An enhanced Eco1 retron editor enables precision genome engineering in human cells without double-strand breaks
Retrons are a retroelement class found in diverse prokaryotes that can be adapted to augment CRISPR-Cas9 genome engineering technology to efficiently rewrite short stretches of genetic information in bacteria and yeast. However, efficiency in human cells has been limited by unknown factors. We identified non-coding RNA (ncRNA) instability and impaired Cas9 activity due to 5' sgRNA extension as key contributors to low retron editor efficiency in human cells. We re-engineered the Eco1 ncRNA to incorporate an exoribonuclease-resistant RNA pseudoknot from the Zika virus 3' UTR and devised an RNA processing strategy using Csy4 ribonuclease to minimize 5' sgRNA extension. This strategy increased steady-state ncRNA levels and rescued sgRNA activity, leading to increased templated repair. This work reveals a previously unappreciated role for ncRNA stability in retron editor efficiency in human cells and presents an enhanced Eco1 retron editor capable of precise genome editing in human cells from a single integrated lentivirus and, in the context of the nCas9 H840A nickase, without creating double-strand breaks.
Authors
- John T. Poirier (ORCID: https://orcid.org/0000-0001-9795-5644)
- Charles M. Rice (ORCID: https://orcid.org/0000-0003-3087-8079)
- Matthew A. Cattle (ORCID: https://orcid.org/0000-0003-0809-1245)
- William M. Schneider (ORCID: https://orcid.org/0000-0001-9407-6118)
- Lauren C Aguado
- Yueyang Wang (ORCID: https://orcid.org/0000-0002-8456-1440)
- Susan Smith (ORCID: https://orcid.org/0000-0002-8213-5915)
- Thales Papagiannakopoulos (ORCID: https://orcid.org/0000-0002-2251-1624)
- Sanjana Venkittu
- Samantha Sze
Institutions
- NYU Langone Health (US)
- New York University (US)
- Rockefeller University (US)
Publication Details
- Journal
- Nucleic Acids Research
- Published
- 2025-07-14
- DOI
- https://doi.org/10.1093/nar/gkaf716
- Citations
- 2
- Primary Topic
- CRISPR and Genetic Engineering
- Type
- article
- Field-Weighted Citation Impact
- 1.61
Funders
- Robertson Foundation
- National Cancer Institute
- National Institute of General Medical Sciences