The role of long non-coding RNAs in spinal muscular atrophy. KU Leuven
Spinal muscular atrophy (SMA) is the second leading cause of infantile death after cystic fibrosis. SMA is an incurable autosomal recessive disease caused by a genetic defect in the SMN1 gene that encodes a protein necessary for survival of motor neurons. SMA manifests in various degrees of clinical severity dependent on the copy number of the highly related SMN2 gene. While the majority of the protein arisen from ...