Genetic engineering or modification refers to the process of using laboratory techniques to alter the DNA makeup of an organism. This means that we can change the genome of any living thing, whether it be food, plants, animals or even us humans. Although it is relatively recent technology, we can clearly see its impact on us right now through genetically modified foods for example. We can now create “better” foods, which increase agricultural efficiency and are healthier for us. Although this is not the focus of this article, the same ideology can be applied to other fields. Couldn’t it be possible to make “better” cells in our body to protect us from dangerous diseases? Gene therapy has provided an answer to this question.
Gene therapy is the modification of genes specifically to cure defects and malfunctions in a cell. It is thus solving the issue at its core. Instead of having to continuously take medicine throughout their life to reduce the symptoms of an illness, a patient could get rid of a disease forever!
In gene therapy, there are three main ways of proceeding: gene addition, gene silencing and gene correction. Gene addition refers to the insertion of a working version of a malfunctioning or missing gene into a cell. This is mainly used when a deficiency is spotted within a patient due to a faulty gene.
Gene silencing is the process of deactivation of a certain protein or messenger in order to modify the expression of genes within the cell. This is often used in cases where an mRNA (messenger) is blocking the expression of a functional gene, silencing thus results in a functional genetic expression.
Finally, gene correction or editing is a mechanism in which the malfunctioning sequence of a gene is “cut” out, the cell then repairs itself when the two resulting ends are joined back together. To do this, scientists use the CRISPR technique, which through the use of RNA derived from bacteria allows them to “cut” out specific parts of a cell’s genetic sequencing. This method can be used to completely remove a faulty gene.
To ensure that the genetic modification is applied to all cells in a patient’s body, scientists modify stem cells, which are the original, generic form of every cell in your body. These cells divide, spreading the modification made to all other cells, as our body is constantly renewing itself.
Gene therapy is mainly used to treat rare genetic diseases such as the sickle cell disease (a blood disease) and cystic fibrosis (a disease which blocks respiratory passageways and can cause infections). Nonetheless, it has also been used experimentally to treat more widespread diseases such as Alzheimer's disease and cancer. Indeed, a protein called beta-amyloid is considered a major cause of Alzheimer’s, which can be reduced considerably through genetic engineering and can reduce the risk of developing Alzheimer’s by a factor of four. To treat cancer, research is currently demonstrating that it is possible to boost cells in the immune system through genetic engineering, allowing them to destroy a cancerous tumour.
Therefore, gene therapy has become an integral part of biotechnology, many studies and advances have been made recently that are only at an experimental stage and havent been used on humans yet. We have yet to explore all the possibilities of genetic modification, but we know it has massive potential, especially in saving people. Its uses can be extrapolated in thousands of different ways and its future thus remains a mystery.
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