Thursday, September 12, 2013
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adeno-associated viral (AAV) strains for improved specificity and
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.
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.
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