National Changhua University of Education Institutional Repository : Item 987654321/11515
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題名: Treatment of Spinal Muscular Atrophy by Sodium Butyrate
作者: Chang, Jan-Gowth;Hsieh-Li, Hsiu-Mei;Jong, Yuh-Jyh;Wang, Nancy M.;Tsai, Chang-Hai;Li, Hung
貢獻者: 生物技術研究所
日期: 2001-08
上傳時間: 2012-06-06T02:04:40Z
出版者: the National Academy of Sciences
摘要: Spinal muscular atrophy (SMA) is an autosomal recessive disease characterized by degeneration of the anterior horn cells of the spinal cord, leading to muscular paralysis with muscular atrophy. No effective treatment of this disorder is presently available. Studies of the correlation between disease severity and the amount of survival motor neuron (SMN) protein have shown an inverse relationship. We report that sodium butyrate effectively increases the amount of exon 7-containing SMN protein in SMA lymphoid cell lines by changing the alternative splicing pattern of exon 7 in the SMN2 gene. In vivo, sodium butyrate treatment of SMA-like mice resulted in increased expression of SMN protein in motor neurons of the spinal cord and resulted in significant improvement of SMA clinical symptoms. Oral administration of sodium butyrate to intercrosses of heterozygous pregnant knockout-transgenic SMA-like mice decreased the birth rate of severe types of SMA-like mice, and SMA symptoms were ameliorated for all three types of SMA-like mice. These results suggest that sodium butyrate may be an effective drug for the treatment of human SMA patients.
關聯: Proc Natl Acad Sci., 98(17): 9808-9813
顯示於類別:[生物技術研究所] 期刊論文

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