• Examine the role of minor splicing pathway in spinal muscular atrophy 

    Yen, Pei-Fen (University of Missouri--Columbia, 2014)
    Spinal Muscular Atrophy (SMA) is an autosomal recessive disorder mainly caused by deletions or mutations of one gene, Survival Motor Neuron (SMN). SMN is crucial in splicing processes for proper gene expression. Previous ...
  • Spinal muscular atrophy and the therapeutic application of trans-splicing RNA 

    Coady, Tristan H., 1980- (University of Missouri--Columbia, 2009)
    Development of RNA modalities to re-direct pathogenic pre-mRNA splicing events is the key to curing monogenic diseases. Improving the efficiency of these molecules in vivo is critical as they move towards clinical applications. ...