Develpment of a new method to treat neuromuscular diseases

2017 Fiscal Year Final Research Report

• Project/Area Number: 16K14575
• Research Category: Grant-in-Aid for Challenging Exploratory Research
• Allocation Type: Multi-year Fund
• Research Field: Neurochemistry/Neuropharmacology
• Research Institution: The University of Tokyo
• Principal Investigator: Tezuka Tohru 東京大学, 医科学研究所, 助教 (50312319)
• Project Period (FY): 2016-04-01 – 2018-03-31
• Keywords: 神経筋接合部

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.

Free Research Field

分子生物学