Isolation, Cloning and Characterization of New Adeno-Associated Virus (AAV) Serotypes
Posted Jul 31 2007 5:00pm
Description of Invention: 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.
Inventors: Michael Schmidt (NIDCR) John A Chiorini (NIDCR)
Licensing Status: A range of licensing opportunities exist, including material licenses, commercial licenses, nonexclusive and exclusive licenses, as well as fields of use directed towards clinical applications.
Collaborative Research Opportunity: The National Institute of Dental and Craniofacial Research, Gene Therapy and Therapeutics Branch, is seeking statements of capability or interest from parties interested in collaborative research to further develop, evaluate, or commercialize adeno-associated viruses. Please contact David W. Bradley, Ph.D. at email@example.com for more information.
Portfolios: Gene Based Therapies Gene Based Therapies - Therapeutics
For Additional Information Please Contact: Patrick McCue Ph.D. NIH Office of Technology Transfer 6011 Executive Blvd. Suite 325, Rockville, MD 20852 United States Email: McCuepat@mail.nih.gov Phone: 301-496-7057 Fax: 301-402-0220