Sickle Cell Anemia: Genetics, Symptoms, and Treatments

Sickle cell anemia (SCA) is a genetic disorder that affects millions of people and is caused by a specific mutation in the HBB gene. It is characterized by the production of abnormally shaped red blood cells, leading to reduced oxygen transport, painful crises, strokes, and various long-term complications. SCA is inherited as a recessive mutation in the HBB gene, which alters the hemoglobin protein. Current treatments, including blood transfusions, hydroxyurea, and bone marrow or stem cell transplants, primarily address symptoms and complications rather than curing the disease. However, recent advancements in gene therapy, particularly the use of CRISPR-Cas9 technology, offer promising avenues for potentially correcting the genetic defect underlying SCA. I will also research how CRISPR can offer new ways to address the problem of the disease and benefit patients. This review explores the genetic basis of SCA, current treatment strategies, and the revolutionary potential of CRISPR-based gene therapies in providing long-term solutions or even a cure for the disease. Despite challenges, these innovations offer hope for the future of SCA treatment, with the potential for broader accessibility and improved patient outcomes.