Gene therapy is the use of genes to treat or prevent diseases. This includes their use as replacement genes for defective ones in an individual, and their use as medicine, as giving an individual specific, new genes can help them combat a disease. Gene therapy is a promising tool, and scientists today are experimenting with it to use it against diseases which currently have no forms of treatment. Gene therapy transports genes to certain parts of the body by injecting carriers (called vectors) into either directly the patient, or into cells which are put into the patient. Vectors are typically viruses which have had a desired gene placed in them. These modified viruses introduce genes to parts of the body and spread them among the cells. Since the techniques currently used in gene therapy have only recently been developed, their safety is questioned, and research is being done to make gene therapy a more viable form of treatment (US National Library of Medicine, 2014).
Research into gene therapy treatment for choroideremia, an X-linked recessive eye disease, is beginning to show promising results (Beall, 2014). Choroideremia is a disease in which retinal cells die due to a defective CHM gene not producing a certain protein, leading to gradual eyesight deterioration and blindness. There is no treatment for choroideremia. This, along with the ease of using gene therapy on the retina due to its structure, was what prompted research into the use of gene therapy for the treatment of the disease (Wykoff, 2014). The research effort, lead by Dr. Robert MacLaren at the University of Oxford, has been in progress for 2 years. Recent trial tests using gene therapy to insert corrective genes have had all six patients with the disease reporting recovery in the form of better eyesight. The treatment focuses on correcting neuron retinal cells which remain active for an individual’s lifetime, expanding on an earlier gene therapy technique used to treat Leber’s congenital amaurosis, another eye disease (Beall, 2014). MacLaren expresses his desire for the treatment technique to ultimately prevent the blindness previously inevitable with the disease. However, other experts remark that, as the disease causes slow deterioration in eyesight, more long-term studies are required to prove the treatment’s effectiveness. MacLaren also stated that the treatment can only prevent the loss of retinal cells, but cannot recover previously damaged cells, something only research into stem cells can accomplish. However, research into the treatment is ongoing.
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I believe that this development is significant in expanding the scope of gene therapy. Research such as this will allow gene therapy to be used more commonly for the treatment of disease, and will promote further research into applications of gene therapy. I believe that such developments will change gene therapy from a rare clinical practice to a practical and efficient treatment option for many diseases.
A Question for You
Given the currently limited use of gene therapy for treatment, do you think that treatments such as this one will be accessible by everyone once they can be used in clinics? Will only those rich enough to afford them be able to use them, or will they be made more accessible to the common person?
Beall, A. (2014, January 16). Gene therapy restores sight in people with eye disease. Newscientist. Retrieved May 10, 2014, from http://www.newscientist.com/article/dn24879-gene-therapy-restores-sight-in- people-with-eye-disease.html
US National Library of Medicine. (2014, May 5). What is gene therapy?. Genetics Home Reference. Retrieved May 10, 2014, from http://ghr.nlm.nih.gov/handbook/therapy/gen
Wykoff, C. (2014, May 7). Gene therapy for retinal diseases is within sight. Medscape. Retrieved May 10, 2014, from http://www.medscape.com/viewarticle/824467