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Dual, glycan-binding adeno-associated viral (AAV) strains for improved specificity and transduction efficiency

Dual, glycan-binding AAV strains could be useful for improving the specificity and efficiency of gene delivery. A chimeric AAV strain was created by grafting a galactose-binding motif from AAV serotype 9 (AAV9) onto the heparan sulfate-binding AAV2. In mouse brain, lung and spleen tissues, the chimeric strain showed transduction efficiency comparable to that of AAV2, whereas in heart, liver, skeletal muscle and kidney tissues, the chimeric strain showed higher transduction efficiency. In mice, a second chimeric strain generated by grafting the same galactose motif on a different AAV2 vector that avoids targeting the liver showed 200-fold lower transduction efficiency in liver tissue than AAV9 but similar selectivity for other tissues. Next steps could include evaluating the delivery of therapeutic genes with the chimeric AAV strains in disease models.

SciBX 6(35); doi:10.1038/scibx.2013.977
Published online Sept. 12, 2013

Patent application filed covering method for re-engineering AAVs, the clones generated and their therapeutic use; licensing status unavailable

Shen, S. et al. J. Biol. Chem.; published online Aug. 12, 2013;
Contact: Aravind Asokan, The University of North Carolina at Chapel Hill, Chapel Hill, N.C.