NIH R01 · 2024
Clonal hematopoiesis and inherited genetic variation in sickle cell disease
Project Summary Sickle cell disease (SCD) is associated with chronic hemolysis, systemic endothelial dysfunction, inflammation and vascular occlusion. This complex pathophysiology leads to severe pain, progressive multi-organ damage and premature death with a median lifespan of 48 years in high- income countries. We and others have determined that young adults with progressive heart, lung, and kidney damage, either individually or in combination, are at particularly high risk for premature death. Many individuals with SCD are candidates for high-risk treatments that can potentially eliminate symptoms and arrest organ damage, including allogeneic hematopoietic stem cell (HSC) transplantation…
From the public funding record at NIH RePORTER. Describes the funded project, not the reviews below.