Gene Therapy Technology

The term transfection is commonly used to describe the process of adding DNA into cells with a view to mediating protein expression of the transgene of interest. Although DNA transfection is the most commonly adopted technique, the term can also be applied to RNA transfection, where the purpose may also be to mediate protein expression or to achieve knockdown of the transcription of a specific gene, either by RNAi, ribozyme or antisense methodologies.

Human Immunodeficiency Virus (HIV)-derived lentiviral vectors provide efficient gene transfer in proliferative and quiescent cells and demonstrate stable, high-level transgene expression both in vitro and in vivo. HIV non specifically integrates its DNA into the human genome, with a preference for active genes. However, integration can be problematic because a variation in gene expression between cells, possible gene silencing, and most importantly insertional mutagenesis, that can provoke malignant transformation.

Gene therapy is a strategy in which nucleic acid is administered for therapeutic purposes for both inherited and acquired diseases. A number of viral and nonviral vectors have been developed to circumvent the barriers for gene delivery, but the safety concerns of viral vectors have not been solved yet. On the other hand, non viral vectors are still inefficient compared to viral vectors but they offer safety as the main advantage and moreover they can be easily formulated as medicines.