Journal Club of the Week: Gene Therapy and Gene Delivery

In the 19th December 2016 journal club meeting, the topic of gene therapy and delivery system was discussed. The success of gene therapy is largely dependent on the development of a vector or vehicle that can selectively and efficiently deliver a gene to target cells with minimal toxicity. Viruses are efficient in transducing cells. However, the safety concerns regarding the use of virus in humans make nonviral delivery systems an attractive alternative. Nonviral vectors are particularly suitable with respect to simplicity of use, ease of large-scale production and lack of specific immune response. However, the viral is difficult to produce, safety issue and high cost ration. Recently, several novel nonviral vectors have been developed that approach viruses with respect to transfection efficiency. A variety of nonviral delivery systems that can be used for gene therapy in different clinical settings are also available. Different nucleic acid polymer such as naked DNA, cationic lipids, RNA/DNA chimera for targeting gene repair, polyplex, CpG immunostimulatory motifs were discussed. Different delivery approaches such as physical, biological and chemical were described. Different approved gene therapeutic drugs and cells types such as somatic and germline cells are categorized in gene therapy.