GFP Adeno-Associated Virus ( AAV-NSVSLYT )

Chronic myeloid leukemia (CML) accounts for approximately 15–20% of all adult leukemias in the Western population. Currently, the most effective and best-tolerated drug against CML is imatinib mesylate, a selective BCR-ABL tyrosine kinase inhibitor. To eliminate the remaining drug-resistant CML cells in patients, novel therapeutic options such as gene therapy must be considered. One potential approach is to transfer vectors containing suicide genes or immunostimulatory genes to these cells. For gene therapy of CML, most standard vector systems lack the required gene transfer efficiency and/or in vivo selectivity. Advances in the development of parvovirus-based adeno-associated virus (AAV) vectors by Müller et al. now allow the generation of rAAV capsid mutants that have higher gene transfer efficiency and potentially higher target cell specificity. The backbone of AAV-NSVSLYT is derived from AAV serotype 2 (AAV2), a well-established serotype known for its efficiency in transducing a variety of cell types. The innovative engineering of this virus involves the insertion of the peptide sequence NSVSLYT at position I588 of the capsid. The capsid modifications in AAV-NSVSLYT were tailored to improve the virus's tropism for tumor cells, specifically CML cells. This targeting is expected to facilitate more efficient delivery of genetic material directly to cancer cells, potentially enabling more precise and effective therapeutic interventions. The peptide inserted into the capsid enhances the virus's ability to track and enter these malignant cells, making it a powerful tool for cancer research and treatment.