Therapeutic strategies for sickle cell disease: towards a multi-agent approach
Therapeutic strategies for sickle cell disease: towards a multi-agent approach, Published online: 04 December 2018; doi:10.1038/s41573-018-0003-2
Although the molecular basis for sickle cell disease (SCD) has been known for decades, progress in developing therapies that ameliorate the complex systemic manifestations of SCD has been slow. This article reviews recent advances that are providing the basis for therapies that could improve outcomes by targeting different aspects of SCD pathophysiology and highlights the opportunity for combination strategies. The potential for curative gene therapy is also discussed.