Development of gene and cell therapy for muscular dystrophies.

2014 Fiscal Year Final Research Report

• Project/Area Number: 24591284
• Research Category: Grant-in-Aid for Scientific Research (C)
• Allocation Type: Multi-year Fund
• Section: 一般
• Research Field: Neurology
• Research Institution: National Center of Neurology and Psychiatry
• Principal Investigator: KIMURA En 独立行政法人国立精神・神経医療研究センター, トランスレーショナルメディカルセンター, 室長 (60433025)
• Project Period (FY): 2012-04-01 – 2015-03-31
• Keywords: 筋ジストロフィー / 遺伝子治療 / レンチウイルスベクター / 幹細胞移植

Outline of Final Research Achievements

Series of Lentiviral vectors were prepared; each carrying different-type of full- and short-version of dystrophin, marker gene, myogenic transcriptional factors, under the control of various combinations of muscle specific promoters and the miR target sequences. The myoD-ER(T), fusion gene with myoD and modified estrogen receptor biding domain for Tamoxifen, was successfully introduced into dermal fibroblasts by lentiviral transduction, and covert themselves to myogenic cells. These cell therapy resources are candidates of genetic modifications by not only viral vectors based technologies and newly developed gene-editing technologies.

Free Research Field

遺伝子/幹細胞治療