International Journal of Pharmaceutical Investigation, 2012, 2, 3, 97-98.
Published: December 2012
Department of Pharmaceutical Sciences, Manchester University College of Pharmacy, 10627 Diebold Road, Fort Wayne, IN 46845, USA
Gene therapy has been investigated a lot in both basic research and clinical trials. The first antisense oligodeoxyribonucleotide (ODN) drug, Vitravene (Fomivirsen), was approved by the United States Food and Drug Administration (FDA) in 2005. After this approval, more and more clinical trials are conducted, not only for ODNs, but also for other nucleic acids drugs, such as plasmid vectors and small interference RNAs (siRNAs). However, delivery efficiency is a big barrier for the clinical application of gene drugs. It is necessary to overcome their large molecular weight, large size, and negative charge. Nucleasemediated degradation is also an issue, decreasing the performance of gene drugs. Currently, there are two major categories of methods for gene delivery, viral vectors and nonviral carriers. Viral vectors have higher delivery efficiency than nonviral carriers; whereas nonviral carriers are less toxic and immunogenic. Another important feature for the nonviral delivery system is that they offer delivery on genes with various sizes, which facilitates the potential application of oligonucleotides, such as antisense ODNs and siRNAs. Read More…