Generalizable features of pegRNA design for prime editing of inherited retinal diseases
We establish a set of recommendations for the generalizable design of the non-engineered pegRNA 3' extension for the correction of several IRDs, enabling overall simplification of design parameters for PE2-based systems.
Authors
- Guei‐Sheung Liu (ORCID: https://orcid.org/0000-0003-3379-724X)
- Rajendra KC (ORCID: https://orcid.org/0000-0002-6403-8919)
- Anthony L. Cook (ORCID: https://orcid.org/0000-0003-1770-7910)
- Mohd Khairul Nizam Mohd Khalid (ORCID: https://orcid.org/0000-0001-7200-3102)
- Sandy Hung (ORCID: https://orcid.org/0000-0002-7496-7092)
- Alex William Hewitt (ORCID: https://orcid.org/0000-0002-5123-5999)
- Minh Thuan Nguyen Tran (ORCID: https://orcid.org/0000-0002-9554-6449)
- Kirsten A. Fairfax (ORCID: https://orcid.org/0000-0002-8394-137X)
- Freya M. Patterson (ORCID: https://orcid.org/0000-0003-0449-1380)
- Thomas Guinan (ORCID: https://orcid.org/0009-0009-9095-8274)
Institutions
- University of Tasmania (AU)
- The University of Melbourne (AU)
- Centre for Eye Research Australia (AU)
- Massachusetts Institute of Technology (US)
Publication Details
- Journal
- Ophthalmic Genetics
- Published
- 2025-11-09
- DOI
- https://doi.org/10.1080/13816810.2025.2576786
- Primary Topic
- Retinal Development and Disorders
- Type
- article
- Field-Weighted Citation Impact
- 0.00