Gene therapy is a promising approach for treating hemophilia. Non-viral vectors, although achieving only transient and low gene expression levels, can be an attractive alternative to viral vectors on the basis of ease of synthesis, low immunogenicity, and unrestricted plasmid size. Gene-based therapies for genetic disorders such as hemophilia where high expression and persistence are required are normally administered intravenously or intramuscularly, which is invasive and inconvenient for many patients. My research focuses on developing effective oral nonviral gene delivery systems, which have the potential to induce tolerance to the therapeutic protein and provide ease of administration for patients.
Lytic peptide-mediated sensitization of TRAIL-resistant prostate cancer cells to death receptor agonists. Barua S, Linton RS, Gamboa J, Banerjee I, Yarmush ML, Rege K. Cancer Lett. 2010 Jul 28;293(2):240-53