Gene Therapy on the External Part of the Body

This semester, we have discussed the idea of injecting programmed/man made vectors into the body in order to help combat diseases and ailments. However, almost everything that has been discussed focuses on the inside of the body, such as diabetes, heart failure, and many other diseases. Despite the multitude of organs inside the body, most people do not associate skin as being an organ, let alone  being the largest organ in the body. This is one of the many reasons that skin is often less prioritized as an organ, and aside from putting sunblock on during the summer, some people don’t even think about their skin’s health or needs. For those with skin conditions such as junctional epidermolysis bullosa, gene therapy might be the answer to the question: do vectors and gene therapy work just as effectively on an external area? How will the body potentially reject it?

Unfortunately, the short answer is we just do not know yet. And we probably will not know for while. For treatment on the boy with junctional epidermolysis bullosa, his skin was extremely fragile and was expected to lead to more health problems throughout his life. The entire procedure was extremely expensive and tailored to his condition and genetic history. Yet, since it worked on him, the entire idea is currently batting 1.000 despite it only being tested on one person. The only definite going forward from this is that performing gene therapy on skin would have a distinct advantage since it is an entirely visible organ- a unique attribute that sets skin a part from anything other organ.

While some gene therapy patients pass away from the condition that they were originally trying to fight, the unfortunate truth is that some also pass away from the body rejecting the vectors in the system- and that makes perfect sense. Is every person’s body going to accept a foreign, man made substance that is manipulating the body from the inside? Of course not. Patients are monitored after undergoing gene therapy in order to see how the body is reacting from the gene therapy. During this process, blood, vital, and other tests are monitored to view how the body is behaving. Having the ability to visually monitor the organ is another asset in terms of detecting signs of the body rejecting the vector. Unfortunately, only time will reveal the effectiveness of this particular field of gene therapy. Here’s a video that explains how gene therapy is used for correcting skin sells:

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