English
Prof. Masafumi Matsuo MD PhD
Prof. Masafumi Matsuo MD PhD
Development of Physical Therapy
Faculty of Rehabilitation
Kobe Gakuin University
Prof. Matsuo is engaging in a translation research to establish treatments for Duchenne muscular dystrophin (DMD). DMD is a fatal muscle wasting disease and no effective treatment is available yet.
Prof. Matsuo proposed exon skipping therapy for DMD in 1995 and this opened a new field for DMD treatment. Currently, many studies to achieve better outcomes of DMD patients are on-going in prof. Matsuo's laboratory.
Progress of exon skipping therapy
Meeting
- Myology 2016
Poster「A new antisense oligonucleotide composed of RNA/ENA chimera (A085) against dystrophin exon 45 significantly increased six-minute walk distance in Duchenne muscular dystrophy」 - IBC's 8th Annual AsiaTIDES（2016.2.24－26, Kyoto）
Keynote presentation「Exon-skipping Therapy Advancing Oligonucleotide Drug Development」
References
- Matsuo M, Masumura T, Nishio H, Nakajima T, Kitoh Y, Takumi T, Koga J, Nakamura H (1991)
Exon skipping during splicing of dystrophin mRNA precursor due to an intraexon deletion in the dystrophin gene of Duchenne muscular dystrophy Kobe.
J. Clin. Invest. 87: 2127-2131. - Takeshima Y, Nishio H, Sakamoto H, Nakamura H, Matsuo M (1995)
Modulation of in vitro splicing of the upstream intron by modifying an intra-exon sequence which is deleted from the dystrophin gene in dystrophin Kobe.
J. Clin. Invest. 95: 515-520. - Takeshima Y, Yagi M, Wada H, Ishibashi K, Nishiyama A, Kakumoto M, Sakaeda T, Saura R, Okumura K, Matsuo M (2006)
Intravenous infusion of an antisense oligonucleotide results in exon skipping in muscle dystrophin mRNA of Duchenne muscular dystrophy.
Pediatr. Res. 59: 690-694. - Matsuo M, Takeshima Y, Nishio H (2016)
Contributions of Japanese patients to development of antisense therapy for DMD.
Brain Dev. 38: 4-9.
