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Modelling Duchenne Muscular Dystrophy in MYOD1-converted Urine-Derived Cells Treated With 3-deazaneplanocin A Hydrochloride

Modelling Duchenne Muscular Dystrophy in MYOD1-converted Urine-Derived Cells Treated With 3-deazaneplanocin A Hydrochloride

Hotake Takizawa, Yuko Hara, Yoshitaka Mizobe, Taisuke Ohno, Sadafumi Suzuki, Ken Inoue, Eri Takeshita, Yuko Shimizu-Motohashi, Akihiko Ishiyama, Mikio Hoshino, Hirofumi Komaki, Shin'ichi Takeda, Yoshitsugu Aoki

Sci Rep. 2019 Mar 7;9(1):3807.

PMID: 30846748

Abstract:

Duchenne muscular dystrophy (DMD) is a severe muscle disorder characterised by mutations in the DMD gene. Recently, we have completed a phase I study in Japan based on systemic administration of the morpholino antisense that is amenable to exon-53 skipping, successfully. However, to achieve the effective treatment of DMD, in vitro assays on patient muscle cells to screen drugs and patient eligibility before clinical trials are indispensable. Here, we report a novel MYOD1-converted, urine-derived cells (UDCs) as a novel DMD muscle cell model. We discovered that 3-deazaneplanocin A hydrochloride, a histone methyltransferase inhibitor, could significantly promote MYOGENIN expression and myotube differentiation. We also demonstrated that our system, based on UDCs from DMD patients, could be used successfully to evaluate exon-skipping drugs targeting DMD exons including 44, 50, 51, and 55. This new autologous UDC-based disease modelling could lead to the application of precision medicine for various muscle diseases.

Chemicals Related in the Paper:

Catalog Number	Product Name	Structure	CAS Number	Price
AP120964456	3-Deazaneplanocin A hydrochloride		120964-45-6	Price

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