Sickle cell anemia. Pathophysiology and treatment

Area of Science:

• Hematology
• Molecular Biology
• Medical Science

Background:

• Sickle cell disease (SCD) is a genetic blood disorder characterized by abnormal hemoglobin.
• The pathophysiology involves the sickling of red blood cells, leading to vaso-occlusion and organ damage.

Purpose of the Study:

• To review current understanding of sickle cell pathophysiology.
• To describe emerging therapeutic strategies for SCD.

Main Methods:

• Literature review of current concepts in sickle cell pathophysiology.
• Description of novel treatment modalities.

Main Results:

• Current understanding of the sickling process is detailed.
• New treatments including induced hyponatremia, membrane-active agents, and gene manipulation are presented.

Conclusions:

• Advances in understanding SCD pathophysiology are paving the way for innovative treatments.
• Emerging therapies hold promise for improving outcomes in patients with sickle cell disease.