CRISPR, ZFNS, and TALENS for Hemoglobinopathies an Analysis

Abstract

Abstract: Sickle Cell Disease is one of the most common genetic disorders in the United States and is incredibly prevalent throughout Africa and the Middle East. By 2050, the annual number of newborns with Sickle Cell Disease is projected to increase by 33%. A similar story can be told about Beta-Thalassemia: another hemoglobinopathy that has no standard treatment. The future of treating hemoglobinopathies looks bleak and more research must be done to prevent fatalities and the lifelong problems associated with it now. Sickle Cell Disease and Beta Thalassemia have one defining similarity: they are both monogenic disorders. This unique characteristic of having a single gene variation allows them to be the ideal candidate for one of the newest breakthroughs in biotechnology: target genome editing. As of now, there are three major competitors in the field, Zinc Finger Nuclease (ZFN), Transcription Activator-like Effector Nucleases (TALENs), and Clustered Regularly Interspaced Short Palindromic Repeat (CRISPR) target genome editing. Here I summarize the possibilities target genome editing provides in terms of research and a potential treatment for both Sickle Cell disease and Beta Thalassemia with a focus on comparing the three target genome editing technologies.