Identification of novel compounds that increase SMN protein levels using an improved SMN2 reporter cell assay.
AuthorsCherry JJ, Evans MC, Ni J, Cuny GD, Glicksman MA, Androphy EJ
JournalJ Biomol Screen
PubMed ID22233647
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