{"id":8,"date":"2016-09-18T21:59:11","date_gmt":"2016-09-19T02:59:11","guid":{"rendered":"https:\/\/eportfolios.isucomm.iastate.edu\/sanddog2\/?p=8"},"modified":"2016-11-28T19:04:03","modified_gmt":"2016-11-29T01:04:03","slug":"gene-therapy","status":"publish","type":"post","link":"https:\/\/eportfolios.isucomm.iastate.edu\/sanddog2\/2016\/09\/18\/gene-therapy\/","title":{"rendered":"Gene Therapy"},"content":{"rendered":"

What is the first thing you think of when you hear gene therapy? Some of the words that come to mind may be unnatural, suspicious, or even has potential. Gene therapy<\/a> is a type of treatment used to treat or prevent diseases. While it is still under research to determine its effectiveness and potential hazards, this technology could be the answer to curing certain diseases.<\/p>\n

What are Genes?<\/strong><\/p>\n

Genes, located inside chromosomes,<\/a> are made up of DNA and have a role in making proteins. In other words, genes act as a blueprint for the body. Every person obtains a copy of each gene from their parents. Everyone’s\u00a0genes are slightly different which explains why we look like our parents but are still unique. Genes can range in size depending on the species being looked at. For example, \u00a0Escherichia coli<\/em> has around 5,500 genes, a mouse has around 23,000, and humans contain around 25,000 genes. Eukaryotes, organisms with a nucleus inside their cells, contain coding and non-coding regions inside their genome. The coding-regions code for functional proteins while the non-coding regions do not. Most plant and animals genomes have a large portion of non-coding regions that are suspected to help regulate how genes are expressed[3]<\/sup>.<\/p>\n

\"geneinchromosome\"<\/p>\n

How does gene therapy work?<\/strong><\/p>\n

Gene therapy can be used to restore a mutation or replace a defective gene with a healthier one. \u00a0In order to place a gene inside of a cell, a vector is needed. The vector, usually a virus, is used to deliver the gene to a designated spot. The vector is genetically engineered so that it will not cause disease when it is inside a human cell. As soon as the DNA sequence is dropped off, it will start producing proteins that restore the cell to its normal function. \u00a0It is rare but a vector can sometimes insert the DNA into the wrong place. If inserted into a gene that regulates when a gene is turned on or off, it can possibly lead to cancer[1]<\/sup>.<\/p>\n

Some methods of insertion include injection, intravenously into a specific tissue, or it can be done in a laboratory. \u00a0A patient’s cells are removed and then introduced to the vector so that the engineered virus will never be in the human body. The cells that now carry the vector and the healthy gene are returned to the patient[4]<\/sup>. Gene therapy is still being tested in clinical trials but there has been success in human<\/a>s. The first successful \u00a0gene therapy trial treated a four year old child who was prone to repeated infections. In 1990, researchers at the National Institutes of Health helped treat this rare genetic disease called adenosine deaminase deficiency[1]<\/sup>.<\/p>\n

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This graph shows the steps in gene therapy<\/p><\/div>\n

What diseases can be treated using gene therapy?<\/strong><\/p>\n

Some of the diseases that have been used in gene therapy human clinical trials include severe combined immunodeficiency diseases, cystic fibrosis, and Cabavan’s disease. Other diseases like cancer, AIDS, Alzheimer’s diseases, \u00a0and cardiovascular diseases are possible candidates for gene therapy. There are about 4,000 diseases have have been traced to gene disorders. [1]<\/sup><\/p>\n

What else can gene therapy do?<\/strong><\/p>\n

According to the American Medical Association, gene therapy can also:<\/p>\n