Gene therapy is the gene transfer into host cells for treatment of acquired and genetic disorders. For this purpose, there are a wide variety of gene delivery methods with special properties including viral and non-viral vectors. The non-viral methods use physical forces or chemical compounds (natural or synthetic) to transfer DNA into a cell. The efficiency of the non-viral gene therapy depends on conquering four different intra-and extra-cellular barriers such as cellular uptake, endosomal escape, nuclear entry, and gene expression. Among various gene carriers, some viral vectors such as Adenovirus, Lentivirus, Vaccinia as well as gene gun and lipofection achieved to clinical trials. In this mini-review, we briefly describe different approaches for gene delivery and their applications in various phases of clinical trials.

Background: Chronic myeloid leukemia (CML), a hematological cancer of stem cells, is caused by the activation of oncogenic factors alone or/with inactivation of tumor suppressor genes. Curcumin is a hydrophobic polyphenol and the main compound of turmeric, which has been used in daily diets for many years. It is also a safe drug. Nanogels and nanobiotechnology have important roles in the diagnosis and treatment of diseases and drug delivery. Materials and Methods: To prepare the nanodrug, chitosan nanogels were prepared in 1% acetic acid and cross-linked with stearate by 1- ethyl- 3 (3-dimethylaminopropyl) carbodiimide (EDC) and N-hydroxysuccinimide (NHS). Subsequently, curcumin was loaded in the chitosan-stearate nanogel. Physical and morphological characteristics of the nanodrug were determined by transmission electron microscopy (TEM), dynamic light scattering (DLS), and Fourier transform infrared spectroscopy. Different nanodrug concentrations were prepared and evaluated on the K562 CML cell line. The apoptotic activities of curcumin and nanodrug on the cells were detected by flow cytometry, MTT assay, and trypan blue staining. Results: DLS revealed that the size of the nanodrug was 150 nm, which was confirmed by TEM. The half maximal inhibitory concentration (IC50) values of curcumin and nanodrug were 50 and 25 μg/ml, respectively P < 0.05). Apoptosis of the K562 cell line occurred at 48 h post-treatment with 25 μg/ml curcumin and 12.5 μg/ml nanodrug. Conclusion: The increase in the cytotoxicity of curcumin and nanodrug was directly related to the drug concentration and time. The nanodrug exhibited more cytotoxic effects on the vital capacity of the cells and stimulated more apoptosis compared with curcumin alone. [GMJ.2018;7:e921]