Article
Oligonucleotide-based strategies to combat polyglutamine diseases.
Nucleic acids research - 1 Jun 2014
Fiszer Agnieszka, Krzyzosiak Wlodzimierz J
Abstract excerpt
Considerable advances have been recently made in understanding the molecular aspects of pathogenesis and in developing therapeutic approaches for polyglutamine (polyQ) diseases. Studies on pathogenic mechanisms have extended our knowledge of mutant protein toxicity, confirmed the toxicity of mutant transcript and identified other toxic RNA and protein entities. One very promising therapeutic strategy is targeting...
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