Spinal muscular atrophy (SMA) is a devastating childhood disease caused by loss of function of the survival motor neuron (SMN) gene, leading to the death of nerve and muscle cells. The first SMN gene replacement therapies have recently been approved…  Continue reading →

In most animal species including humans, commissural axons connect neurons on the left and right side of the nervous system. This communication between the two sides of the brain and spinal cord is necessary for a series of complex function,…  Continue reading →

Our laboratory has been working for several years on the role of non-cell autonomous homeoprotein transcription factors in regulating cerebral cortex physiology. We have shown that in mice the OTX2 homeoprotein is expressed in the choroid plexus, secreted into cerebrospinal…  Continue reading →

Neurobiological research on alcohol addiction had grown, but no mechanistically novel medications have been approved in more than 15 years. Promising candidates have failed in development. What have we been missing? Three themes have emerged from our attempts to address…  Continue reading →