DNAzyme Cleavage of CAG Repeat RNA in Polyglutamine Diseases
Nan Zhang, Brittani Bewick, Jason Schultz, Anjana Tiwari et autres
CAG repeat expansion is the genetic cause of nine incurable polyglutamine (polyQ) diseases with neurodegenerative features. Silencing repeat RNA holds great therapeutic value. Here, we developed a repeat-based RNA-cleaving DNAzyme that catalyzes the destruction of expanded CAG repeat RNA of six polyQ …
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