Abstract
The cornea is an ideal target for gene therapy due to its accessibility, immune-privileged nature, and ability to perform frequent non-invasive assessment. Gene therapy has been studied in animal models of ocular corneal graft rejection, neovascularization, wound healing, fibrosis, macular degeneration, optic neuropathy, and retinal degeneration. Medical research has widely used animal models in the pursuit of improved therapeutic strategies intended to aid people. In this review we summarize the vectors currently used in ocular gene therapy and the potential applications of emerging new strategies for use in veterinary medicine.
Highlights
Almost all advances in ocular and vision research require the testing of new therapeutic strategies in animal models prior to pilot use in clinical trials
Gene therapy has been studied in animal models of ocular corneal graft rejection, neovascularization, wound healing, fibrosis, macular degeneration, optic neuropathy, and retinal degeneration [6,7,8,9,10,11,12,13,14]
The results of our study demonstrated that adenoassociated vector serotype 5 (AAV5) is an effective vector for gene therapy in canine corneal fibroblasts and myofibroblasts in vitro, based on the successful transduction of green fluorescent protein (GFP) with AAV5 vector into CCFs and canine corneal myofibroblasts (CCMs) as determined via fluorescent microscopy (Figure 1)
Summary
Almost all advances in ocular and vision research require the testing of new therapeutic strategies in animal models prior to pilot use in clinical trials. Pioneer efforts in gene therapy for congenital LCA has demonstrated significant improvement of visual impairment in affected Briard dogs and is being used to help restore vision in children [2]. This wellknown scientific advancement serves to demonstrate the importance of animal models of retinal degeneration and the essential role they have played in the discovery of underling genetic defects, mechanism of disease, and development of therapeutic strategies. Successful vision restoration through the use of gene therapy in patients affected with LCA has demonstrated the ability of gene therapy to potentially cure ocular disease and prevent long-term blindness [3,4]. The success of corneal gene therapy depends on the type of vector, the extent of therapeutic gene expression, and adverse immune responses [15]
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