Considered one of the more controversial up comings in the world of science, it’s hard to ignore what amazing benefits gene therapy has to offer. With many potential treatments aimed to target some rather serious diseases, much support has been gained for the field. Although gene therapy remains at the forefront of change, much discussion has been created due to several potential drawbacks. These drawbacks, however substantial, must not be overlooked for the risk of lives isn’t a fair price to pay for innovation of healthcare reform.
Gene therapy is used as a way to treat or minimalize defective genes that lead to or cause diseases. Genes are hereditary units which are base sequences that provide the encoding instruction of a protein. Different proteins are made depending on what the specific amino acid sequence had encoded. Proteins can be different in size, shape, and function. Many times in the event of a mutation, genes become transformed so that the protein supposed to be encoded either isn’t produced or is made with inhibiting functionality, thus resulting in a potential genetic disorder. Therapy for this usually involves the implementation of new genes in cells to take the place of non-functioning or absent ones. This process involves a virus that is used to carry the genetic information to the patient’s cells. These viruses have been modified to find and detect specific problem target areas. To do this, scientists many times will use viral vectors, or vehicles that are used as the form of transportation for the virus.
Avoiding toxicity in the target cell is the elemental key in order for gene therapy to be successful. Certain viral vectors have proven to perform better than others with this. Retroviral vectors use retroviruses which are a type of virus that has its RNA genome transformed into DNA inside the diseased cell. Viral genes can be switched with transgenes by way of controlling and alternating the viral genome. The viral proteins are then made by the packaging cell which will contain the genome. The packaging cells are grown in a culture medium, which is a type of liquid or gel that is made to maintain the growth of microorganisms or cells. The main concern with this is that retroviral vectors are hardly reliable in their capability to infect cells that are non-dividing. However, many time if it is possible, the target cells are extracted and grown “in vitro,” or meaning “within glass” in Latin. These are known as test tube experiments where recombinant retroviral vectors are used a method to infect the target cell that has been isolated. (Vermia & Somia).
Gene therapy was first approved for clinical trials in the United States in May of 1989. Since the therapy has been in fairly experimental stages, every individual nation involved in the technology has specific approval sequences that must take place. These rules and regulation are put in place as a way to protect all parties involved, specifically patients. Within...