Gene Technology: Gene Therapy. Biological Basis Of Gene Therapy

Gene Technology: Gene Therapy
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Gene Technology: Gene Therapy
1 Biological Basis Of Gene Therapy
Gene therapy is one of the fast growing disciplines in medicine where genes are introduced to humans with the aim of treating genetic conditions (U.S. National Library of Medicine, 2018). Genes take part in heredity and are the basic rules that determine specific functions of a cell. Gene therapy aims to introduce genes to supplant or correct abnormal cell functions. Two forms of gene therapy used today include germ-line gene therapy and somatic gene therapy. In germ-line gene therapy, genes are inserted into the reproductive cells or embryos to correct any genetic aberrations that could potentially be passed to other future generations. Somatic gene therapy seeks to introduce genes at cellular or tissue level to treat genetic defects.
1 Description of gene therapy
Gene therapy introduces a genetic material into cells as a compensation for a defective gene or to allow the cells to synthesize proteins with beneficial functions. For instance, if gene mutation results in a missing or faulty protein, the technique can be used to introduce a functional copy of the gene with the aim of restoring the protein function (U.S. National Library of Medicine, 2018). Genes are introduced into cells through carriers known as vectors that are genetically engineered for gene delivery. Viruses are often used to introduce the new genes through infection. While viruses such as retroviruses work by integrating their genetic materials into the human chromosomes, adenoviruses directly introduce their own genetic materials into the cell nucleus without integrating into the chromosomes (Scientific American, 2018). The vectors can be given intravenously into specific body tissues or samples of patient’s cells can be taken and exposed to the vectors before returning them to the body of the patient.
2 What gene therapy accomplishes
While researchers have been working for years to bring the technology of gene therapy into practice, very few individuals with genetic disorders have been successful in accessing any effective treatment through gene therapy. Gene therapy seeks to correct any aberrations in gene function. A defective gene is replaced with a functional gene to allow the cell to make proteins for the desired function. A number of diseases have been found to be caused by a single-gene mutations. In this case, the defective gene is replaced with the gene that can synthesize the protein that does the function it is expected to perform. An example of diseases that has been treated this way is cystic fibrosis which is caused by a single-gene mutation.
3 Scientific principles that make the gene therapy possible
Scientists have long appreciated the concept that gene mutations can result in hereditary diseases such as sickle-cell anemia, cystic fibrosis and hemophilia. At the same time, errors in chromosomal count can lead to conditions such as Turner’s syndrome or Down syndrome. Advances in molecular biology led to the scientific understanding that alteration in genetic sequence can predispose humans to diseases such as cancer, atherosclerosis and schizop...