Establishing a standardized therapeutic testing protocol for spinal muscular atrophy

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

研究成果: 雜誌貢獻文章同行評審

50 引文 斯高帕斯(Scopus)

摘要

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.

原文英語
頁(從 - 到)286-295
頁數10
期刊Neurobiology of Disease
24
發行號2
DOIs
出版狀態已發佈 - 十一月 2006
對外發佈

ASJC Scopus subject areas

  • 神經內科

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