Related Experiment Video
Updated: Jul 12, 2026
Morphometric Analyses of Retinal Sections
Published on: February 19, 2012
Establishing Preclinical Quantitative Parameters for Future Assessment of Corneal and Retinal Therapeutics for
Sif G Kaad1, Andrea J Korecki1, Elizabeth M Simpson1,2,3
1Centre for Molecular Medicine and Therapeutics at BC Children's Hospital, The University of British Columbia, Vancouver, British Columbia, Canada.
Purpose:
Aniridia is a rare congenital-blinding disorder caused by pathogenic variants in PAX6 for which there is no vision-saving treatment. Here we aim to deliver quantitative mouse data sufficient to provide preclinical testing of therapeutic approaches to aniridia with reliable power calculations.
Methods:
We histologically quantify the peripheral thickness of the cornea, retina, and their sublayers in the B6129F1-Sey mouse model of aniridia, using the largest cohort of Wt and Sey mice to date (67 mice, 134 eyes).
Results:
We show a highly significant thinning of the total cornea and the corneal epithelium. However, the corneal stromal-endothelial layer thickness was unaffected by Sey. Nevertheless, the epithelium and stromal-endothelial layer thickness were highly significantly correlated in Sey mice only. We also show a highly significant thinning of the total retina, and a very-to-highly, significant thinning of the ganglion cell layer (GCL), inner nuclear layer (INL), and outer nuclear layer (ONL) sublayers. In the retina, we found that the GCL layer thickness was very significantly correlated with the ONL and INL thickness, in Sey mice only. Using the data generated here, we calculated the number of mice needed to demonstrate a full or partial correction of the Sey aniridic phenotype. For example, at 80% power a 50% effect will require: for the epithelium, 21 Wt and 21 Sey mice; total cornea, 58 Wt and 58 Sey mice; and total retina, 28 Wt and 28 Sey mice.
Conclusions:
We deliver quantitative mouse data, which provides preclinical research testing of therapeutic approaches with the information needed to make reliable power calculations.
Insights
Aniridia (a congenital blinding disorder) shows significant thinning in the cornea and retina. This study provides crucial mouse data for developing and testing new vision-saving treatments.
Area of Science:
- Ophthalmology
- Genetics
- Developmental Biology
Background:
- Aniridia is a rare congenital disorder causing blindness, linked to PAX6 gene mutations.
- Currently, no vision-saving treatments exist for aniridia.
- Preclinical research requires robust data for therapeutic development.
Purpose of the Study:
- To provide quantitative histological data in a mouse model of aniridia.
- To enable reliable power calculations for preclinical testing of aniridia therapies.
- To support the development of vision-saving treatments for aniridia.
Main Methods:
- Histological quantification of cornea and retina thickness in B6129F1-Sey aniridia mouse model.
- Analysis of peripheral cornea, retina, and their sublayers.
- Utilized the largest cohort to date (67 mice, 134 eyes) for robust statistical power.
Main Results:
- Significant thinning observed in total cornea and corneal epithelium.
- No significant change in corneal stromal-endothelial layer thickness.
- Significant thinning in total retina, ganglion cell layer (GCL), inner nuclear layer (INL), and outer nuclear layer (ONL).
- Correlated epithelial and stromal-endothelial layer thickness in Sey mice.
- Correlated GCL thickness with ONL and INL in Sey mice.
- Calculated sample sizes for preclinical trials based on observed effect sizes and power.
Conclusions:
- Quantitative data provided for preclinical aniridia research.
- Enables reliable power calculations for therapeutic approach testing.
- Aids in the development of effective vision-saving strategies for aniridia.
