Gene Therapy

Scientists at University of Rochester, Department of Neurology report research in gene therapy

Published in Gene Therapy Weekly, November 1st, 2007

Scientists discuss in 'Myotonic dystrophy: RNA-mediated muscle disease' new findings in gene therapy. "' The aim of this review is to highlight recent progress in elucidating the disease mechanism in myotonic dystrophy type 1 and type 2. Research on myotonic dystrophy has led to the recognition of a novel RNA-mediated disease process," scientists writing in the journal Current Opinion In Neurology report.

"In myotonic dystrophy it is the RNA rather than protein product of a disease gene that has deleterious effects on muscle cells. These unusual RNAs, which contain a long expanse of CUG or CCUG repeats, have far reaching effects on cell function by influencing...

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Source: Gene Therapy Weekly (2007-11-01)

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