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2017 Fiscal Year Final Research Report

Elucidation of the significance of MAPT polymorphism associated with sporadic tauopathies by using genome editing method.

Research Project

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Project/Area Number 16K15479
Research Category

Grant-in-Aid for Challenging Exploratory Research

Allocation TypeMulti-year Fund
Research Field Neurology
Research InstitutionNiigata University

Principal Investigator

Onodera Osamu  新潟大学, 脳研究所, 教授 (20303167)

Project Period (FY) 2016-04-01 – 2018-03-31
Keywordsゲノム編集 / タウオパチー
Outline of Final Research Achievements

MAPT gene polymorphism is known in diseases that allow accumulation of tau, but it was difficult to analyze the significance of the polymorphism. By using the CORRECT method applying CRISPR-Cas 9 method, we introduce disease-related MAPT polymorphisms and investigate the expression level of tau gene and its effect on splicing. The introduction rate of genome editing improves 1.0% by standard method up to 4.5%. However, the efficiency was still low, and it was revealed that there is a possibility that it may be markedly different depending on genes. When splicing of MAPT was examined using the edited cells, it was confirmed that the ratio of 4R/3Rtau was increased. This cell line can be applied to drug screening to improve the ratio.

Free Research Field

神経内科

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Published: 2019-03-29  

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