Gene Therapy Essay

Gene therapy is now considered a new therapeutic area of study in modern medicine. Genes are special segments of DNA that provide the information to the body to properly function. It involves the transfer of genetic information into the tissues and organs of patients. As a result, it can be used to eliminate diseased genes or restore their normal functions. Another application of gene therapy involves the inclusion of different function to the cells, in order to either fight cancer or other diseases. The main target for gene therapy in the early days includes inherited diseases that are caused by single gene defects, for example cystic fibrosis (1). Gene therapy can either be performed by the direct transfer of genes or by the usage of living

Apart from the self-renewal capability, the use of stem cells may reduce the need for repetitive administrations. There are several reasons for the use of stem cells in gene therapy, first being that they can readily be removed from the body. Even though the cells are present in small numbers, they can be removed from the bone marrow of adults, or via circulating blood or the umbilical cord blood of neonatal children. Furthermore, stem cells can be easily detected and controlled in the lab before being injected back into the patients. Hematopoietic stem cells have the ability to produce numerous types of blood cells including natural killer cells, T and B lympohyctes, macrophages, monocytes, megakaryocytes, granulocytes, eosinophils and basophils. Hence, once the altered stem cells differentiate, the blood cells will contain the therapeutic transgene produced. Thus, the applications these cells provide in gene therapy not only confine to treating disorders but also extend to organ transplantation. Hematopoietic stem cells are the only type of human stem cells being utilized in trials for gene therapy, however the other types that are being studied include myoblasts, muscle forming stem cells, neural stem cells and osteoblasts, bone forming stem cells