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Gene Therapy Technologies

Gene therapy holds the tantalizing promise of permanently correcting inherited genetic defects that are often untreatable with conventional therapeutics. University of Florida researchers sought to improve on current gene therapy protocols and avoid limitations of conventional techniques through the use of a tiny, harmless virus called adeno-associated virus (AAV) as a vector for gene delivery. AAV is ideal for gene therapy applications for a number or reasons, most importantly, the viral vector itself is harmless and does not elicit immune response in the patient. Further, the vector integrates into a known location in the genome, avoiding side effects caused by random integration.

The University of Florida is actively seeking companies interested in commercializing one or more of the following gene therapy technologies, many of which utilize AAV as a viral vector in gene therapy protocols. For more information on these technologies, please contact Elizabeth Garami, UF Office of Technology Licensing, at (352) 392-8929 or egarami@ufl.edu.

Gene Therapy Technologies
PDF ID Lead Inventor Title
11434 Bloom, David Insulation Cassette that Enables Controlled, Sustained Gene Therapy Delivery
10606 Byrne, Barry Gene Therapy for Hemophilia A
10859 Byrne, Barry Gene Therapy for Pompe Disease
10643 Conlon, Thomas Gene Therapy for Fatty Acid Metabolism Defects
10851 Flotte, Terence Method of Targeting Dendritic Cells
11249 Flotte, Terrence Gene Therapy for Cystic Fibrosis
10834 Laipis, Philip Gene Therapy for Phenylketonuria (PKU)
10011 Lewin, Alfred Novel Technique to Enhance Ribozyme Specificity and Delivery
11431 Mah, Cathryn Method for Increasing Efficiency of Transduction in Targeted Areas
  10824 Muzyczka, Nicholas Gene Therapy for Applications in the Nervous System
11184 Nash, Kevin Gene Therapy for Alzheimer's and Other Diseases
11178 Scarpace, Philip Gene Therapy for Weight Loss
10612 Song, Sihong Gene Therapy for Preventing Diabetes
  10870 Snyder, Richard Production and Purification Methods for AAV Serotypes and Capsid Mutants
10848 Zolotukhin, Sergei Method for Molecular Breeding of Gene Therapy Vectors
11372 Zolotukhin, Sergei Method for Scaled-Up Production of Recombinant Adeno-Associated Viral (rAAV) Vectors