Contact Information Available for logged-in reporters only Newswise LA JOLLA, Calif., April 11, 2014 A team of scientists led by Pier Lorenzo Puri, M.D., associate professor at Sanford-Burnham Medical Research Institute (Sanford-Burnham), in collaboration with Fondazione Santa Lucia in Rome, Italy, have published details of how a class of drugs called HDACis drive muscle-cell regeneration in the early stages of dystrophic muscles, but fail to work in late stages. The findings are key to furthering clinical development of HDACis for Duchenne muscular dystrophy (DMD), an incurable muscle-wasting disease. A symphony to rebuild muscle The research, to be published April 15 in Genes and Development, used mouse models of DMD to show how fibro-adipogenic progenitor cells (FAPs) act like orchestra conductors in the music of muscle regeneration. FAPs sit in the space between muscle fibers and coordinate a complex symphonyreceiving the notes that muscle has been damaged and directing muscle stem cellssatellite cellsto rebuild muscle. HDACis create an environment conducive for FAPs to direct muscle regenerationbut only during the early stages of DMD progression in mice, said Puri. At some point, DMD progresses to a pathological point of no return and become permanently resistant to muscle-regeneration cures and to HDACis. HDACis … Continue reading
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