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

With an increased prevalence and understanding of central nervous system (CNS) injuries and neurological disorders, nucleic acid therapies are gaining promise as a way to regenerate lost neurons or halt disease progression. While more viral vectors have been used clinically as tools for gene delivery, non-viral vectors are gaining interest due to lower safety concerns and the ability to deliver...

Several improvements have been made in liposomal delivery, thus making this technology potentially useful for treatment of certain diseases in the clinic. Success in non-viral delivery is complicated and requires optimization of several components. These components include nucleic acid purification, plasmid design, formulation of the delivery vehicle, administration route and schedule, dosing, ...