{
  "abstract": "The best studied Cas9 enzyme, from Streptococcus pyogenes, is thought to be too large to be efficiently packaged into a single adeno-associated virus (AAV) together with a single-guide (sg) RNA cassette, requiring a dual-vector system. Here we have circumvented this limitation and packaged the full CRISPR system into a single AAV by using a single promoter driving the expression of both the Cas9 enzyme and a sgRNA. We compared the ability of the dual and single vector systems to induce contraction of CAG/CTG repeats in the striatum of a Huntington's disease model. We show that the all-in-one system led to larger contractions of the CAG/CTG repeats. This approach provides a more efficient and less costly approach for in vivo gene editing and improves the translational potential of AAV-mediated delivery of Cas9.",
  "abstract_shown": true,
  "affiliations": [
    "Dementia Research Institute at Cardiff University"
  ],
  "article_type": "New results",
  "arxiv": null,
  "author_total": 4,
  "authors": [
    {
      "name": "A. Murillo"
    },
    {
      "name": "A. N. Aston"
    },
    {
      "name": "M. Lelos"
    },
    {
      "affs": [
        0
      ],
      "name": "V. Dion"
    }
  ],
  "bibtex": "https://research.draconitic.com/paper/dp2mnf2mzp8cc.bib",
  "cited_by": 0,
  "doi": "10.64898/2026.10.30.762787",
  "fulltext_url": "https://www.biorxiv.org/content/10.64898/2026.10.30.762787v1.full",
  "id": "dp2mnf2mzp8cc",
  "issns": [],
  "landing_url": "https://www.biorxiv.org/content/10.64898/2026.10.30.762787v1",
  "language": null,
  "licence": "CC-BY",
  "licence_label": "CC BY",
  "licence_source": "article",
  "licence_url": "https://creativecommons.org/licenses/by/4.0/",
  "metadata_licence": null,
  "native_subjects": [
    [
      "biorxiv",
      "neuroscience"
    ]
  ],
  "openalex": null,
  "pdf_url": "https://www.biorxiv.org/content/10.64898/2026.10.30.762787v1.full.pdf",
  "pmcid": null,
  "pmid": null,
  "published": "2026-10-30",
  "publisher": null,
  "ris": "https://research.draconitic.com/paper/dp2mnf2mzp8cc.ris",
  "sources": [
    {
      "first_seen": "2026-10-31T12:21:07Z",
      "native_id": "10.64898/2026.10.30.762787",
      "source": "biorxiv",
      "url": "https://www.biorxiv.org/content/10.64898/2026.10.30.762787v1"
    }
  ],
  "subjects": [
    {
      "field": "biology",
      "origin": "biorxiv:neuroscience",
      "subfield": "neuroscience"
    }
  ],
  "title": "Single-AAV Delivery of the SpCas9 Nickase Improves CAG/CTG Repeat contraction in a mouse model of Huntington's disease",
  "title_text": "Single-AAV Delivery of the SpCas9 Nickase Improves CAG/CTG Repeat contraction in a mouse model of Huntington's disease",
  "type": "preprint",
  "updated_at": "2026-11-01T00:21:16Z",
  "url": "https://research.draconitic.com/paper/dp2mnf2mzp8cc",
  "venue": "bioRxiv",
  "versions": []
}