Targeted Gene Delivery: Importance of Administration Routes
نویسندگان
چکیده
Gene therapy is a promising approach to treat intractable and refractory diseases at the genetic level. Basically, in gene therapy, target gene expression is induced by delivering foreign genes. Downregulation of target gene expression or gene silencing can also be performed using miRNA, siRNA or shRNA expression vectors [1]. Gene therapy is useful for both genetic and acquired diseases. For genetic diseases, the first clinical trial was performed for adenosine deaminase deficiency in 1990 [2]. Subsequently, numerous clinical trials were carried out for other congenital genetic defects such as familial hypercholesterolemia and cystic fibrosis [3]. Gene therapy clinical trials were also performed for acquired diseases such as cancers, cardiovascular diseases and infectious diseases [3].
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