Objectives: At the end of the lesson, the learners should be able to:
o Discuss the ethics and implications of Gene Therapy.
o Discuss the major impacts (both potential and realized) of Gene therapy.
ENGAGE
1. What do you think are some ethical issues surrounding gene therapy? 2. Should people be allowed to use gene therapy to enhance basic human traits such as height, intelligence, or athletic ability? 3. How does gene therapy work?
DISCUSSIONS
What is Gene Therapy?
Gene therapy is designed to introduce genetic material into cells to compensate for abnormal genes or to make a beneficial protein. If a mutated gene causes a necessary protein to be faulty or missing, gene therapy may be able to introduce a normal copy of the gene to restore the function of the protein. Additionally, Gene Therapy is an experimental technique that uses genes to treat or prevent disease. In the future, this technique may allow doctors to treat a disorder by inserting a gene into a patient‟s cells instead of using drugs or surgery. Researchers are testing several approaches to gene therapy, including:
1. Replacing a mutated gene that causes disease with a healthy copy of the gene. 2. Inactivating, or “knocking out,” a mutated gene that is functioning improperly. 3. Introducing a new gene into the body to help fight a disease.
Although gene therapy is a promising treatment option for a number of diseases (including inherited disorders, some types of cancer, and certain viral infections), the technique remains risky and is still under study to make sure that it will be safe and effective. Gene therapy is currently being tested only for diseases that have no other cures. How does Gene therapy works?
Gene therapy techniques
There are two different types of gene therapy depending on which types of cells are treated: 1. Somatic gene therapy: transfer of a section of DNA to any cell of the body that doesn‟t produce sperm or eggs. Effects of gene therapy will not be passed onto the patient‟s children. 2. Germ line gene therapy: transfer of a section of DNA to cells that produce eggs or sperm. Effects of gene therapy will be passed onto the patient‟s children and subsequent generations.
Gene augmentation therapy
This is used to treat diseases caused by a mutation that stops a gene from producing a functioning product, such as a protein. This therapy adds DNA containing a functional version of the lost gene back into the cell. The new gene produces a functioning product at sufficient levels to replace the protein that was originally missing. This is only successful if the effects of the disease are reversible or have not resulted in lasting damage to the body. For example, this can be used to treat loss of function disorders such as cystic fibrosis by introducing a functional copy of the gene to correct the disease. Below is a simple illustration that shows how gene augmentation therapy is done.
Gene inhibition therapy
Suitable for the treatment of infectious diseases, cancer and inherited disease caused by inappropriate gene activity. The aim is to introduce a gene whose product either: inhibits the expression of another gene interferes with the activity of the product of another gene The basis of this therapy is to eliminate the activity of a gene that encourages the growth of disease-related cells. For example, cancer is sometimes the result of the over-activation of an oncogene (gene which stimulates cell growth). By eliminating the activity of that oncogene through gene inhibition therapy, it is possible to prevent further cell growth and stop the cancer in its tracks. Killing of specific cells
Suitable for diseases such as cancer that can be treated by destroying certain groups of cells. The aim is to insert DNA into a diseased cell that causes that cell to die. This can be achieved in one of two ways: the inserted DNA contains a “suicide” gene that produces a highly toxic product which kills the diseased cell the inserted DNA causes expression of a protein that marks the cells so that the diseased cells are attacked by the body‟s natural immune system. It is essential with this method that the inserted DNA is targeted appropriately to avoid the death of cells that are functioning normally.
How is DNA transfer done?
A section of DNA/gene containing instructions for making a useful protein is packaged within a vector, usually a virus, bacterium, or plasmid. The vector acts as a vehicle to carry the new DNA into the cells of a patient with a genetic disease. Once inside the cells of the patient, the DNA/gene is expressed by the cell‟s normal machinery leading to production of the therapeutic protein and treatment of the patient‟s disease. The illustration shows the transfer of a new gene into the nucleus of a cell via a viral vector
Is gene therapy safe?
Gene therapy is under study to determine whether it could be used to treat disease. Current research is evaluating the safety of gene therapy; future studies will test whether it is an effective treatment option. Several studies have already shown that this approach can have very serious health risks, such as toxicity, inflammation, and cancer. Because the techniques are relatively new, some of the risks may be unpredictable; however, medical researchers, institutions, and regulatory agencies are working to ensure that gene therapy research is as safe as possible (Genetics Home Reference)
Challenges of gene therapy
Delivering the gene to the right place and switching it on: it is crucial that the new gene reaches the right cell delivering a gene into the wrong cell would be inefficient and could also cause health problems for the patient even once the right cell has been targeted the gene has to be turned on cells sometimes obstruct this process by shutting down genes that are showing unusual activity Avoiding the immune response: The role of the immune system is to fight off intruders. Sometimes new genes introduced by gene therapy are considered potentially-harmful intruders. This can spark an immune response in the patient that could be harmful to them. Scientists therefore have the challenge of finding a way to deliver genes without the immune system „noticing‟. This is usually by using vectors that are less likely to trigger an immune response. Making sure the new gene doesn‟t disrupt the function of other genes: Ideally, a new gene introduced by gene therapy will integrate itself into the genome of the patient and continue working for the rest of their lives. There is a risk that the new gene will insert itself into the path of another gene, disrupting its activity. This could have damaging effects, for example, if it interferes with an important gene involved in regulating cell division, it could result in cancer. The cost of gene therapy: Many genetic disorders that can be targeted with gene therapy are extremely rare. Gene therapy therefore often requires an individual, case-by-case approach. This may be effective, but may also be very expensive.
References:
Macnamara, D., Valverde, V., & Beleno III, R. (2018). Science, Technology, and Society. Quezon City: C&E Publishing, Inc.
Serafica, J. J., Pawilen, G. T., Caslib, B. N., & Alata, E. P. (2018). Science, Technology, and Society. Sampaloc, Manila: Rex Book Store, Inc.
Taguiling, M. (2019). Science, Technology and Society. Plaridel, Bulacan: St. Andrew Publishing House.
https://www.yourgenome.org/facts/what-is-gene-therapy Name: ____________________________________________ Program, year, and section: _______________________
Activity 15: Answer the following questions in 3-5 sentences. Detach the worksheet once finished.
1. How can “good” and “bad” uses of gene therapy be distinguished?
___________________________________________________________________________________________ ___________________________________________________________________________________________ ___________________________________________________________________________________________ ___________________________________________________________________________________________ ___________________________________________________________________________________________ ___________________________________________________________________________________________ ___________________________________________________________________________________________ ___________________________________________________________________________________________ 2. Could the widespread use of gene therapy make society less accepting of people who are different? ___________________________________________________________________________________________ ___________________________________________________________________________________________ ___________________________________________________________________________________________ ___________________________________________________________________________________________ ___________________________________________________________________________________________ ___________________________________________________________________________________________ ___________________________________________________________________________________________ ___________________________________________________________________________________________ ___________________________________________________________________________________________
3. Will the high costs of gene therapy make it available only to the wealthy? ___________________________________________________________________________________________ ___________________________________________________________________________________________ ___________________________________________________________________________________________ ___________________________________________________________________________________________ ___________________________________________________________________________________________ ___________________________________________________________________________________________ ___________________________________________________________________________________________ ___________________________________________________________________________________________ ___________________________________________________________________________________________ ___________________________________________________________________________________________
