Gene therapy, physiological applications, problems and prospects - a review

Abstract

Gene therapy can be defined as the use of DNA as a pharmaceutical agent to treat disease. It is also an experimental medical treatment that manipulates a gene or genes within cells in order to produce proteins that change the function of those cells. The physiological applications, problems and prospects of gene therapy are reviewed in this study. The different types of gene therapy such as germline gene therapy, somatic gene therapy and chimeraplasty gene therapy are discussed. Polymerase chain reaction (PCR), nanoparticles, sonoporation, electroporation and gene gun are the techniques used in gene therapy. Polymerase chain reaction (PCR) is used in medical and biological research. Nanoparticles have been widely used in the field of drug and gene delivery to target cells. Sonoporation allows uptake of large molecules of DNA into the cell, in a process called cell transformation. Electroporation is highly efficient for the introduction of foreign genes in tissue culture cells, in tumor treatment and cell-based therapy. A gene gun is a device for injecting cells with genetic information to plant cells. Gene therapy is applied in medicine, agriculture, loss and gain of function, tracking and expression studies. Some problems bedeviling gene therapy include insertional mutagenesis, mutagenic disorders, problem of viral vectors, immune response etc. Gene therapy has the potential to eliminate and prevent hereditary diseases such as cystic fibrosis and is a possible cure for Alziehmer’s disease and cancer, enhance agricultural productivity of farm animals, and in the production of genetically modified animals (GMOs) which will further help in medical and biomedical research.

Keywords: Gene therapy, Chimeraplasty, Nanoparticles, Sonoporation, Electroporation, Gene gun, Polymerase chain reaction