Salmeterol enhances the cardiac response to gene therapy in Pompe disease
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- 作者：Sang-oh Han ; Songtao Li ; Dwight D. Koeberl
- 关键词：Pompe disease ; Glycogen storage disease ; Adeno-associated virus vector ; Mannose-6-phosphate receptor ; Enzyme replacement therapy
- 刊名：Molecular Genetics and Metabolism
- 出版年：2016
- 出版时间：May 2016
- 年：2016
- 卷：118
- 期：1
- 页码：35-40
- 全文大小：982 K
文摘
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GAA knockout mice for treated with 4 new drugs in combination with an adeno-associated virus (AAV) vector encoding GAA
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Heart GAA activity was increased by either salmeterol (p < 0.01) or DHEA (p < 0.05), in comparison with untreated mice
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Glycogen content was reduced in the heart by adding DHEA (p < 0.001), salmeterol (p < 0.05), formoterol (p < 0.01), or clenbuterol (p < 0.01) to the AAV vector, in comparison with untreated mice
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Wirehang testing revealed that salmeterol and the AAV vector significantly increased performance in comparison with the vector alone (p < 0.001)
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The most effective individual drugs had no significant effect in absence of vector, in comparison with untreated mice