Health Check

Gene therapy for sickle cell disease

Informações:

Sinopsis

Are genetic therapies for sickle cell disease beginning to come of age? Claudia Hammond talks to David Williams and Erica Esrick of Boston Children’s Hospital about their promising results with a gene therapy for the disease in a pilot trial involving six young patients. Their report appears in the latest edition of the New England Journal of Medicine alongside encouraging results of a CRISPR gene editing therapy for sickle cell disease. Both approaches target the same gene – the result of which is to make bone marrow cells to produce foetal haemoglobin to compensate for people’s faulty adult haemoglobin. BBC Global health correspondent Naomi Grimley has a coronavirus global round up for us, and we report on the discovery of a pair of salivary glands new to medical science – the first new set of organs to be discovered for centuries. Dutch researchers detected them with a sophisticated form of body scanning, hiding where the back of the nasal cavity meets the top of the throat. It’s an anatomical revelation