Pros And Disadvantages Of Gene Therapy

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Gene therapy is now considered a new therapeutic area of study in modern medicine. Genes are special segments of DNA that provide the information to the body to properly function. It involves the transfer of genetic information into the tissues and organs of patients. As a result, it can be used to eliminate diseased genes or restore their normal functions. Another application of gene therapy involves the inclusion of different function to the cells, in order to either fight cancer or other diseases. The main target for gene therapy in the early days includes inherited diseases that are caused by single gene defects, for example cystic fibrosis (1). Gene therapy can either be performed by the direct transfer of genes or by the usage of living…show more content…
This is done by first harvesting and propagating cells of the patients or other sources in the lab. Then introducing the therapeutic gene into the cells through the direct gene transfer method. And finally, returning the newly modified cells into the patient. Both the methods have certain advantages and disadvantages. As the direct gene transfer method has the advantage of simplicity, however due to the over simplified strategy, this method does not allow for advanced manipulation and control over the therapeutic gene. At times the gene that is transferred remains non-integrated with the patient’s chromosome, hence just persisting in the targeted tissues. Another drawback is that there is not always easy access to the targeted tissue or organ. Whereas the second method of using living cells have the bigger advantage of advanced manipulation of the therapeutic genes as they are altered in-vitro rather than in-vivo, where it cannot be controlled. One of the types of living cells that can be used are stem cells, which have the ability to localize to certain parts of the human body hence is useful in applying the therapeutic gene localized specificity…show more content…
The human neural stem calls are genetically modified by the researches to produce cytosine deaminase, a protein that has the ability to transform a nontoxic precursor drug into a cancer cells killing active form. These genetically modified cells are then injected into the brains of stimulated human derived gliomas mice. Results have been found that gene therapy with the precursor drug has shrunk the tumor size by 80 % within two weeks. Through this experiment, it was also found that neural stem cells have the ability to rapidly and precisely situate glioma cells, regardless of the distance of injection of stem cells from the tumor (7). In another experiment conducted using osteoblasts, which are boneforming stem cells, to repair and regenerate bone, researches genetically modified osteoblasts to produce a bone growth factor. A biodegradable matrix was utilized as a scaffold as osteoblasts were added to it for new bone formation. This matrix was inserted into mice and results have been found that new bone formation was detected within a month of insertion. Even tough this is still in its very early stages and ongoing studies are in place, it offers an alternative to orthodox bone grafting methods

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