Somatic Gene Therapy Research Paper

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Somatic gene therapy has the potential has to be extremely advantageous to individuals but it also has the potential to do more harm than good to an individual. The concept of gene therapy is impeccable and has the capacity to achieve extraordinary results. In the process of somatic gene therapy, individuals with severe diseases are treated by an injection of a vector with the corrected gene for the disease in which they are attempting to fix. Typically, that vector is a deactivated virus; this way, the patient receives the corrected gene in an ideal manner but will not contract whatever disease is associated with the particular virus. However, although somatic gene therapy has potentially promising results, as of now the process still has …show more content…
Commonly used viral vectors such as adenovirus as AAV have unique immunogenic epitopes (identifier molecules) which induce an inflammatory response around the injection site. In addition, in a clinical trial that that took place in France that involved ten children resulted in the cure of boys with X-linked SCID (severe combined immunodeficiency) but in return, four children out of the ten involved developed leukemia along with one other child involved in a similar study taking place in the UK.
Additionally, a gene therapy clinical trial was being conducted at the University of Pennsylvania. An 18 year old patient by the name of Jesse Gelsinger was involved in the trial with hopes to correct his ornithine transcarbamylase (OTC) deficiency which a dangerous disease which can lead to the buildup of ammonia in the body. Jesse received the viral vector through a catheter through the groin artery which then traveled to his liver. Within hours of treatment, Jesse developed a fever and blood clots throughout his body. He then died four days after the attempted

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