- Anemia is a lack of hemoglobin in blood. Having anemia can
makes feel tired and weak.
- There are many forms of anemia each with aits own cause ,
sickle cell anemia is one of them.
- Sickle cell disease is a hemoglobinopathy which affects
approximately 100,000 individual in United States.
- It is caused by point mutation in
- globin gene resulting in synthesis of pathological hemoglobin
- Pathophysiological studies shown dense dehydrated red cell play
a significant role in acute and chronic clinical manifestations of
- This is due to modulation of innate immunity and increased iNKT
lymphocytes, increase level of vascular active cytokine such as
endothelium combined with the final contribution of platelet.
- Hydroxyurea is the gold standard treatment for SCD.
- Various option for treatment are :
- Molecule targeting hemolysis induced vasculopathy.
- Agents that modulate the abnormal tone.
- Agents interfering with red cellvascular adhesion event.
- Molecules interfering with the physical properties of the red
cell endothelial adhesion process. Molecules specifically
interfering with sickle cell endothelial adhesive mechanism.
Molecule affecting platelet function. These are still under
- N- acetyl cysteine an exogenous thiol donor.
In the last two decades, progress in hemopoietic stem cell
transplantation strategies have allowed to offer a new curative
Development of CRISPR/Cas9 genome editing has been reported to
represent a new potential therapeutic tool.
A new field of combination therapy require a holistic approach
considering the improvement of patient quality of life.
( Matte A et.al.2018.New therapeutic options fro the treatment
of sickle cell disease. MJHID;11:1-12)