Myostatin inhibitor peptides could be directly infused into

muscular dystrophy patients. In addition, a delivery system using myogenic cells is also possible. Furthermore, myostatin inhibition could be combined with other therapeutic approaches. Myostatin inhibition is considered to be most effective when combined with gene correction or other ways of delivering dystrophin (24). In this sense, one advantage of myostatin inhibitor peptides is their application to combined Inhibitors,research,lifescience,medical therapy for muscular dystrophy. If cDNAs for myostatin inhibitor peptides can be expressed in myogenic stem cells, cell-mediated therapy with myostatin inhibition would become possible (Fig. ​(Fig.1).1). By using this method,

defective genes such as dystrophin would be amended by myogenic stem cells. Alternatively, viral vectors containing myostatin inhibitor peptides could be combined with other possible therapies for muscular dystrophy, such as exon-skipping Inhibitors,research,lifescience,medical reagents or genes (24). Figure 1 Potential delivery systems for myostatin inhibitors in vivo. Studying the role of myostatin in tissues Inhibitors,research,lifescience,medical other than skeletal muscle is important to avoid the possible adverse effects of myostatin inhibition. In this respect, it is important to determine whether or not myostatin acts solely on skeletal muscles. Adipose tissues are affected by myostatin signaling. Reduction of adipose tissue mass is observed in myostatin-null mice. Whether myostatin directly acts on adipocytes

or factors from hypertrophied skeletal muscle secrete factors affecting adipocyte remains to be determined. Finally, ethical issues must be considered Inhibitors,research,lifescience,medical for use of myostatin inhibition. Athletes are already Selleckchem VRT752271 interested in myostatin for increasement of their muscle strength. There is a discussion that myostatin inhibition would be non-steroidal doping methods that are difficult to identify. Inhibitors,research,lifescience,medical In summary, I have presented an outline of myostatin inhibition therapy for muscular dystrophy with emphasis on a myostatin inhibitor derived from follistatin. I hope that this novel therapeutic strategy will prove useful toward establishing realistic therapies for intractable diseases, Ergoloid such as muscular dystrophy. Acknowledgments This research was supported by grants from the Ministry of Health, Labour and Welfare.
Various backbone chemistries of antisense oligonucleotide have been tested to overcome the problems of in vivo breakdown of DNA or RNA. Recent explorations of drug-like characteristics of AOs have lead to the development of oligonucleotides that contain phosphorothioate linkages throughout their length and 2’O-modifications of the ribose moiety (e.g. 2’-O-methyl, 2’-O-methoxyethyl). Previously we have shown that intramuscular injection of 2’-O-methyl antisense oligonucleotides (2OMeAO) can restore dystrophin expression (10).