Abstract
Gene therapy is the most promising treatment for recessive dystrophic epidermolysis bullosa (RDEB), however genetic cargo delivery efficiency is still a technical limitation. Viruses are the traditional vector of preference for gene therapy, as virus trophism increase tissue specificity. However, drawbacks related with safety and high manufacturing costs have facilitated the expansion of non-viral vectors, such as liposomes and cationic polymers. Our group is focused on the development of highly branched cationic polymers for gene therapy to treat RDEB.
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