Establishing a standardized therapeutic testing protocol for spinal muscular atrophy

Li Kai Tsai, Ming Shung Tsai, Tzer Bin Lin, Wuh Liang Hwu, Hung Li

Research output: Contribution to journalArticlepeer-review

52 Citations (Scopus)


Several mice models have been created for spinal muscular atrophy (SMA); however, there is still no standard preclinical testing system for the disease. We previously generated type III-specific SMA model mice, which might be suitable for use as a preclinical therapeutic testing system for SMA. To establish such a system and test its applicability, we first created a testing protocol and then applied it as a means to investigate the use of valproic acid (VPA) as a possible treatment for SMA. These SMA mice revealed tail/ear/foot deformity, muscle atrophy, poorer motor performances, smaller compound muscle action potential and lower spinal motoneuron density at the age of 9 to 12 months in comparison with age-matched wild-type littermate mice. In addition, VPA attenuates motoneuron death, increases spinal SMN protein level and partially normalizes motor function in SMA mice. These results suggest that the testing protocol developed here is well suited for use as a standardized preclinical therapeutic testing system for SMA.

Original languageEnglish
Pages (from-to)286-295
Number of pages10
JournalNeurobiology of Disease
Issue number2
Publication statusPublished - Nov 2006
Externally publishedYes


  • Animal model
  • Motoneuron disease
  • Neuronal degeneration
  • Phenotype
  • Spinal cord
  • Spinal muscular atrophy
  • Valproic acid

ASJC Scopus subject areas

  • Neurology


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