Medical Genomics/Gene Therapy Medical Symposium Medical Genomics/Gene Therapy By: Molly Weiman, Ben Snyder, Bharadwaj Sudarsan, Jacob Myers, & William Grice

Introduction Video

The Genetic Link to Gene Therapy By: Jacob Myers As you have seen in the video, gene therapy replaces the dysfunctional genes, or puts in functional genes that are modified to not have the “defect” the old genes had. When we look at our defects, or our likelihood to get a disease or something else, we have to look back.

Continued Consider: We get all of our traits from all the generations before us. Naturally, we each have our own probability of something in our body not working as it should the second we are born. These include diseases we can get, to small things like poor sight. For millennia, we have not been able to change the base chances of us catching a disease, we could only try our best to help prevent it.

Procedure of Gene Therapy By: William Grice There are two cases in which Gene Therapy would be used. A requirement is, however, that there must be some disease or defect that needs to be fixed. Either the person is completely missing the needed gene, or the current gene they have is not working correctly. Nevertheless, the procedure generally stays the same, unless all that is needed is to “kick out” the bad genes. Say we are genetically modifying someone’s bone marrow to have more white blood cells.

Continued…. Scientists take out the defective genes, and modify them into healthy genes. Scientists start by attaching the normal, healthy gene they want to give the patient to a harmless virus called a vector. They remove genetic material from the vector and replace it with genetic instructions to make a healthy copy of the person’s missing gene. They then mix the vector and the new gene with bone marrow or blood from the patient.   The vector carrying the normal gene will penetrate the stem cells in the patient’s bone marrow/blood and replace the defective gene with the healthy gene. Doctors then grow these corrected cells in an incubator (this step is basically spreading the healthy genes). Once they have enough, they inject them into the patient. The bone marrow will gradually absorb them. The new cells can then start making healthy white blood cells able to fight infection.

Current Medical Practices VS. By: Molly Weiman By: Bharadwaj Sudarsan Medical Genomics Current Medical Practices Gene therapy uses genes to treat diseases. Previously, surgery, drugs, and other sorts of treatments were given to treat diseases. Gene Therapy involves injecting genes into the patient so that the genes can treat or prevent the disease(s) (like cancer, diabetes, etc.). They can replace the mutated gene with a healthy version of it, “knock out” an improperly functioning mutant gene, or introducing a new gene to help fight the mutant gene. They also normally have no noticeable side effects.

E x a m p l e s Previously, in cancer, the only 3 forms of treatment were surgery (surgically removing tumors), radiation therapy (Using X-rays to destroy tumors), and chemotherapy. Now, with gene therapy, one can just prevent cancer by injecting him/herself with a healthy gene. This procedure can be done to many other diseases like aneurism, thyroid, diabetes, etc. There used to be no cure for Chronic Granulomatous Disorder, a genetic disease in the immune system that makes patients unable to fight off bacterial and fungal infections. Now, investigators in Germany have used gene therapy to cure 2 of these patients, and for about 2 years, they have been able to successfully fight off microbial diseases. Others Include: Parkinson’s Disease, Hemophilia, and more…

The Future of Medical Genomics… By: Ben Snyder Grand Challenge I: Comprehensively Identify the Structural and Functional Components Encoded in the Human Genome The next phase of genomics is to catalogue, characterize and comprehend the entire set of functional elements encoded in the human and other genomes, which will be a challenge. Known classes of functional elements and others must be better understood. Other types of procedures must also be looked into. Similarly, a better understanding of epigenetic changes is needed to comprehend the full amount of ways in which DNA can encode information.

Grand Challenge II: Develop Strategies for Identifying the Genetic Contributions to Disease and Drug Response Deciphering the role of genes in human health and disease is a formidable problem for many reasons. Yet this problem can be solved. Development of new tools to catalyze advances in understanding the genetics of common disease and in other areas are needed. Prominent among these will be a detailed map of the human genome that can be used for whole-genome association studies of all diseases in all populations, as well as further advances in technology. More efficient strategies for detecting rare alleles involved in common disease are also needed.

Medical genomics can also be a hard topic for a patient’s mentality. Grand Challenge III: Genomics to Society Promoting the use of genomics to maximize benefits and minimize harms has been at the forefront of giving serious attention, to the impact of science and technology on society. Medical genomics can also be a hard topic for a patient’s mentality. Just as developments have spawned new areas of research in basic biology and in health, they have also created many opportunities for research on social issues.

Although the major benefits to be realized from genomics are in the area of health, genomics can also contribute to other aspects of society. Just as the HGP and related developments have spawned new areas of research in basic biology and in health, they have also created opportunities for research on social issues, even to the extent of understanding more fully how we define ourselves and each other.

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