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Ethical Considerations of Genetic Research, Therapy & Technology

Instructor: Kerry Gray

Kerry has been a teacher and an administrator for more than twenty years. She has a Master of Education degree.

In this lesson, we will define important concepts in genetic research, including genomes, DNA, gene therapy, cloning, and genetic engineering. We will discuss ethical considerations of genetic research, therapy, and technology.

Why Genetic Research?

Imagine a cure for cancer that doesn't involve drugs or surgery. It may be closer than we think. Genetic research is still underway to determine if gene therapy can be used to treat a variety of medical disorders, including some cancers. Gene therapy includes replacing unhealthy genes with healthy ones, destroying mutated genes that cause illness, and adding disease-fighting genes to cells. Let's find out more about gene therapy and the ethical considerations of this line of research.

Gene Therapy
Gene Therapy

Genetic Concepts

What are the nuts and bolts of gene therapy? Every cell in our body that contains a nucleus (center) contains a genome. Genomes contain a complete set of DNA, or deoxyribonucleic acid, which is all the hereditary information needed to create and maintain everything about you.

Scientists have figured out how to duplicate genomes for the purpose of cloning and genetic engineering in animals. Cloning creating a biological duplicate of genes, cells, tissues, or organisms. Genetic Engineering is reproducing animals with desirable traits using DNA technology. For example, more muscular cows can be produced. In theory, these techniques could also be used to clone or genetically engineer human beings, but ethical guidelines prohibit using genetic research in this way.

Ethical Considerations of Germline Gene Therapy

Genetic research on humans has focused on treating bone marrow or blood cells rather than germline therapy. Germline gene therapy is the treatment of egg and sperm cells, which could be passed on to offspring. Germline gene therapy could potentially stop a genetic disorder from continuing on to the next generation, but knowledge is limited regarding long-term side effects. This creates an ethical dilemma as offspring are unable to provide consent for this type of treatment that could create problems down the road. Currently, the United States does not use federal tax dollars to be spent on research using germline gene therapy on humans due to controversy surrounding this practice.

However, the crude methods in which even somatic (cells that are not germline) cells are injected with genes creates the potential that germline cells would be affected. Currently, gene transfers take place within a virus that could potentially reach unintended cells.

Clinical Trials

Initial studies are promising, but at this point, the only way to receive gene therapy is through clinical trials. Gene therapy has had some success in trials at treating some immune deficiency disorders, such as Severe Combined Immune Deficiency (SCID) and Adenosine deaminase (ADA) deficiency. To treat these disorders, stem cells are removed from the patient, treated with functioning genes, and then the stem cells are returned to the patient. For both disorders, the immune deficiency improved, but for SCID, the trial was abandoned when some patients developed leukemia.

Studies on hereditary blindness have shown that gene therapy has the potential to slow or reverse the disease for a while. However, after a few years, the retina continued to degenerate. The eye appears to be a good organ for using somatic gene therapy as it contains the virus, preventing it from spreading to germline cells.

Gene therapy appears to be effective for introducing clotting genes to hemophiliacs. Other blood disorders, such as sickle cell anemia, can be treated by introducing genes that produce healthy red blood cells after stem cells are removed from the patient.

Injecting dopamine-producing genes into brain cells has been shown to improve muscle control in patients with Parkinson's disease.

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