The desire to find a cure for genetic disorders that have such a damaging impact on a person’s life has led scientists down the road of gene therapy. Although gene therapy is still very much in its infancy in terms of medical research, it does provide a significant hope for the future in terms of finally finding a cure for such disorders.
Gene therapy is an experimental process whereby a healthy copy of a specific gene replaces or substitutes a malfunctioning gene in the body. Genes are intended to carry out a specific purpose which often involves carrying proteins and other bodily fluids to the organs around the body – if one gene is unable to function effectively then the body will suffer some often serious consequences. The theory is that if a healthy version of a malfunctioning gene can be accepted by the body then it follows that this healthy version will be able to take over the role that the flawed gene is not able to carry out successfully. If the healthy gene can carry out the role, it is then expected that the symptoms of a genetic disorder will be eliminated as there is no longer a deficiency in the work of that particular gene.
Gene therapy treatment can be carried out in two different ways:
- A carrier molecule – known as a vector – can be injected into the body loaded with the healthy copy of the gene and the vector transports this healthy material to the intended location. This vector is most commonly a virus. This is known as in vivo gene therapy.
- Alternatively, cells are removed from the body via the blood or bone marrow and are exposed to the virus carrying the healthy gene in the laboratory. The virus gets into the cells and unloads the healthy gene before the cells are injected back into the body. This is known as ex vivo gene therapy.
Once the healthy genes are successfully inside the cells it is then the intention that they are accepted by the body and can begin work on their specific purpose. Ultimately it is seen that this would cure a disorder and at the very least prevent the onset or progression of symptoms. Gene therapy differs to traditional drug-based treatments because it is intended to treat the underlying cause of the problem rather than its symptoms.
Although research surrounding gene therapy has been ongoing for a number of years, the medical understanding of the treatment is still very much in its infancy and as of yet it is not used widely as a treatment for genetic disorders. Clinical trials have been carried out in vast numbers and whilst these trials have proven hopeful for an eventual cure they have still not convinced the medical world that they are safe to use as a standard treatment for illnesses.
Although the theory sounds relatively straightforward, the huge amount of debate and scepticism surrounding gene therapy means that there is still a long way to go for scientists before they can realise and indeed prove its full potential to the world. However, with more research, more knowledge and a better understanding of the concept, gene therapy can be a shining light in the future of medical science.
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