Exploring Gene Therapy Paper - Gene therapy is an experimental technique that uses genes to treat or prevent disease In the future this technique may

Exploring Gene Therapy Paper - Gene therapy is an...

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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. There are multiple types of methods researchers are looking into to accomplish this task. These methods include replacing a mutated gene that causes disease with a healthy copy of the gene, inactivating a mutated gene that is functioning improperly, or introducing a new gene into the body to h elp fight a disease. When evaluating a potential gene therapy disease, scientists look for five conditions in the candidate. Firstly, whether the condition results from mutations in one or more genes. Secondly, which specific genes are involved. Thirdly, what is known about the biology of the disorder, and whether adding a normal copy of the gene will fix the problem in the affected tissue. Lastly, whether it is possible to deliver the gene to cells of the affected tissue. Hallmarks of a successful gene delivery in a candidate include targeting the right cells, activating the gene, integrating the gene into the cells, and avoiding harmful side effects. An example of a candidate for gene therapy is Cystic Fibrosis, or CF. Cystic fibrosis is a single gene disorder viewed as a good candidate for gene therapy because the affected gene is known, and the target tissue, the lung, is accessible. The CFTR gene encodes a transmembrane protein that regulates chloride transport, but information regarding the pathology, usually complicated by bacterial issues, is murky. Retroviral

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