ArticleNAR molecular medicine2024
Cas9 nickase-mediated contractions of CAG/CTG repeats are transcription-dependent and replication-independent.
Article in NAR molecular medicine, 2024. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 3 papers.
What it found
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The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
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Who cites it
3 citing papers in PubMed.
- Therapeutic Strategies Targeting the Molecular Pathogenesis of Myotonic Dystrophy Type 1: Current Status and Future Directions.Molecular diagnosis & therapy · 2026Review
- Emerging drivers of DNA repeat expansions.Biochemical Society transactions · 2025Review
- Recurrent DNA nicks drive massive expansions of (GAA)Proceedings of the National Academy of Sciences of the United States of America · 2024Article
Corrections and comments
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Authors and funding
9 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
There is currently no disease-modifying treatment for expanded CAG/CTG repeat disorders. Given that longer repeat tracts lead to an earlier age of disease onset and faster progression, contracting them is expected to improve symptoms and/or delay onset. We have previously demonstrated that the Cas9 D10A nickase can effectively contract CAG/CTG repeats when targeted to the repeat tract itself. However, the mechanism remains unclear. Here, we tested whether nickase-mediated contractions depend on transcription or on replication using human cell models. We find that transcription promotes contractions and that they occur independently of the rate of cell division. These results support the therapeutic potential of this approach in non-dividing cells.
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Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.