Prinses Beatrix Spierfonds grant for Rick Wansink

Wansink Rik

Rick Wansink, Dept. of Cell Biology, theme Nanomedicine, has been awarded a € 250,000 grant by the Prinses Beatrix Spierfonds. The project entitled Do antisense transcripts make pathobiological sense in myotonic dystrophy? is designed to explore a potentially toxic function of a (CAG)n-repeat containing RNA.

A rare, antisense (CAG)n repeat RNA may be a new therapeutic target in myotonic dystrophy type 1, a severe neuromuscular disorder affecting 1:8.000 people worldwide. An archetypal member of the trinucleotide repeat disorder family, myotonic dystrophy is caused by an unstable (CTG·CAG)n repeat on chromosome 19. Sense transcription of this repeat, resulting in the formation of DMPK (CUG)n RNA, has been widely studied since 1992. Recent findings now show that antisense transcription of this locus produces DM1 antisense RNA carrying a (CAG)n repeat with a yet unknown function.

This Prinses Beatrix Spierfonds project will explore structure and function of this expanded antisense (CAG)n RNA in skeletal muscle from patients with myotonic dystrophy and its potential relevance as a novel target in therapy development.

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