Substantial advance in the development of treatment for Marfan’s syndrome.

“Reinforcement of microfibril rescues microfibril disorder of Marfan’s syndrome”.

Donor organ transplantation is currently an essential therapeutic approach to the replacement of a dysfunctional organ as a result of disease, injury or aging in vivo. Recent progress in the area of regenerative therapy has the potential to lead to bioengineered mature organ replacement in the future. A research group led by Professor Takashi Tsuji (Professor in the Research Institute for Science and Technology, Tokyo University of Science, and Director of Organ Technologies Inc.) has provided a proof-of-concept for bioengineered mature organ replacement as a future regenerative therapy.

The research group, M. Saito et al., reports that A disintegrin-like metalloprotease domain with thrombospondin type I motifs like (ADAMTSL) 6β which is a microfibril-associated extracellular matrix protein plays an essential role in the rescuing microfibril disorder in an Marfan’s syndrome (MFS). MFS is a severe, systemic disorder of connective tissue formation and can lead to aortic aneurysms, ocular lens dislocation, emphysema, bone overgrowth and severe periodontal disease. MFS has an estimated prevalence of 1 in 5,000-10,000 individuals. In our current study, we report that ADAMSL6β is essential for the development and regeneration of the connective tissue including aorta and periodontal ligament (PDL). Using an animal model of MFS microfibril disorder, we demonstrate that ADAMSL6β expression can rescue microfibril formation through the promotion of microfibril assembly. This study thus represents a substantial advance and demonstrates the novel therapeutic approach to the treatment of MFS through the promotion of ADAMTSL6β-mediated microfibril assembly.

Dr. Saito is a research team member in “Health Labor Sciences Research Grant: Research on Research project on overcoming incurable disease (Domain Leader: Professor Shinya Murakami of Osaka University, Japan)”. This research was collaborated research with Professor Kiyotoshi Sekiguchi (Institute for Protein Research Osaka University, Japan), Professor Toshiyuki Yoneda (Department of Molecular and Cellular Biochemistry, Graduate School of Dentistry, Osaka University, Japan) and Professor Toshihide Noguchi (Department of Periodontology, School of dentistry, Aichi gakuin University, Japan), Professor Toshio Teranaka (Department of operative dentistry, School of Dentistry, Kanagawa Dental College, Japan), and Professor Naoto Suda (Department of Orthodontics, School of Dentistry, Meikai University, Japan). It was announced in an Advance Online Publication in “Journal of Biological Chemistry"

Full paper can be downloaded below

Published: 29 Oct 2011

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http://www.sut.ac.jp/en/ Tokyo University of Science http://www.jbc.org/ Journal of Biological Chemistry

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Medicine