Copies for CAG and CTG triplets repeat themselves numerous times and disrupt normal gene sequences in some neurodegenerative diseases, a new study reports.
Researchers have identified small molecules which allow for control over a genetic defect responsible for adult onset muscular dystrophy.
Recent findings by an international collaboration including IRCM researchers hold new implications for the pathogenesis of myotonic dystrophy.
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The new approach could have implications for many genetic diseases. While RNA is an appealing drug target, small molecules that...