GENE THERAPY

Literature Review: Genes and Health-Implications for gene therapy in the future



Literature Review: Genes and Health-Implications for gene therapy in the future

Introduction

In search of causes of various diseases, medicine has now made significant advancement. We are now able to reach at the molecular level. Genetics and genomics has opened its ways to far deeper and newer understanding of bodily processes, as well as, providing tools for extremely precise and targeted interventions, when bodily functions are disturbed. For the first time, we have a diagnosis and therapy meeting at a common ground (Playfair, 2007).

Genomics and gene therapy have opened new doors for prompt diagnosis of genetic metabolic diseases, which has received much attention in the past two decades. The cause of genetic metabolic diseases is blamed on a defective gene, resulting in an enzyme to be absent or being ineffective in the catalyzing process of a particular metabolic reaction. Gene therapy is a future hope in curing the diseases. This technique involves replacing of the defective or absent gene by a working gene so that the body can proceed normally; thus, eliminating the root cause of the disease (Gene Therapy -An Overview, 1990).

Gene therapy was initially aimed for treating inherited genetic disorders, such as haemophilia, cystic fibrosis, and so forth; however, in the past few years, it has expanded its reach to combat acquired diseases, like cancer, AIDS, Parkinson's, Alzheimer's, and several infectious diseases. Minimum requirement for successful gene therapy is continual production of therapeutic gene products without any harmful effects (Bulyenkov et al., 2000, pp. 136-146).

Gene therapy defined so far all have a common factor; that is the treatment is limited for somatic tissues only. On the contrary, an option of replacing the defective genes in the germ-line cells is also available. Gene therapy for germ-line cells holds the potential of treating the individual, as well as, his or her children. Hence, it can be said germ-line can eventually alter the genetic pool of the whole human species, affecting the future generations. These ethical problems have lead to cessation of the therapy for trials on humans, in the near future. In order to commence a gene therapy, accurate diagnosis of the genetic defect is essential. This leads to a greater influence on the therapy from biotechnology in the future. Moreover, a development in distinguishing the defective gene and its normal counterpart also holds immense importance in the near future (Gene Therapy -An Overview, 1990).

Discussion

A Brief History of Gene therapy

During the year of 1989, Rosenberg and his team completed a clinical trial of gene therapy for the first time in humans. They employed retrovirus which established specific gene coding providing resistance to neomycin directly in the tumour-infiltrating lymphocytes of humans, prior to its infusion in 5 patients suffering from advanced stage melanoma. The study recognized the possibility of transducing retroviral gene in humans, as well as, became the primary stepping stone for further studies. Ever since, there have been more than 900 clinical trials which have ...