GENETICS

Chapter Review Gene therapy, the Perfect Cure. How close is science to create a genetically perfect human? To what extend is ethical the modification genes?

womb (University of Utah). Back in the history of medicines, genetic diseases were incurable. However, thanks to the discovery of DNA in the 90st and the understanding of Many people would argue that the purpose of science is to discover better ways to live. Creating the perfect human it is not juts Hollywoods dream. As far as science is concern, it is nearly impossible to create a human with perfect genes. Many of ones unique abilities and personalities are consequence of the surrounding environment one live. If we could in reality create a perfect human with super strength, good morals and super intelligence; this person would not be consider a human; he would be an automated living specie design to be what people believe to be perfect. In other words, science cannot create a human with predestinated genes. However, science can in reality fix underlying genetic problems that affect an individuals health. There are two types of disease: genetic diseases and non-genetic diseases. Genetic diseases are characterized by the malfunction of protein synthesis in the genes of an individuals body (University of Utah.). Nongenetic diseases are illnesses that are cause by the environment and individual resides (University Of Utah). Genetic diseases can be inherited from family ancestors, but often times genetic diseases are not inherit; they are the consequence of bad mutation in humans cause by carcinogens during the fetus development in the mothers
(Public Domain)

protein synthesis and amino acids, scientist discover a way to cure genetic disorder (Mcvister). The process of curing genetic disorder is call Gene Transfer or Gene Therapy. Its purpose is to find the underlying protein causing the health complications in an individual (University Of Utah).

the body (FDA). However, once the gene has been is located and the problem has been identified, the process Gene therapy is defined as the process of modification of DNA or RNA of a specific malfunctioning cell with the purpose to change the biological properties express in cells organ (Steinbrook). That is, the modification of the protein molecule produced by the cells DNA responsible for the malfunction of the organ. Gene therapy offers a new way to address genetic disorders fixing it from the source of the problem (University Of Utah). This is the reason gene therapy is different from the traditional drug-based treatment. Instead of controlling the symptoms with drugs, gene therapy fixes the underlying genetic flaw within the organs cell (University Of Utah). can begin. Scientist will then try to modify the corresponding protein molecule in the cell in order to contra rest the health problem (University Of Utah). Later on to transfer a corresponding protein molecule to try to teach the cell how to produce that same protein molecule fabricated in the lab. Gene therapy can be very risky for several reasons. If the protein molecule is transferred to the wrong gene in the cell, the cell will mutate, causing another genetic disorder and perhaps the dead of the person being treated (FDA). Also the fact that gene therapy is a topic that has not been research as much makes the process highly dangerous since scientist are not sure what exactly is the function of each protein molecule in the individuals body. We have to Gene therapy may seem as the perfect cure, however little research has been done since it was first discover making it a dangerous medical procedure. This is why the FDA has set very strict ethical guidelines concerning gene transfer; if the procedure does not cure the patient, the patient may die (NIH). Doctors have to experiment with the patients DNA in order to find the protein molecule causing the patients health to decay. Each cell in the human system produces about 5,000 protein molecules; it is a difficult job for scientists to isolate the one single protein molecule and be sure that is the molecule responsible for all the damage in keep in mind that everybody is different; therefore each individuals system is unique and the each does different functions. Discoveries are being made slowly since nineties, not all genetic disorders are suitable for gene therapy for now.

For scientist to consider Gene therapy as an option, there are certain standards a disorder must reach (University of Utah). The mutation must occur in one or more genes (University Of Utah). Once the mutation has been located, the gene is copied and kept in the laboratory for further studies. The studies consist on learning how the mutation affects the tissues, what tissues it affects, and it what organ or organs (University Of Utah). After studying the mutation, scientists explore what would be the best possible way to address the problem. One of the most common ways used is adding a new copy of gene that has not been mutated. However, the risk of adding a new copy of the gene is the possibility that the original mutation in the person might be producing a protein contra resting the proteins of the unmated gene created in the laboratory (University Of Utah). Mutations that act this way when a new artificial non mutated copy of the gene is added are called dominant negative. A possible way to deal with the dominant negatives is either get rid of the mutated genes product or get rid of the mutated gene. There are several techniques to do this process. In this technique instead of replacing the entire gene, SMaRT repairs the mRNA transcript hosting the mutation responsible for the genetic disorder (University Of Utah). In other word, SMaRT targets and repairs the damaged transcript by the mutation (University Of Utah). It does not get rid of the protein molecule in the DNA responsible for of the patients illness.

This technique prevents the mutated gene to be transcript by the RNA. It delivers short pieces of DNA called oligonucleotides that binds in between the double strand of mutated genes DNA (University Of Utah). This helps the produce a triple-helix structure that prevents the mutated DNA to be transcript into the mRNA (University Of Utah). This way preventing the mutated DNA produced the bad protein molecule. This technique is very similar to Antisense technique with the difference that this technique completely prevents the production of mutated protein molecule.
Retrieved from The University of Utah, Genetics. A visual of Antisense Technique.

Retrieved from the University Of Utah, Genetics. A visual of Ribozyme Technique.

Retrieved from the University Of Utah, Genetics. A visual of Ribozyme

This technique attempts to in a sense disabled the mutated gene in a cell by isolating the messenger RNA that has been copied from the mutated gene (University Of Utah).

Technique.

Once the scientists have chosen a correct technique to deliver and fix the underlying problem, the gene is delivering. The big question is, is the tissue accessible? To what extent the tissue is accessible? Usually, tissues such as blood, skin, and lungs are the easiest to reach in contrast with internal organs such as pancreas or the nervous system (Utah University). Once all these questions and issues are address, the cure gene is delivering. Scientists are trying to modify viruses with the purpose to use them as vectors without getting the patient sick (University Of Utah). Three of the advantages that viral vectors are: They are very good at isolating the target and accessing into the cell, some of these viral vectors are designed to target specific kinds of cells and they can be modified so that they do not replicate and get the patient sick (University Of Utah). Nevertheless, viral vectors have disadvantages as wells. Three of the disadvantages are the fact that protein molecule can be too big to fit in the viral There is no best way of delivery in gene therapy. However, a successful way of delivery consists in getting the fixed DNA in the cell in order for the DNA to start producing a non-mutated protein molecule for the tissue to work right (University Of Utah). The DNA delivery transportation is known as vector. This is the most difficult part of gene therapy; finding the best way to deliver the fixed DNA (University Of Utah). As mention before, if the fixed DNA is delivering to the wrong cell, the patient may die. Despite of the difficulty of delivering the fixed DNA to the cell, scientists have discovered two vectors for the same purpose of delivery. Non-viral vectors consist on using plasmid, a circular DNA molecules used in bacteria to transfer gene from cell to cell. Plasmids are usually used to replicate DNA of interest from any organism (University of Utah). The only disadvantage that non-viral vectors might have is if the vector is not targeting the right cell. vectors, patients may become sick and patients immune system can act up against viral victor (University Of Utah).

should be treated as rat of laboratory; on the contrary, patients should be treated as humans (Steinbrook).

As you have learned, gene therapy is a very complicate technique to use. Even more complicated are the Ethical guidelines the government has set on the procedure. After the Bubble boy case, the kid who lived in the bubble his entire life little research has been done on the topic (Mcvister). Especially since Bubble boy died a day after the procedure was done. Another case that raised many questions about the Ethical guidelines concerning Gene therapy is the Gelsinger Case. The eighteen year old boy, after being diagnose with a severe liver disorder, the boy was asked by the doctor is the University of Pennsylvania to work on his case (Steinbrook). After several studies about the genetic disorder, Gelsinger was exposed to artificial genetic material that later was delivered to the wrong cells causing his dead (Steinbrook). Many argued that the death of Gelsinger could have been prevented. The University was more interested in making a profit out of the discovery rather than saving the life of the young boy (Steinbrook). This is the reason the government has set strict guidelines concerning gene therapy. Gene therapy is a new procedure that can potentially be the answer of many other illnesses like cancer (FDA). However, this does not mean patients

Gene therapy has the potential to cure genetic disorder from the source of the problem. The idea of creating a super human by using gene therapy is unethical and impossible in scientific terms. Scientist would have to experiment with other humans and this might include not respecting there rights. Is important to remember that gene therapy is design for special case where the only answer is the modification of the Individuals DNA. Gene therapy is a dangerous tool and must be used only in extreme case. Nevertheless, more research should be done in gene therapy in order to find the perfect cure.