Activation of betaglobin through the use of transcriptor activator-like effectors to combat sickle cell anemia
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This research is particularly important to Sickle Cell Anemia, but also for other genetic diseases. As this is relatively new research, most of the research is more specific to the structure of TALEs and certain genes instead of actual TALEs involved in diseases. As mentioned before, the sickled cells in Sickle cell anemia occur because of mutations in the beta globin gene of the red blood cells. Our research is to upregulate or activate the beta globin gene in these cells. The reason for this is because we want the RNA of the beta globin gene. We need that RNA so that we can detect it with Molecular Beacons and find its exact location. In order to transcribe the beta globin DNA into RNA, we need to first activate or “turn on” the transcription of that gene within the cell. Once a gene is activated then we will be able to use the molecular beacons to detect the specific sequence that we want in the nucleic acid. By constructing the TALEs that can do this, we would also help further gene therapy developments of other disease models.