Gene therapy

Correction of SCD by gene therapy requires efficient insertion of a gene into repopulating haemopoietic cells and regulated

Figure 7.7 Outcome after transplantation for 59 children with advanced, symptomatic sickle cell disease. Kaplan-Meier estimates for survival and event-free survival following marrow transplantation are shown. An event is defined as death, graft rejection or recurrence of sickle cell disease. A cumulative incidence curve for graft rejection and return of sickle cell disease is also depicted (from Walters etal., 2001, Biology of Blood and Marrow Transplantation 7: 665, with permission).

Figure 7.7 Outcome after transplantation for 59 children with advanced, symptomatic sickle cell disease. Kaplan-Meier estimates for survival and event-free survival following marrow transplantation are shown. An event is defined as death, graft rejection or recurrence of sickle cell disease. A cumulative incidence curve for graft rejection and return of sickle cell disease is also depicted (from Walters etal., 2001, Biology of Blood and Marrow Transplantation 7: 665, with permission).

expression in erythropoietic lineage. An anti-sickling haemoglobin, constituting 20-30% of the total haemoglobin, would be enough to produce clinical response. Mouse models of sickle cell disease have considerably helped in the effort to develop gene therapy, and correction of sickling phenotype has been demonstrated in such animals.

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