Gene delivery and clinical applications

Both genetic and acquired human diseases are directly or indirectly associated with overexpression or suppression of endogenous cellular genes and consequently, treating diseases at the genetic level would be much precise and effective while minimizing the off-target effects frequently seen with conventional therapy that targets multiple proteins in reality. Hence, gene therapy employing either a viral or a nonviral carrier to transport gene(s) of interest and facilitating production of the desirable therapeutic protein(s) holds tremendous prospects particularly for the treatment of different hereditary diseases, cardiovascular diseases, neurodegenerative diseases, acquired immunodeficiency syndrome, viral hepatitis, and various cancers. Although viral vectors have shown highly promising therapeutic outcomes in preclinical and clinical trials by virtue of their favorable pharmacokinetics with enhanced capacity in endosomal escape and nuclear translocation, they could potentially be very immunogenic and carcinogenic. On the contrary, although nonviral synthetic particles are in general inefficient in transgene delivery and expression, they are increasingly considered as potential tools for safer delivery of genetic materials.