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

48 引文 (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Yes

指紋

Spinal Muscular Atrophy
Valproic Acid
Motor Neurons
Therapeutics
Spinal Muscular Atrophies of Childhood
Foot Deformities
Muscular Atrophy
Action Potentials
Ear
Tail
Muscles
Proteins

ASJC Scopus subject areas

  • Neurology

引用此文

Establishing a standardized therapeutic testing protocol for spinal muscular atrophy. / Tsai, Li Kai; Tsai, Ming Shung; Lin, Tzer Bin; Hwu, Wuh Liang; Li, Hung.

於: Neurobiology of Disease, 卷 24, 編號 2, 11.2006, p. 286-295.

研究成果: 雜誌貢獻文章

Tsai, Li Kai ; Tsai, Ming Shung ; Lin, Tzer Bin ; Hwu, Wuh Liang ; Li, Hung. / Establishing a standardized therapeutic testing protocol for spinal muscular atrophy. 於: Neurobiology of Disease. 2006 ; 卷 24, 編號 2. 頁 286-295.
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