Chiorini, John (Jay) (NIDCR)
Adeno-associated viruses (AAV) are used in gene delivery, but with limited success due to toxicity. The novel AAVs described in this technology may be more effective and useful in gene therapy applications.
This invention relates to new adeno-associated viruses (AAV), vectors and particles derived therefrom and also provides methods for delivering specific nucleic acids to cells using the AAV vectors and particles. The inventors cloned and sequenced the genomes of AAVs found in twelve (12) simian adenovirus isolates and determined that the AAVs were novel. Ten (10) of these isolates had high similarity to AAV1 and AAV6 (>98%). Despite the high homology to AAV6, these novel AAVs demonstrated distinct cell tropisms and reactivity towards a panel of lectins, suggesting that they may use a distinct entry pathway.
- AAVs can be used as delivery systems in gene therapy
- AAV’s also have gene transfer applications
- Vectors based on these new AAV serotypes may have a different host range and different immunological properties, thus allowing for more efficient transduction in certain cell types than previously used AAV.
- Gene therapy has tremendous potential in treating several life threatening diseases, and this technology has the potential to benefit millions of patients that could benefit from the proper use of gene therapy treatments. Additionally, the gene therapy market is now a multi-million dollar industry can substantially benefit from the use of this technology.