Viral and Non-viral Nanoparticles for Gene Therapeutics

Abstract

The recent accomplishment of the human genome and DNA discovery has led to the diagnosis of many diseases caused by imperfections in genes. These diseases involve gross disturbances in the number or arrangement of a person's chromosomes. Hence, gene therapy has become a promising new therapy for the treatment of somatic diseases, for example, malignant tumours [1], severe infectious diseases, such as AIDS [2], and many genetic disorders, including haemophilia and cystic fibrosis [3]. Gene therapy introduces a gene into human cells to replace, delete, or correct gene function to produce a therapeutic protein with the desired action. This adjustable gene can be used to cure any disease. In 1990, a gene therapy clinic was initiated to find treatment for severe combined immunodeficiency (SCID). However, the first success of gene therapy was not observed until 2000 when Cavazzana calvo et al. [4] reported a success using gene therapy for the treatment of SCID [4]. While it has been 30 years since the first gene therapy trial, gene therapy is still a high-risk treatment, and only a few drugs have been approved, such as Glybera® , Gendicine®, and Strimvelis®.