• Defining the therapeutic window in spinal muscular atrophy : 

    Robbins, Kate Lynn (University of Missouri--Columbia, 2013)
    Spinal muscular atrophy (SMA) is caused by the loss of a single gene, survival motor neuron-1 (SMN1), which results in the rapid deterioration of motor neuron integrity and function, most often leading to infantile death. ...
  • 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 ...