BioCentury
ARTICLE | Targets & Mechanisms

Hedgehog battles Down syndrome

September 26, 2013 7:00 AM UTC

Researchers at The Johns Hopkins University School of Medicine have proposed that mental retardation caused by Down syndrome could be prevented or mitigated after birth with a small molecule activator of the sonic hedgehog homolog pathway.1 The group's mouse data show improvements in brain development, learning and memory. However, the narrow therapeutic window and the long gap in time between treatment and evaluation of cognitive endpoints in patients will pose significant translational challenges.

Down syndrome arises from an extra copy of chromosome 21, leading to multiple craniofacial and neurological abnormalities. Compared with brains from healthy controls, the brains of patients with Down syndrome have smaller cerebellums and hippocampuses-regions that control motor activity and memory, respectively...