Spinal muscular atrophy: Mechanisms & treatment strategies.

Project Narrative Spinal muscular atrophy (SMA) is a frequently fatal motor neuron disorder caused by low SMN protein, and although raising SMN levels appears to benefit patients, very little is known about how the protein protects the motor neurons or if restricted delivery of currently available SMA drugs to these cells suffices as a cure. Here we wish to investigate these vitally important questions. Our research will result in a better understanding of the biology of SMA and is thus expected to inform newer treatments for this and similar motor neuron disorders.