Pros And Cons Of Gene Therapy
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In the last century, there have been a number of discoveries regarding the treatment of human disease and genetic conditions. The current on-going research is in the field of gene therapy, an experimental technique that uses genes to treat and replace the defective genes of an affected person. Instead of treating disease symptoms, this has the potential to correct the underlying cause (1). Besides its high costs and ethical concerns (therapy involving germ line treatment), this technique also poses a considerable amount of risk. Thus, gene therapy is currently only being tested on the diseases for which there is no cure. This article shall look primarily into cystic fibrosis, as well as X-linked severe combined immunodeficiency (SCID), as examples to describe the potential of gene therapy in medicine.
Cystic fibrosis is a single gene disorder, with an autosomal recessive pattern of inheritance. The gene is located on the long ‘q’ arm of chromosome 7 and is called the cystic fibrosis transmembrane conductance regulator (CFTR) gene (2). The gene product is an apical membrane …show more content…
On one hand, it seeks to root out the cause of diseases which have no cure, rather than only treating its symptoms. However, treatments vary from disease to disease. In the case of cystic fibrosis, the effects of treatment do not last very long, and in SCID-X1, the treatment has led to risks of leukaemia. There is an ethical concern that it could modify human capabilities, thus altering the standards of normal human life. Gene therapy is also a very expensive form of treatment and hence should be regulated effectively. Gene therapy has a remarkable therapeutic potential (14) and this should be exploited. Through effective research and regulation, gene therapy has the potential to cure genetic diseases, eliminate any possible side effects and usher in a new standard of
In the last century, there have been a number of discoveries regarding the treatment of human disease and genetic conditions. The current on-going research is in the field of gene therapy, an experimental technique that uses genes to treat and replace the defective genes of an affected person. Instead of treating disease symptoms, this has the potential to correct the underlying cause (1). Besides its high costs and ethical concerns (therapy involving germ line treatment), this technique also poses a considerable amount of risk. Thus, gene therapy is currently only being tested on the diseases for which there is no cure. This article shall look primarily into cystic fibrosis, as well as X-linked severe combined immunodeficiency (SCID), as examples to describe the potential of gene therapy in medicine.
Cystic fibrosis is a single gene disorder, with an autosomal recessive pattern of inheritance. The gene is located on the long ‘q’ arm of chromosome 7 and is called the cystic fibrosis transmembrane conductance regulator (CFTR) gene (2). The gene product is an apical membrane …show more content…
On one hand, it seeks to root out the cause of diseases which have no cure, rather than only treating its symptoms. However, treatments vary from disease to disease. In the case of cystic fibrosis, the effects of treatment do not last very long, and in SCID-X1, the treatment has led to risks of leukaemia. There is an ethical concern that it could modify human capabilities, thus altering the standards of normal human life. Gene therapy is also a very expensive form of treatment and hence should be regulated effectively. Gene therapy has a remarkable therapeutic potential (14) and this should be exploited. Through effective research and regulation, gene therapy has the potential to cure genetic diseases, eliminate any possible side effects and usher in a new standard of