Identification of novel compounds that increase SMN protein levels using an improved SMN2 reporter cell assay.
Authors:Cherry JJ, Evans MC, Ni J, Cuny GD, Glicksman MA, Androphy EJ
Journal:J Biomol Screen
PubMed ID:22233647
Spinal muscular atrophy (SMA) is a neurodegenerative disorder that is characterized by progressive loss of motor neuron function. It is caused by the homozygous loss of the SMN1 (survival of motor neuron 1) gene and a decrease in full-length SMN protein. SMN2 is a nearly identical homolog of SMN1 that, ... More