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Code : 19300-2136      Created Date : Sunday, April 28, 2019   Update Date : Tuesday, April 30, 2019    Visit : 811

English Lecture Series: Dr. Faranak Mavandadnejad

The next lecture of summer semester english lecture series (2019) was held by Dr. Faranak Mavandadnejad on April 15, 2019 in the title of Tamoxifen therapy in a murine model of myotubular myopathy.

Summary of the talk:

Myotubular myopathy (MTM) is a severe X-linked disease without existing therapies. Here, we show that tamoxifen ameliorates MTM-related histopathological and functional abnormalities in mice, and nearly doubles survival. The beneficial effects of tamoxifen are mediated primarily via estrogen receptor signaling, as demonstrated through in vitro studies and in vivo phenotypic rescue with estradiol. RNA sequencing and protein expression analyses

revealed that rescue is mediated in part through post-transcriptional reduction of dynamin-2, a known MTM modifier. These findings demonstrate an unexpected ability of tamoxifen to improve the murine MTM phenotype, providing preclinical evidence to supportclinical translation.







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