Neuroscience

Getting gene therapy to the brain

A lone genetic mutation can cause a life-changing disorder with effects on multiple body systems. Lysosomal storage diseases, for example, of which there are dozens, arise due to single mutations that affect production of ...

Immunology

Scientists develop new mouse model of Hirschsprung's disease

About one in every 5,000 babies is born without enteric neurons in the distal colon resulting in Hirschsprung's disease. Because of the missing neurons, contents of the gut cannot pass normally, resulting in constipation ...