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Genome Editing: Next generation medical treatment?

Updated on July 15, 2016

Molecular Biology of HIV

Medical treatment procedures have been completely revolutionized in the last few decades. We are now able to beat deadly diseases with novel antibiotics and other highly efficient targeted drugs. However same cannot be said about the cure for genetic disorders. Research pertaining to treatment of hereditary genetic disorders is still in its infancy. Many of these diseases are still classified as incurable. Scientists are actively engaged in finding possible therapeutic methods for their treatment.

Recently genome editing was proposed as an efficient alternative option to the conventional methods of treatment of genetic disorders. Genome editing is the process of manipulating the expression of genes in targeted cells with the help of specially designed enzymes like nucleases. The manipulation is designed in such a manner that it corrects the disease causing genetic mistakes. For example a single fault during the expression of beta globulin gene leads to Thalassemia, also called Mediterranean anemia (a deadly blood disorder). No definite and sustainable treatment strategy is present for this disease.

A group of Chinese researchers working on Thalassemia came out with a possible cure for the disease by implementing complex enzyme editing tools and genome editing procedures. Research showed that experimental restoration of the beta globulin gene to its original state by genome editing cured the disease. Subsequently it was found that genome editing can also contribute to the treatment of other incurable diseases like AIDS and cancer by correcting the malfunctioning genes responsible for the induction of the disease. It was suggested that application of such advanced molecular biology techniques may transform medical treatment methods in the future.

However, further research on genome editing as a clinical treatment method was stopped due to ethical concerns raised by many scientists. Half of the scientific community agrees with the concept of application of modern gene technologies for the cure of incurable diseases while the other half is completely against it, as they think it is a way of man playing the role of god and may lead to disastrous results.

In the current scenario a thorough and elaborate discussion on the possible social, ethical and legal complications is imperative, so as to access the practical viability of genome editing as a treatment to diseases in the future.


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