ArticleNeurotherapeutics : the journal of the American Society for Experimental NeuroTherapeutics2021
DNAzyme Cleavage of CAG Repeat RNA in Polyglutamine Diseases.
Article in Neurotherapeutics : the journal of the American Society for Experimental NeuroTherapeutics, 2021. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 8 papers.
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Who cites it
8 citing papers in PubMed, 17 citations in OpenAlex.
- DNAzyme-Based Nanostructures for Dynamic Cell Regulation and Sensing.ChemistryOpen · 2026Review
- Exosomes as nanocarriers for brain-targeted delivery of therapeutic nucleic acids: advances and challenges.Journal of nanobiotechnology · 2025Review
- Facilitating DNAzyme transport across the blood-brain barrier with nanoliposome technology.Scientific reports · 2025Article
- Treatment of neurological pathology and inflammation in Machado-Joseph disease through in vivo self-assembled siRNA.Brain : a journal of neurology · 2025Article
- Experimental Treatment with Edaravone in a Mouse Model of Spinocerebellar Ataxia 1.International journal of molecular sciences · 2023Article
- DNAzyme as a rising gene-silencing agent in theranostic settings.Neural regeneration research · 2022Article
- Mechanistic and Therapeutic Insights into Ataxic Disorders with Pentanucleotide Expansions.Cells · 2022Review
- The genetic and molecular features of the intronic pentanucleotide repeat expansion in spinocerebellar ataxia type 10.Frontiers in genetics · 2022Review
Corrections and comments
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Authors and funding
11 authors at 3 institutions in 1 country.
Funding
Abstract
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 diseases with high potency. DNAzyme preferentially cleaved the expanded allele in spinocerebellar ataxia type 1 (SCA1) cells. While cleavage was non-allele-specific for spinocerebellar ataxia type 3 (SCA3) cells, treatment of DNAzyme leads to improved cell viability without affecting mitochondrial metabolism or p62-dependent aggresome formation. DNAzyme appears to be stable in mouse brain for at least 1 month, and an intermediate dosage of DNAzyme in a SCA3 mouse model leads to a significant reduction of high molecular weight ATXN3 proteins. Our data suggest that DNAzyme is an effective RNA silencing molecule for potential treatment of multiple polyQ diseases.
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Registered trials
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