Gene-Editing Approaches Might Lead to New Treatment Methods for Sickle Cell Disease

Published Date : May 03, 2016

Among various hemoglobinopathy disorders, sickle cell anemia is the most common inherited blood disease. According to the World Health Organization (WHO), around 5% of the world’s population has genes for hemoglobinopathy disorders such as thalassaemia and sickle cell anemia. While sickle cell anemia is most common across Africa, various forms of thalassaemia are prevalent in the Middle East, the Mediterranean basin, and Asia. In sickle cell disease, half moon shape of red blood cells causes poor blood oxygen levels and blood vessel blockages. This leads to anemia among the patients. The rapid rise in number of people affected by the disease has led governments and regulatory authorities to initiate awareness programs decrease hemoglobinopathies-related mortality rate. For example, in 2010, the Centers for Disease Control and Prevention (CDC) started Thalassaemia Data Collection Project and Blood Safety Monitoring. All these factors are expected to positively impact the growth of the global hemoglobinopathies market.

Expensive Bone Marrow Transplantation has led to Exploration of New Treatment Alternatives

Genetic testing has become the most cost-effective diagnostics for diagnosis of diseases such as sickle cell anemia. In sickle cell disease, while parents unknowingly pass a copy of the genetic mutation for the disease to their progeny, unusual genetic mutations counteract the effects of sickle cell disease. Researchers are focussing on this aspect to save the lives of patients suffering from this inherited disease. Though not being a cure, this compensatory treatment holds potential to save around 300,000 infants every year who are born with sickle cell disease. Healthcare companies such as Novartis are working on gene-editing approaches to explore new sickle cell treatments. Currently, the only known cure for sickle cell disease is bone marrow transplantation that provides a new circulatory system. However, high cost and dearth of technical expertise has made it difficult for patients to afford the treatment