Inês Martins, PhD, managing science editor —

Treatment with blood stem cells genetically engineered to block interleukin-1 beta (IL-1b) signaling may be a promising approach for reducing brain inflammation and cognitive decline in Sanfilippo syndrome type A, a study in mice reports. The study, “Haematopoietic stem cell gene therapy with IL…

An abnormal accumulation of heparan sulfates — a hallmark of Sanfilippo syndrome type A — in the retina causes cell death and induces eye symptoms by impairing a process called autophagy, which cells use to clear out unwanted components, a study of mice has found. Notably, eye symptoms developed…