Chiorini, John (Jay) (NIDCR)
Afione-Wainer, Sandra (NHLBI)
Agbandje-Mckenna, Mavis (University of Florida)
Halder, Sujata (University of Florida)
- In vivo data available (animal)
Scientists at the NIH disclosed a mutated adeno-associated virus (AAV) serotype 5 by modifying sialic acid binding regions which mediate viral entry into host cells. Preliminary results from animal studies suggest that this modification can increase transduction by 3-4 folds in salivary glands and muscles, and can significantly decrease the potential of being neutralized by preexisting antibodies compared to the wild type AAV. Thus, the modified AAV5 vectors seem to be optimal for gene therapy. This invention overcomes two major issues in AAV-based gene therapy: the ability to efficiently transduce the target cells and the ability to evade the immune response to vectors.
- Genetically engineered AAV5 vectors for gene therapy.
- Enhanced transduction activity.
- Reduced the potential for being neutralized by preexisting antibodies.