This week in techniques



Licensing status

Publication and contact information

Assays & screens

Humanized mice to select for improved recombinant adeno-associated vectors (AAVs) for gene therapy

Humanized mice could help screen for and select clinically relevant AAVs for gene therapy. The transduction efficiency of recombinant AAVs in animal models often does not translate to humans. Chimeric mice with humanized livers were used to select for AAVs capable of efficient cellular uptake, internalization and replication. In the mice, delivery of a fluorescent reporter gene using one of the most effective AAVs transfected about 43% of human hepatocytes, whereas AAV8, a clinically tested, liver-specific vector, transfected about 4% of cells. Next steps could include using additional humanized mouse models to identify AAVs selective for other human tissues.

SciBX 7(4); doi:10.1038/scibx.2014.125
Published online Jan. 30, 2014

Patent application filed; available for licensing

Lisowski, L. et al. Nature; published online Dec. 25, 2013;
Contact: Mark A. Kay, Stanford University School of Medicine, Stanford, Calif.