Gene therapy for sickle cell disease
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...