2010 Fiscal Year Final Research Report
Protei n anchoring-therapy indefectof extra cellar matrix at skeIeta muscle
| Project/Area Number |
21700443
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| Research Category |
Grant-in-Aid for Young Scientists (B)
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| Allocation Type | Single-year Grants |
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| Research Field |
Neurophysiology and muscle physiology
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| Research Institution | Nagoya University |
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Principal Investigator |
ITO Mikako 名古屋大学, 大学院・医学系研究科, 助教 (60444402)
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| Project Period (FY) |
2009 – 2010
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| Keywords | 先天性筋無力症 / 遺伝子治療 / collagen Q / アセチルコリンエステラーゼ欠損症 / 分子欠損症 |
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| Research Abstract |
Congenital defects of ColQ cause endplate AChE deficiency and myasthenic syndrome. The intravenous administration of adeno-associated virus COLQto Colq-/-mice recovers motor functions, synaptic transmission, as well as the morphology of the NMJ. ColQ-tailed AChE is specifically anchored to NMJ. Furthermore, the in vivo injection of recombinant ColQ-tailed AChE protein complex into the gluteus maximus muscle of Colq-l- mice led to accumulation of AChE in non-injected forelimbs. We demonstrated for the first time in vivo that the ColQ protein contains a tissue-targeting signal that is sufficient for anchoring itself to the NMJ.
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