Sickle-cell disease

A condition that affects many people of African descent is finally meeting its therapeutic match.

As multiple genetic strategies advance through the clinic, important safety questions remain to be answered.

Early trials show promise, but the challenge will be reaching those most in need.

Despite the continent being the epicentre of sickle-cell disease, too few Africans are included in genome-wide association studies.

Programmes to test newborn babies for sickle-cell disease can improve chances of survival, but they lack financial help.

A growing number of promising treatments is set to bring hope to people living with the disease.

Pain in sickle-cell disease is poorly understood — and patients face both medical and socioeconomic problems when seeking delivery from discomfort.

The United States has reduced child mortality from this condition — but comprehensive care for adults has not kept pace.

Clinical haematologist Adlette Inati explains how the disease arrived in the region, what affects its spread and how to control it.

Optimized conditions for ribonucleoprotein delivery of Cas9–sgRNA complexes enables precise and efficient gene editing to restore fetal hemoglobin expression in sickle cell disease patient-derived HSCs

Our data offer a proof of concept for the treatment of SCD by MHC disparate non-myeloablative T cell-depleted HSCT, using anti-3rd party central memory veto CD8-positive T-cells.

Sickle cell disease includes genetic conditions that are caused by mutations in one of the genes encoding haemoglobin. Mutant haemoglobin molecules can polymerize, causing the red blood cells to acquire a characteristic crescent shape that gives the disease its name.

A single therapeutic base edit of the BCL11A enhancer in human HSPCs can ameliorate cellular defects associated with sickle cell disease and β-thalassemia in vitro and efficiently induce fetal hemoglobin expression upon engraftment in mice in vivo.

A custom adenine base editor can edit the variant of the β-globin gene that causes sickle cell disease into a non-pathogenic variant in human and mouse cells, and transplantation of the edited cells rescues sickle cell disease in mice.