Figure 1.

Generalized process for gene therapy. Nonviral vectors can be used to facilitate the delivery of DNA and RNA molecules by first mediating cellular uptake through endocytosis mechanisms. The vectors also provide protection to the genetic cargo and prompt compartmental escape as the endosome gradually acidifies. Once in the cytosol, siRNA-and miRNA-based cargo must be loaded into the RNA-induced silencing complex (RISC); whereas, mRNA must bind to cellular ribosomes to promote translation. Conversely, DNA requires translocation to the nucleus.