Sickle Cell Disease: From Genetics to Curative Approaches

Abstract excerpt

Sickle cell disease (SCD) is a monogenic blood disease caused by a point mutation in the gene coding for β-globin. The abnormal hemoglobin [sickle hemoglobin (HbS)] polymerizes under low-oxygen conditions and causes red blood cells to sickle. The clinical presentation varies from very severe (with acute pain, chronic pain, and early mortality) to normal (few complications and a normal life span). The variability...

Topics

Share this publication in a Topic to start or enrich a Post.

• Share to CRISPR and Genetic Engineering
• Share to Hemoglobinopathies and Related Disorders
• Share to Iron Metabolism and Disorders