Transplantation of gene-corrected motor neurons as a therapeutic strategy for spinal muscular atrophy

Mol Ther. 2013 Mar;21(3):502-3. doi: 10.1038/mt.2013.23.
No abstract available

MeSH terms

  • Animals
  • Disease Models, Animal
  • Genetic Therapy / methods
  • Humans
  • Mice
  • Motor Neurons / cytology
  • Motor Neurons / transplantation*
  • Muscular Atrophy, Spinal / genetics*
  • Muscular Atrophy, Spinal / therapy*
  • Pluripotent Stem Cells / cytology*
  • Survival of Motor Neuron 2 Protein / genetics

Substances

  • SMN2 protein, human
  • Survival of Motor Neuron 2 Protein