AAV vectors offer unique advantages over other vector systems in gene therapy applications. Studies have shown that these replication deficient parvovirus vectors can deliver DNA to specific tissues and confer long-term transgene expression in a variety of systems. Although many studies have looked at the tissue-specific expression elicited by each of the AAV serotypes, a true understanding of how AAV transduces these tissues is still unclear. Of the large AAV family, only a few receptors or co-receptors have been identified for any of the parvoviruses. The ability to better target transduction to specific tissues on the basis of the receptors that each serotype uses for entry, is essential to enable users to pick a serotype given the receptor expression in specific tissue, or to exploit altered receptor expression under disease conditions.
AAV6 has been reported to effectively transduce muscle, lung, brain, and multiple types of tumors, including gliomas and lung adenocarcinomas, and to elicit lower serum-neutralizing antibody concentrations when compared with AAV2. As such, there exists a need for improving the treatment of patients suffering from diseases such as cancer, which could be treated by AAV6 vector based gene therapy.
USPTO Backgrounds
USPTO Backgrounds