Eye
University of Oxford (MacLaren, Cehajic-Kapetanovic, Xue) | UC Berkeley IGI (Wilson)
The Eye Theme is working to develop and test a set of gene therapy strategies for inherited retinal diseases (IRDs) and age-related macular degeneration (AMD), as well as the vehicles to deliver these therapies.
We are pursuing three parallel projects, to develop a comprehensive suite of tools for treating ophthalmic genetic disease.
Research projects
Automated cell culture and screening at scale
Using the CellXpress.ai platform for faster and more standardised testing of potential therapies
Non-AAV delivery to retina
Investigating alternatives to adeno-associated viral (AAV) vectors, to deliver base editors to the eye
Gene knockdown in Wet AMD
Working to control AMD using CRISPR
Gene replacement in Retinitis Pigmentosa
Working to restore functional protein expression
Theme publications
CRISPR targeting of SNPs associated with age-related macular degeneration in ARPE-19 cells: a potential model for manipulating the complement system
This study explores a novel CRISPR targeting approach to manipulate the complement cascade in AMD using the reliable ARPE19 cell line as an in vitro model. Our results demonstrate efficient DNA editing with CRISPR in ARPE-19 cells, with notable allele specificity in the editing of various AMD-related SNPs in the complement system. The key factor influencing allele specificity is the distance of the target SNP from the PAM site.
Central Retinal Sensitivity Decline in RPGR-related Retinal Phenotypes
Purpose: To characterize the natural history of central retinal sensitivity decline in RPGR-associated retinopathy, and to evaluate and quantify phenotypic differences in disease progression using microperimetry-derived metrics.
Design: A retrospective cohort study SUBJECTS: 50 patients with genetically confirmed RPGR mutations (36 rod-cone, 14 cone-rod dystrophy [CORD]), contributing 357 microperimetry assessments.
Potential CRISPR Base Editing Therapeutic Options in a Sorsby Fundus Dystrophy Patient
Team
Dr Maram Abdalla Elsayed
DPhil Student, University of Oxford
Marco Bellini
Project Manager, Clinical Ophthalmology Research Group, University of Oxford
Grace Borchert
DPhil Student, Ramsay Scholar and Clarendon Scholar, University of Oxford
Dr Jasmina Cehajic Kapetanovic
MRC CoRE TG Eye Theme Co-Lead
MRC CoRE TG Patient and Public Involvement and Engagement Co-Lead
MRC Clinician Scientist and Consultant Vitreo-Retinal Surgeon, University of Oxford
Professor Robert MacLaren
MRC CoRE TG Eye Theme lead
Professor of Ophthalmology and Consultant Vitreoretinal Surgeon, University of Oxford
Benjamin Ng
DPhil Student, Fight for Sight Scholar, University of Oxford
Dr Celia Sourd
Postdoctoral Research Scientist, University of Oxford
Professor Ross Wilson
Dr Kanmin Xue
MRC CoRE Eye Theme Co-Lead
Associate Professor & Wellcome Clinical Research Career Development Fellow, University of Oxford