AAV Vector-Based Anti-Inflammatory Therapeutic for Macular Degeneration and Other Tissue-Specific Inflammatory Diseases

Gene Therapy Vectors Deliver Secretable, Cell-Penetrating Viral Therapeutics to Targeted Host Cells

This AAV vector-based therapeutic treats inflammatory responses in mammals by delivering tissue-specific anti-inflammatory therapeutic proteins to targeted host cells. These transvected host cells then inhibit a variety of pro-inflammatory syndromes, which cause or exacerbate diseases and disorders caused by chronic inflammation. AAV vector-based gene therapy is useful in treating a number of diseases, including congestive heart failure, Parkinson’s disease, and hemophilia, by delivering therapeutics to targeted cells. Unfortunately, no available treatments combat tissue-specific inflammation, such as dry age-related macular degeneration. Age-related macular degeneration affects as many as 11 million people with global cost of visual impairment at $343 billion in the United States alone. University of Florida researchers have developed AAV vector constructs that are optimized for delivering anti-inflammatory peptides to selected mammalian cells and tissues. This tissue-specific treatment addresses symptoms of oxidative stress and inflammation by delivering secretable, cell-penetrating anti-inflammatory proteins to treat specific inflammatory diseases, such as dry age-related macular degeneration.

Application

AAV vectors deliver modified viral protein as tissue-specific therapy for chronic inflammation

Advantages

• Delivers viral proteins to host cells, providing gene therapy treatment for inflammation
• Delivers gene product directly to affected cells, increasing chance it can be used for many other tissue-specific inflammatory diseases
• Targets two key pro-inflammatory signaling pathways within cells, giving it the capacity to treat diseases over prolonged period of time

Technology

AAV vector-based therapy has been utilized in treating a number of genetic diseases; however, gene therapy has not traditionally been used to treat inflammatory diseases. This tissue-specific method of treating inflammation targets inflammatory responses by administering AAV vectors coupled with secretable, cell-penetrating anti-inflammatory proteins derived from viruses. These modified viral proteins penetrate target host cells; these suitably-transvected host cells then inhibit the key cellular inflammatory pathways that cause or exacerbate a variety of chronic progressive diseases, disorders, and conditions. Researchers have used this AAV vector-based therapy to treat dry age-related macular degeneration as an example of the therapy’s use in inhibiting pro-inflammatory responses in a number of mammalian diseases.