GFP Adeno-Associated Virus ( AAV-LS4 )

Chronic myeloid leukemia (CML), also known as chronic myeloid leukemia, is a cancer that originates in the bone marrow – the soft, spongy tissue within the bones that produces blood cells. CML is characterized by an overproduction of white blood cells, particularly granulocytes, which interferes with the production of normal blood cells. This interference can lead to a variety of health complications, including anemia, infections, and bleeding disorders. For clinical gene therapy approaches, effective target cell specificity and safe vector systems are absolutely essential. AAV-based vectors have a favorable safety profile, as they have low immunogenicity, lack any associated pathogenicity, and are located extrachromosomally. As a result, AAV vectors, especially those based on serotype 2 (AAV2), have been extensively studied and used in clinical and preclinical studies. However, for efficient gene transfer into primary human PBPCs, vectors based on standard recombinant AAV2 (rAAV2) lack the required efficiency. AAV-LS4 is engineered from AAV serotype 2 (AAV2) with a modified capsid that includes the insertion of the peptide sequence NSVQSSW at position I588. By inserting the NSVQSSW peptide at position I588 of the AAV2 capsid, the researchers used molecular design to redirect viral targeting to specific cell populations. In this case, the target cells included tumor cells, particularly those associated with chronic myeloid leukemia (CML), and CD34+ cells, a marker for hematopoietic stem cells. This precise engineering gave the virus enhanced specificity and efficacy in targeting specific cell types, greatly expanding its potential research and therapeutic applications.