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Peptide-mediated delivery of CRISPR-Cas genome editors in the eye

This project is part of the Therapeutic Genomics Centre, a multidisciplinary programme of innovative approaches to treating rare genetic disorders and providing an enhanced training experience for DPhil applicants.

Academic Supervisors: Assoc. Prof. Jasmina Cehajic-Kapetanovic, Assoc. Prof. Kanmin Xue, Prof. Robert E MacLaren

Other Supervisors: Dr. Ross C Wilson, Dr. Fyodor Urnov (Innovative Genomics Institute, University of California, Berkeley)

Host Dept: Nuffield Department of Clinical Neurosciences (NDCN), Oxford

Delivery of CRISPR editing systems in vivo is a major challenge for clinical translation and therapy development. Specific to the eye, adeno-associated viral (AAV) vectors have limited cargo capacity, induce long-term expression of the editor, which is undesirable, and can be associated with inflammatory adverse effects. Therefore, there is a great need for an improved CRISPR delivery system for the neurons of the retina where most inherited retinal degenerations occur.

Recently our collaborators from the Innovative Genomics Institute at the University of California, Berkeley (UCB), have developed a new technology based on cell-penetrating peptides coupled with ribonucleoprotein (RNP) complex and demonstrated efficient gene editing in lymphocytes and central nervous system neurons with low toxicity (see Foss et al. Nat Biomed Eng 2023 https://www.nature.com/articles/s41551-023-01032-2 and He et al. Chem Sci 2020 https://pubs.rsc.org/en/content/articlelanding/2020/sc/d0sc00929f). In this DPhil project, we aim to explore novel delivery methods for therapeutic gene editing and specifically test this approach in retinal organoids and mouse models of retinal degeneration.

Studentship code: MRCTGCORE2024008

Please read these guidance notes for detail on how to apply

Click here to apply: IPP login screen (ox.ac.uk)

For more information on DPhil in Paediatrics: DPhil in Paediatrics | University of Oxford

For more information about our MRC-Oxford Doctoral Training Programme see: https://www.medsci.ox.ac.uk/study/graduateschool/mrcdtp