Clinical Trials and Research

AbstractBackground: Sickle cell anemia (SCA) is a genetic disease marked by abnormal hemoglobin S and sickle-shaped red blood cells. It is highly prevalent in sub-…

AbstractBackgroundSickle cell disease (SCD) is an inherited blood disorder characterized by the presence of sickle-shaped erythrocytes, leading to haemolytic anemia…

Gene therapies for sickle cell disease are already changing lives in high-income countries, but in places like Uganda—where the burden of disease is among the…

Sickle cell anemia is a severe inherited hemoglobinopathy with a large global burden, particularly in sub-Saharan Africa.1 Hydroxyurea, which is standard therapy…

AbstractObjectivesSickle Cell Disease (SCD), characterized by persistently low hemoglobin (Hb) and hematocrit levels, is among the leading causes of under-five…

AbstractSickle cell disease (SCD) is characterized by recurrent vaso-occlusion, hemolysis, and chronic inflammation, leading to acute painful crises and progressive…

Haifa AlKhodier, Fahad Bin Saleh, Abdulmajeed AlRumi, Tariq AlOjaymDepartment of Dentistry, King Faisal Specialist Hospital and Research Centre, Riyadh, Saudi…

Study OverviewBrief SummaryThis study is not a treatment protocol and no experimental treatments are involved. Study participants may be seen as needed for clinical…

AbstractSickle cell disease (SCD) is caused by a point mutation in the β-globin gene that promotes hemoglobin polymerization, leading to chronic hemolytic anemia,…

HighlightsWhat are the main findings?Gene therapy for sickle cell disease has progressed from lentiviral gene addition strategies to precise genome editing…