The Need

Recombinant adeno-associated viruses (rAAVs) have been effective tools for delivering gene therapy treatment and for manipulation of acquired diseases. Unfortunately, there is no effective method to target gene therapy for adipose tissue and transduction efficiency is low in naturally occuring AAV serotypes. In addition, current methods of gene therapy can result in off-target transgene expression. New high transduction, effective viral vectors that avoid detrimental effects and are capable of genomic delivery to include adipose tissue exclusively are needed.

The Technology

Researchers at The Ohio State University have developed a unique approach using novel rAAV expression plasmids to simultaneously deliver genes efficiently to adipose tissue while severely restricting off-target expression in liver. This technology provides a vehicle to genetically manipulate adipose for basic research as well as treat genetic and acquired diseases such as obesity, metabolic syndromes, and cancer. For instance, patients with a genetic defect in leptin are obese and require life-long treatment of leptin, a protein produced primarily in adipose tissue. This technology has been shown to rescue mice with leptin deficiencies such that blood glucose levels decreased to normal and mice weights were within healthy ranges.

Commercial Application

• Gene therapy
• Obesity management

Benefits/Advantages

• Targets a specific gene therapy for there is currently no delivery method
• Lower dose of vector required compared to commonly used systemic gene deliveries
• Expands the capabilities of gene therapy for adipose related health concerns