its ability to persist for long periods of time in the host without causing any apparent disease. Wild type virus persists by integration at a specific site on human chromosome 19. The viral Rep protein is required for site specific integration, but current AAV vectors do not have the gene required for Rep expression. Ongoing work in the laboratory is designed to test whether Rep+ AAV vectors will function as well as those without the gene in terms of the level and duration of gene expression; also the question of pathogenicity of such vectors will be addressed.
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