Gene therapy for sickle cell disease
globin gene variant transferred to hematopoietic stem cells can correct sickle cell anemia.
Dec 17, 2001
Sickle cell disease (SCD) is caused by the homozygous inheritance of a single point mutation in the β globin gene (hemoglobin S) that manifests itself as an anemia with characteristic sickle-shaped red blood cells. Currently available treatments are only symptomatic, but in December 14
Pawliuk
"Before gene therapy of SCD may be proposed for human patients...