This protocol will be investigating the use of stem cell transplantation, in related donors,
to cure sickle cell disease. Sickle cell disease is a recessive disorder caused by a point
mutation that results in the substitution of valine for glutamic acid at the sixth position
in the B-chain of hemoglobin. This leads to sickling of the red blood cells under many
conditions, such as hypoxia, dehydration, and hyperthermia. The sickling leads to
vaso-occlusion, which causes irreversible damage in almost all systems in the body, including
the central nervous system (CNS), lungs, heart, bones, eyes, liver, and kidneys.
Phase:
Phase 3
Details
Lead Sponsor:
Hackensack Meridian Health Hackensack University Medical Center