RNA Research

The French Institute I-Stem Realizes First Innovative Screens Using Stem Cells to Identify Drugs for Myotonic Dystrophy, DM1 Disease

Published in Gene Therapy Weekly, April 2nd, 2009

Four research teams of I-STEM[*] have joined forces in a collaborative project that has just achieved a first pilot therapy-oriented screen of compounds and RNA interference aiming at reversing the altered phenotypes observed in human embryonic stem cells carrying the mutant gene for myotonic dystrophy type1. This assay inaugurates a series of R&D planned in 2009.

Human embryonic stem (hES) cells lines carrying the mutant gene responsible for diseases may replicate associated molecular defects associated and be used, therefore, to analyse pathological mechanisms and search for treatments. I-STEM teams have shown that hES cell lines carrying the mutant gene responsible...

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Source: Gene Therapy Weekly (2009-04-02)

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