Abstract
In today’s society, there are numerous children that are born with a childhood disease that is life-threatening. Many children do not live past the age of five with some of these debilitating diseases. As a result, researchers are tackling the crisis by editing the mutated gene of the child while in gestation. Therefore, this will eliminate the possibly that the child is born with a disease. Many of the current studies are in animal trials but will soon be in human trials. Prenatal gene therapy will help a number of people but will also bring about ethical questions that may be difficult to answer at times.
Introduction
In today’s society, many people are affected by many diseases which can be deadly. A plethora of these diseases are contributed to genes being mutated in their DNA. As a result, researchers have looked into correcting the mutation with gene therapy. Gene therapy can be seen in many forms. Gene therapy is the process of removing or inactivating a certain unwanted gene. (Cox & Nelson, 2008) Furthermore, gene therapy is on the cusp of becoming a great tool for elevating harmful diseases that possess life-threating affects. Researchers are now able to specify what level of gene therapy they can work upon. Now, prenatal gene therapy is an upcoming technique that could possibly save many lives and give a better quality of life to both infants and parents.
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In terms of the regular prenatal care, the mother will have to have extra scans and tests to examine that the fetus is not presenting the mutated gene anymore and the serum is working and replicating the correct gene instead of the mutated one. Also, doctors will examine the mother to ensure that the mother is adjusting to all the new changes. The fetus will be subjected to many regular blood drawings and ultrasounds that a normal pregnancy would not have to be subjected to. Also, once the child is born, more tests will be completed in order to determine how the child is managing the treatment and to see how effective the treatment was.
Moreover, there are numerous childhood diseases, syndromes, and cancers that affect infants every day. Many of these aliments are life-altering, not only to the child, but also to the parents. Some are so incredible lethal that the child sometimes dies in the womb. Therefore, prenatal gene therapy comes into play and helps to alleviate this hardship for both parties. Prenatal gene therapy is being unveiled as a revolutionary technique due to the fact that scientists now have the tools and methods to identify and eliminate potential life-threating mutated genes. As a result, the serum will correct the affected enzymes to signal the correctly folded protein or directly from the correct protein for the specific usage. (Cox & Nelson, 2008) Prenatal gene therapy involves injecting a serum that has been treated and placed in a viral vector to treat the mutation that is causing the disease to appear into the fetus. (DeWeerdt, 2018) The serum can be injected into the fetal tissue, umbilical blood vessel, or the amniotic fluid; this procedure is most likely done with the assistance of an ultrasound probe. (DeWeerdt, 2018)
Mechanism of Action
Understanding how genes function within the body is an extremely important task for gene therapy. Before one can manipulate genes and expect a pharmaceutical effect from the procedure, researchers first had to go to a microscopic level and understand the roles of genes. Genes are classified as a section of DNA that controls the function of making proteins. (Allison, 2012) These sections of DNA have sequences that can be easily mutated and cause adverse effects. Thus, gene therapy can be utilized and alleviate what issues that stems from the mutations. (HemDifferently) Gene therapy functions by being administered through an injection. The serum inside the injection is formulated by a vector that contains the corrected version of the gene that has been altered. (HemDifferently) This entire process, once inside the host, begins a cascading affect. The corrected form of the gene will first enter the nucleus of the target cell, then, the new sequence will become intertwined with the chromosome. (HemDifferently) As a result, the new gene will instruct the body to make the correct protein. (HemDifferently) Although most of the harmful mutations would have been alleviated, the mutated gene will still be present, but not at the extent before the correction. Moreover, prenatal gene therapy is very similar to the broad-spectrum gene therapy. The only difference is that prenatal gene therapy is administered in a more specific site and during a specific time. The injection will be administered to a part of the mother that is directly connected to the fetus, or to the fetus itself. Also, it will be given while the fetus is still in gestation. (HemDifferently) Therefore, the mutated genes are altered before the fetus is fully developed and enhances the chance of the fetus of having a lower chance at an abnormal genome. (HemDifferently)
Clinical Uses
Moreover, the immune system of a fetus is highly underdeveloped and is extremely attractive for prenatal gene therapy due to the changing environment. (Mehta, et al., 2012) Also, the stem cells are actively proliferating and will be able to form into anything that it is being induced to become. (Mehta, et al., 2012) Researchers are now developing a goal to use prenatal gene therapy for relevant techniques in the clinical setting to not ameliorate diseases while in utero, however, most studies are currently in animal studies. (Mehta, et al., 2012) Furthermore, these models are efficient for analysis of potential harm and risk to both the fetus and the mother. (Mehta, et al., 2012) The animal model is congruent to humans and will allow researchers to determine how the technique will affect the human.
Moreover, testing is very important to keep a data log of what is happening to the animal, therefore, researchers will be able to determine how prenatal gene therapy will affect both the fetus and the mother. (Mehta, et al., 2012) The animal model that is used is a sheep and is very docile to a series of blood sampling and slightly invasive ultrasound-led blood tests. Also, for long-term monitoring, scientists use a technique called telemetric to measure the different functions of the body. (Mehta, et al., 2012) Telemetric is the collection of measurements or data for a certain accessible point. These measurements are taken to monitor how both fetus and mother are doing during the process. (Mehta, et al., 2012) Lastly, to ensure that the fetus is healthy, a catheter can be placed to detect fetal blood during gestation. (Mehta, et al., 2012) Furthermore, researchers have found a way to screen the events near birth and long-term neonatal supplement, which will be important for the translation from an animal model to human studies for the therapy. (Mehta, et al., 2012)
Case Studies
Researchers are now conducting a trial in mouse studies that test to see if a brain disorder can be corrected by gene therapy. The mice used in this trial had a defect in a gene called GBA, which codes for the enzyme inducing the breakdown of glucocerebroside. (DeWeerdt, 2018) The inactivation of this enzyme will lead to a build-up of glucocerebroside in the brain, which subsequently leads to brain damage that is irreversible. As a result, the mice would typically die within two weeks of birth. (DeWeerdt, 2018) This model is in correlation with the human disease called acute neuronopathic Gaucher’s disease. (DeWeerdt, 2018) This disease is a genetic childhood disease that usually takes the life of the child by the age of two. Fortunately, in this experiment, scientists using gene therapy elevated the problem by injecting the mice with the GBA in their brains while they were still in gestation. (DeWeerdt, 2018) Results showed that the mice that were treated were born normally and lived eighteen weeks before showing signs of brain damage symptoms. (DeWeerdt, 2018) Researchers in this experiment are concluding that gene therapy can significantly prolong life and diminish the signs of pathology while continuing gestation.
Moreover, hemophilia B can be treated by prenatal gene therapy. Hemophilia B is a mutation in the clotting factor gene, F9. (Waddington, et al., 2014) As a Result, hemophilia B will cause an uncontrollable bleed to a puncture wound. Currently, the disorder is treated by recombinant protein substitution, which is expensive and has its limitations. (Waddington, et al., 2014) Thus, studies are being conducted in prenatal gene therapy to determine if it can be a helpful tool to cure the disorder before the child is born. In this study, mice were used to study the lasting therapeutic correction of the disease without the developing of an immune-mediated response. (Waddington, et al., 2014) The therapy was administered by a HIV-based vector that encodes for the human factor IX protein into the fetal circulation. After the rounds of treatments, scientists discovered with a coagulation assay, that the mice that were tested, bleeding stopped after being punctured. (Waddington, et al., 2014)
Ethics
When dealing with alterations of the body, such as prenatal gene therapy, these topics can lead to many questions and concerns. There are many pros and cons to weigh out when dealing with prenatal gene therapy. One should look at both sides of the problem before deciding what course of action should be taken. After asking the many questions like: “Is it morally correct to alter the basic instructions of life?” “Is it morally sound to play God?” One should further their study and look on the side of how helpful the procedure could be. (Genetic Home Reference) Although, there are plenty of possible wrongdoings in the procedure, there are also many outcomes that can be beneficial for a family. By undergoing the procedure of prenatal gene therapy, many children could be born without aliments, that possible previous generations would have not had the opportunities. (Genetic Home Reference) Also, this could help later generations to be born without the mutation. Furthermore, research has shown that many of the studies in prenatal gene therapy has been successful and could possibly soon be found in human trials and decrease the child death rate due to childhood illness.
Moreover, another aspect of prenatal gene therapy to consider is the possibility of vanity and self-interest. Although prenatal gene therapy has so many great values, it can also be a tool to separate the rich from the poor even more. Because some people will not be able to afford the procedure, this will cause the less fortunate to continue to deal with deadly childhood diseases that could be possible altered if they had the financial abilities. Another question that may arise is what should be considered as operable and what is out of scope for prenatal gene therapy. (Genetic Home Reference) For example, should prenatal gene therapy be used to enhance the looks of one’s child or make the child intelligent? (Genetic Home Reference) When looking at prenatal gene therapy this way, one can say that people may try to make “superkids” rather than using the treatments for medical reasons. Overall, prenatal gene therapy is a great technique, but it also comes with a plethora of concerns that could possibly outweigh the good.
Discussion
There are many factors that play a part in making prenatal gene therapy successful. Although the different ways to use prenatal gene therapy is currently still in clinical trials, researchers can see that prenatal gene therapy will have a promising future for humans once it makes it to human trials.
Conclusion
In conclusion, in the near future, children will have the ability to live a prolonged healthy life without the disease that could have possibly killed them. Researchers now can elevate the possible chance of presenting a genetic disorder. (Journal of Genetic Syndromes & Gene TherapyOpen) Prenatal gene therapy allows researchers to correct the affected gene about the tenth and thirteenth weeks of pregnancy. (Journal of Genetic Syndromes & Gene TherapyOpen) The process involves measuring certain levels of metabolites in the mother’s circulatory system and receive an ultrasound. These tests will allow researchers and doctors to elevate the genetics of the fetus to determine if a prenatal gene therapy intervention is what is best for the patient. (Journal of Genetic Syndromes & Gene TherapyOpen) In addition, prenatal genetic testing can be useful for the prognosis of a high-pregnancies. The testing is performed on either skin, hair, blood, other tissue, or amniotic fluid. (Journal of Genetic Syndromes & Gene TherapyOpen) Resulting in a positive result, means that a particular gene is mutated, a chromosome is altered, or a protein is improperly folded. A negative result simply means no alteration was found in the genes, chromosomes, or the protein. (Journal of Genetic Syndromes & Gene TherapyOpen)
Moreover, prenatal gene therapy is performed by injecting a serum into the fetus via the mother. This process is currently in animal trails and the method is very successful in the animal models. Many of these trails are performed in animals that have a similar mechanism of action to humans. (Cross, 2018) Some of the debilitating diseases that are apparent in children can be reversed by prenatal gene therapy and studies have shown just that. (Cross, 2018) Prenatal gene therapy has a great many of positive outcome through the treatment, however, there are some draw backs to the method. Many people are still outweighing the “good” versus the “bad” of prenatal gene therapy. (Genetic Home Reference) However, there is a momentous amount of good within this treatment. Millions of children could live well past the age of five and not have a life-threatening disease.
Bibliography
- Waddington, S. N., Nivsarkar, M. S., Mistry, A. R., Buckley, S. M. K., Kemball-Cook, G., Mosley, K. L., … Coutelle, C. (2004, November 1). Permanent phenotypic correction of hemophilia B in immunocompetent mice by prenatal gene therapy. Retrieved from http://www.bloodjournal.org/content/104/9/2714.full.
- DeWeerdt, S. (2018, December 12). Prenatal gene therapy offers the earliest possible cure. Retrieved from https://www.nature.com/articles/d41586-018-07643-z.
- Allison, L. A. (2012). Fundamental molecular biology. Hoboken, N.J: Wiley.
- Welcome to HemDifferently by BioMarin. (n.d.). Retrieved from http://hemdifferently.com/.
- Mehta, V., Abi-Nader, K. N., Carr, D., Wallace, J., Coutelle, C., Waddington, S. N., … David, A. L. (2012). Monitoring for potential adverse effects of prenatal gene therapy: use of large animal models with relevance to human application. Retrieved from https://www.ncbi.nlm.nih.gov/pubmed/22648778.
- Cox, M. M., & Nelson, D. L. (2008). Principles of Biochemistry: Lehninger. New York: W H Freeman and Company.
- Journal of Genetic Syndromes & Gene TherapyOpen Access. (n.d.). Retrieved from https://www.longdom.org/scholarly/prenatal-genetic-testing-journals-articles-ppts-list-2032.html.
- Cross, R. (2018, July 18). Fetal gene therapy could treat genetic diseases in the womb. Retrieved from https://cen.acs.org/pharmaceuticals/gene-therapy/Fetal-gene-therapy-treat-genetic/96/i30.
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