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2015 Fiscal Year Final Research Report

Use of a human artificial chromosome for delivering trophic factors

Research Project

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Project/Area Number 25461315
Research Category

Grant-in-Aid for Scientific Research (C)

Allocation TypeMulti-year Fund
Section一般
Research Field Neurology
Research InstitutionTottori University

Principal Investigator

Watanabe Yasuhiro  鳥取大学, 医学部, 講師 (20335540)

Project Period (FY) 2013-04-01 – 2016-03-31
Keywords筋萎縮性側索硬化症 / 人工染色体 / 神経栄養因子 / 細胞移植
Outline of Final Research Achievements

A human artificial chromosome (HAC) can transfer multiple and/or large transgenes along with their regulatory elements thereby resembling native chromosomes. Using this HAC system, we established mesenchymal stem cells (MSCs) that simultaneously expressed hepatocyte growth factor, glial cell line-derived neurotrophic factor and insulin-like growth factor 1, termed HAC-MSCs. We then introduced the cells for the treatment of a neurodegenerative disorder, amyotrophic lateral sclerosis (ALS). The HAC-MSCs were transplanted via the fourth cerebral ventricle (CV) or intravenous (IV) infusion at various ages of recipient mice. We confirmed a statistically significant increase in lifespan via CV transplantation at 100 days compared to the controls. This effect was not seen in mice transplanted with MSCs lacking the HAC. We successfully enhanced the trophic potential of the MSCs using the HAC. This strategy could be a promising direction for the treatment of neurodegenerative disorders.

Free Research Field

臨床神経内科学

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Published: 2017-05-10  

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