Large Animal Models of Retinitis Pigmentosa in Therapy Development and Preclinical Testing

Large animal models are valuable for developing and testing translational therapies for inherited retinal dystrophies such as retinitis pigmentosa (RP). Gene augmentation therapy has been developed utilizing such models. Adeno-associated viral (AAV) vectors have been frequently utilized and delivered by intravitreal or subretinal injection. In vivo longitudinal assessments of therapeutic outcomes are essential. These include regular ophthalmic examinations as well as detailed fundus assessments including confocal scanning laser ophthalmoscopy (cSLO) and high-resolution cross-sectional imaging of the retina by spectral domain-optical coherence tomography (SD-OCT). Retinal function assessment includes vision testing and electroretinography (ERG).