Katherine A Fitzgerald
Title: Transgenic Expression of DnaseI in vivo Delivered by an Adeno-associated Virus Vector. UMMS15-38; Patent Pending.
This invention uses AAV therapy to deliver an important enzyme, DNaseI, for cyctic fibrosis (CF) treatment. Traditional clinical methods of DnaseI delivery use solely nebulizers, which limit the delivery of the enzyme to the lung. With this new quality-controlled AAV vector for delivery, DnaseI can now be delivered locally to other organ systems affected by CF for more comprehensive disease management.
Title: INTRACELLULAR DNA RECEPTOR. UMMS09-11; Patent 8,334,101
The innate immune system recognizes non-self genetic material and mounts a defensive immune response. However, self cytosolic DNA at times gets targeted and the process by which this phenomenon occurs has been largely unknown. The inventors have discovered PISA (PYHIN protein stimulating ASC), a receptor found to be necessary for activation of the ACS/caspase-1 axis of innate immunity in response to recognition of self cytosolic DNA. This invention discloses methods for identification and compounds found to modulate the PISA receptor and it’s downstream immune response.