Identification of Patient Specific Neutralizing Epitopes on the AAV8 Virus for Personalized Gene Therapy.

Abstract

Gene therapy using viral vectors is quickly becoming a mainstream approach of many biotechnology companies to treat genetic and rare diseases. The AAV8 vector is becoming a more common choice because it has high liver tropism, is nonpathogenic and is very closely related to the well characterized AAV2 vector. Although AAV2 has been so well characterized, there is still much research to be done for AAV8. This thesis project utilized multiple types of in-vitro assays to identify a region of epitopes on the AAV8 vector that are likely contributing to the neutralizing antibodies found in human serum. This research also suggests that different types of epitopes (linear vs conformational) influence the severity of neutralization.