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PreprintCC BY-NC-NDbioRxiv

Single-vector AAV delivery of En-ISPmu1 and donor DNA for in vivo targeted genome integration

  • A. Conci
  • I. Bonuzzi
  • M. Ciciani
  • F. Esposito
  • P. Piccolo
  • F. Collaud
  • G. Ronzitti
  • A. Auricchio
  • N. Segata
  • A. Cereseto1
  1. 1University Trento
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Abstract

TnpBs are compact RNA-guided nucleases ideally suited for in vivo delivery. By screening novel TnpBs identified from large metagenomic datasets, ISPmu1 was discovered and its protein and {omega}RNA were engineered to generate En-ISPmu1, which achieved editing efficiencies exceeding 65% across 12 human loci. Owing to its compact size, En-ISPmu1 was delivered together with a donor DNA in a single AAV vector, yielding up to 12.7% targeted integration in mouse liver. These findings establish En-ISPmu1 as a promising versatile platform for in vivo therapeutic genome editing applications.

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Cite

BibTeXRIS
BibTeX
@misc{conci2026singlevector,
  title = {{Single-vector AAV delivery of En-ISPmu1 and donor DNA for in vivo targeted genome integration}},
  author = {A. Conci and I. Bonuzzi and M. Ciciani and F. Esposito and P. Piccolo and F. Collaud and G. Ronzitti and A. Auricchio and N. Segata and A. Cereseto},
  howpublished = {bioRxiv},
  year = {2026},
  month = oct,
  doi = {10.64898/2026.10.26.761871},
  url = {https://www.biorxiv.org/content/10.64898/2026.10.26.761871v1},
  note = {CC BY-NC-ND},
}
RIS
TY  - UNPB
TI  - Single-vector AAV delivery of En-ISPmu1 and donor DNA for in vivo targeted genome integration
AU  - A. Conci
AU  - I. Bonuzzi
AU  - M. Ciciani
AU  - F. Esposito
AU  - P. Piccolo
AU  - F. Collaud
AU  - G. Ronzitti
AU  - A. Auricchio
AU  - N. Segata
AU  - A. Cereseto
PY  - 2026
DA  - 2026/10/26
PB  - bioRxiv
DO  - 10.64898/2026.10.26.761871
UR  - https://www.biorxiv.org/content/10.64898/2026.10.26.761871v1
AB  - TnpBs are compact RNA-guided nucleases ideally suited for in vivo delivery. By screening novel TnpBs identified from large metagenomic datasets, ISPmu1 was discovered and its protein and {omega}RNA were engineered to generate En-ISPmu1, which achieved editing efficiencies exceeding 65% across 12 human loci. Owing to its compact size, En-ISPmu1 was delivered together with a donor DNA in a single AAV vector, yielding up to 12.7% targeted integration in mouse liver. These findings establish En-ISPmu1 as a promising versatile platform for in vivo therapeutic genome editing applications.
ER  -