The Implications of Sickle Cell Disease and CRISPR Cas-9

Abstract

This paper investigates the use of CRISPR technology to treat Sickle Cell diseases, diving into the specifics of the disease and the mechanism, as well as the complications and controversy that arise. Many people around the world suffer from a variety of genetic disorders, one in particular being Sickle Cell disease. The people who suffer from this genetic disorder face different levels of severity and symptoms, suffering from anemia and pain (due to the distorted shape of their blood cells), and the disease can be inherited through different mechanisms (Rees et al.).

Without a doubt, our future depends on our ability to utilize and implement effective treatments and therapies to reduce and diminish the existence of genetic disorders, including Sickle Cell disease. Most notably, gene therapy for the treatment of genetic diseases/disorders has the potential to serve as an excellent tool to treat diseases that currently have no treatment or cure, all encoded by our highly cryptic, highly specialized DNA. If we delve into our research and developments, we could have the possibility to manipulate genetic variability, as well as create facilitated life circumstances and new discoveries that correspond with the human genome (Sharma et al.). 

Sickle cell disease (SCD), is caused by a mutation in the hemoglobin protein, and inhibits  red blood cells from effectively delivering oxygen to other parts of the body, resulting in fatigue and weakness throughout the day (Elendu et al.). Given the severity of this disease, CRISPR Cas-9 is needed to address the root cause of the disease, and it must target the HBB gene mutation.

Ultimately, Genetic disorders, repair mechanisms, and clinical trials all act together as a dynamic system, allowing us to expand and advance our knowledge of gene editing and therapies.