Genetic therapies for sickle cell disease

After decades with few novel therapeutic options for sickle cell disease (SCD), autologous hematopoietic stem cell (HSC) based genetic therapies including lentiviral gene therapy (GT) and genome editing (GE) now appear imminent. Lentiviral GT has advanced considerably in the past decade with promising clinical trial results in multiple disorders. For β-hemoglobinopathies, GT strategies of gene addition and fetal hemoglobin (HbF) induction through BCL11A regulation are both being evaluated in open clinical trials.
Source: Seminars in Hematology - Category: Hematology Authors: Tags: 55/3 Sickle Cell Disease Source Type: research