Gene Therapy 2 Overview On Gene Therapy CHEM 120 ✓ Solved

Gene Therapy 2 Overview on Gene Therapy CHEM 120: Intro

Gene therapy is an experimental design that uses genes for preventive or therapeutic measures. In the nearest future, this approach may allow health professionals to treat various disorders by introducing a gene into a patient’s cells rather than using medications or surgical approaches. Researchers are analyzing several concepts of gene therapy, including exchanging a mutated gene that causes disease with a healthier copy, deactivating a malfunctioning mutated gene, and initiating a new gene into the body to help combat diseases.

Although gene therapy presents an intriguing treatment option for numerous inherited disorders, certain forms of cancer, and specific viral diseases, it is currently undergoing experimental analysis solely for deadly, incurable conditions. The new gene can alter how cells behave by either destroying the mutated version to modify its effects or replacing a faulty gene. The primary target of treatment in gene therapy is genetic disorders. However, gene therapy is still in its experimental phase, and health professionals are uncertain not only about its full potential but also about the long-term effects on treated patients.

Some risks identified thus far include cancer, inflammation, and toxicity. One of the major concerns related to gene therapy is the possible immune response toward the new gene. A notable case involved a patient named Jesse Geisinger, who died in 1999 from complications due to an inflammatory response after his immune system reacted negatively to experimental gene therapy aimed at treating a rare liver disorder. This incident led to a suspension of gene therapy and raised ethical questions about the benefits versus the risks of gene therapy. Although safer methods for administration have been developed, including pre-treatment with immune suppressants or delivering modified viruses to cells outside the body, the ethical implications of experimenting on humans have not been completely resolved.

While some argue that modifying an individual’s genes may carry unpredictable outcomes, others express concerns that the expensive nature of gene therapy could limit access to wealthy individuals. Despite recognizing Jesse's death and the potential financial barriers, proponents argue that the benefits of gene therapy are significant and could be adapted to benefit a broader population as more knowledge becomes available. One of the most promising benefits discussed is the potential to address genetic diseases in developing embryos.

If gene therapy develops successfully, medical practitioners could identify genetic diseases in infants and potentially administer treatments that would cure these conditions at an early age. However, this advancement raises ethical questions, particularly from religious groups that object to the procedure because it cannot be consented to by babies. Concerns include the potential misuse of gene therapy for non-life-threatening conditions like vision correction or even selecting physical traits. This fear parallels historical abuses, such as those associated with efforts to promote a so-called “master race.”

As summarized by Moshier, there are significant concerns regarding the potential for social discrimination against individuals who do not undergo genetic enhancement. The health community acknowledges that while gene therapy could enable parents to select desirable traits for their children, their focus remains on addressing chronic and life-threatening genetic disorders. The historic first recipient of gene therapy was a four-year-old girl with a severe immune deficiency disease. After the procedure, which involved genetically modifying her cells, she began producing the necessary enzyme and has since lived a relatively normal life.

Successful applications of gene therapy in treating conditions such as immune deficiencies, hereditary blindness, and blood disorders have fostered optimism among medical scientists about the potential to cure more complex diseases like cancer and sickle cell disorders. The insights gained from ongoing gene therapy research are invaluable, enhancing our understanding of DNA and hereditary diseases and manipulating viruses to target mutated genes effectively.

While the advantages of gene therapy seem to outweigh the risks for patients with severe chronic conditions, its long-term effects on health and future generations remain uncertain. Some patients facing dire health situations may prioritize immediate benefits, yet researchers caution that the repercussions may extend to their offspring. With gene therapy designed to deliver permanent alterations, there is a risk that the modified gene may produce adverse mutations in subsequent generations.

Research into mitochondrial function, through studies involving stem cells, has revealed that even optimistically engineered treatments can revert to having harmful effects. An instance of this involved three-parent babies where faulty mitochondria were exchanged, but the original defective genes eventually resurfaced three times, indicating the potential for unforeseen setbacks in gene therapy. Despite the experimental nature of gene therapy and inherent risks, the possibility of permanently curing certain diseases offers hope and excitement for the future of medicine.

Thus, while it's essential to approach gene therapy with ethical sensitivity and respect for human dignity, research in this field should not be entirely halted. The future of medical intervention might hinge on how well we understand the pros and cons of gene therapy, making it vital to continue exploring this promising avenue of treatment.

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References

• Genetics Home Reference. (2020). What is gene therapy? Genetics Home Reference.
• NIH. (2018). Gene Therapy: A Focus on Safety.
• Lewis, A. (2015). The Future of Genetic Treatments.
• University of Utah. (n.d.). The Gene Therapy Trials.
• Moshier, E. (2017). Ethical Concerns in Genetic Manipulation.
• Zallen, D. (2014). Ethical Issues Around Genetic Enhancement. Nature.
• Neimark, J. (2017). Studying Mitochondria and Gene Therapy.
• Better Health. (2017). Understanding Gene Therapy Effectiveness.
• Rocholl, J. (1996). Initial Success in Gene Therapy: A Case Study.
• RSRevision. (2015). Gene Therapy and Its Implications.