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There is an urgent need for health policymakers to pay attention to recent developments in gene therapy for sickle cell disease (SCD), a treatment approach directed principally at treating the cause rather than the symptoms of genetic diseases. Sickle cell anaemia (SCA), one of the variants of SCD, is an inherited disease in which mutated red blood cells are curved into sickle (crescent) shapes, resulting in poor oxygenation of cells. It occurs globally, with more than 230 000 infants born with the condition in Africa annually. Patients with SCA suffer from recurrent anaemia, infections, tissue damage, strokes, excruciating pain and fatal organ failures, with vaso-occlusive crises being the hallmark of its acute presentation. Sadly, 50–80% of infants born with the disease die before the age of 5 years in Africa. The financial impact is devastating, especially for rural households in SubSaharan Africa – where 60% of the population live – with very limited access to even routine health care.

Gene therapy renews hope to lower the global rural sickle cell disease burden