Abstract: The present invention provides for a process for producing a plurality of SUMOylation target-site modified AAV vectors, SUMOylation target-site modified AAV vectors, and application of the SUMOylation target-site modified AAV vectors in gene therapy. The present invention provides for manipulation of SUMOylation specific amino acids on AAV2 capsid protein, thereby regulating role of the same. Furthermore, the development of SUMOylation target modified AAV2 vectors presents an exciting opportunity for hepatic or ocular gene transfer with the safest AAV vector in human gene therapy applications. The plurality of SUMOylation target-site modified AAV vectors are not immunogenic in comparison to wildtype AAV2 vectors and possess significantly higher gene expression, with respect to wild type, thereby improving efficiency of hepatic and ocular gene transfer.

Abstract: The present invention provides a process for producing a plurality of Neddylation site modified AAV vectors. In one embodiment, the process comprises predicting a plurality of Neddylation sites on AAV VP1 capsid protein sequence based on relatively high confidence targets, wherein the AAV VP1 capsid protein sequence being set forth in Protein ID-YP_680426.1; and producing the plurality of Neddylation site modified AAV vectors based on predicted plurality of neddylation sites on AAV VP1 capsid protein sequence. The present invention for the first time highlights the role of Neddylation during AAV infection and its impact on generating novel AAV vectors with improved efficiency for ocular and hepatic gene therapy.