ABSTRACT

Successful gene therapy depends on the capability of delivering the desired gene or cDNA to the tissue of interest, attaining the desired level of expression and, most importantly, the desired clinical response. In vivo gene therapy methods can be broadly divided into two categories, viral delivery and nonviral delivery. Both viral and nonviral delivery methods have uses in gene therapy, depending on the location, level, and expression time course necessary for the therapeutic transgene. However, each delivery type has associated problems. e primary issue with nonviral gene therapy is low transfection e¤- ciency, while for viral delivery, the potential for insertional mutagenesis or the induction of an immune response are of concern.