| Budget Amount *help |
¥3,640,000 (Direct Cost: ¥2,800,000、Indirect Cost: ¥840,000)
Fiscal Year 2017: ¥1,820,000 (Direct Cost: ¥1,400,000、Indirect Cost: ¥420,000)
Fiscal Year 2016: ¥1,820,000 (Direct Cost: ¥1,400,000、Indirect Cost: ¥420,000)
|
|---|
| Outline of Final Research Achievements |
The neuromuscular junction (NMJ) is a synapse between a motor neuron and skeletal muscle and is required for muscle contraction. Defects in NMJ formation are found in various neuromuscular diseases including myasthenia, muscle dystrophy, and motor neuron degenerative diseases, suggesting that enhancement of NMJ formation mitigates pathology of these diseases. In this study, treatment with an adeno-associated virus vector carrying the DOK7 gene, an essential gene for NMJ formation, was demonstrated to enhance the formation of NMJs, motor activity, and life span in a mouse model of amyotrophic lateral sclerosis (ALS), a progressive, multifactorial motor neuron degenerative disease.
|
