Many mutations in a person's genes result in severe medical condition. These mutations cause the protein encoded by that gene to malfunction and cells that rely on this protein cannot function properly. This can cause problems for the tissues or organs. Conditions related to gene mutations are called genetic disorders. One way to fix the internal problems is gene therapy. By adding a corrected copy of the gene, the affected cells, tissues, and organs may work properly. As opposed to drug-based approaches, gene therapy repairs the underlying genetic defect.
Gene Therapy is the process to treat or alleviate diseases by genetically modifying the cells of the infected person, causing the gene to function properly. When a human disease gene has been recognized, molecular genetics tools can be used to explore the process of the gene in both
normal and pathogenic states. From there, the gene is transferred either in vivo or ex vivo and the body begins to identify the new gene. Gene therapy has to be repeated several times for the infected patient to continually be relieved.
Currently, gene therapy is still being experimented with and products are not approved by the U.S. Food and Drug Administration. There have been several set backs in the last 15 years that have restricted further measures on gene therapy. As there are unsuccessful attempts, there continues to be a growing number of successful gene therapy transfers which has furthered the research.
Major diseases that can be treated with gene therapy: infectious diseases (result of infection by a virus or bacterial pathogen), cancers, inherited disorders, immune system disorders
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