Disruption of the Survival Motor Neuron (SMN) gene in pigs using ssDNA

  title={Disruption of the Survival Motor Neuron (SMN) gene in pigs using ssDNA},
  author={Monique A. Lorson and Lee D. Spate and Melissa S. Samuel and Clifton Neal Murphy and Christian L. Lorson and Randall S Prather and Karen Wells},
  journal={Transgenic Research},
Spinal Muscular Atrophy (SMA) is an autosomal recessive neurodegenerative disease that is a result of a deletion or mutation of the SMN1 (Survival Motor Neuron) gene. A duplicated and nearly identical copy, SMN2, serves as a disease modifier as increasing SMN2 copy number decreases the severity of the disease. Currently many therapeutic approaches for SMA are being developed. Therapeutic strategies aim to modulate splicing of SMN2-derived transcripts, increase SMN2 gene expression, increase… CONTINUE READING