Abstract
The eye is at the forefront of developing therapies for genetic diseases. With the FDA approval of the first gene-therapy drug for a form of congenital blindness, numerous studies have been initiated to develop gene therapies for other forms of eye diseases. These examinations have revealed new information about the benefits as well as restrictions to using drug-delivery routes to the different parts of the eye. In this article, we will discuss a brief history of gene therapy and its importance to the eye and ocular delivery landscape that is currently being investigated, and provide insights into their advantages and disadvantages. Efficient delivery routes and vehicle are crucial for an effective, safe, and longer-lasting therapy.
Highlights
There are over 300 genes associated with inherited retinal degenerations (IRDs), which lead to the dysfunction and degeneration of photoreceptors and/or the retinal pigment epithelium (RPE) [8]
The patients can present a spectrum of clinical manifestations ranging from congenital or juvenile-onset diseases to adulthood onset diseases, such as some forms of retinitis pigmentosa, rod-cone dystrophy and cone-rod dystrophy [9,10,11]
The scaffold matrix attachment region (S/MAR)-containing sequence has been shown to maintain the plasmid in an episomal state and bind to nuclear scaffold proteins [44]. This allows efficient attachment to the nuclear matrix and DNA entry into the nucleus. These plasmid modifications have been shown to be effective in gene delivery in Rpe65−/−, Abca4−/− and Rhodopsin−/− [41,45,46]
Summary
Publisher’s Note: MDPI stays neutral with regard to jurisdictional claims in published maps and institutional affiliations. The photoreceptors (rods and cones) account for >70% of the cell types in the retina and are the first responders to light. They contain the photopigment opsin that isomerizes in response to light and generates action potential. The importance of cones in maintaining our quality of life is exemplified by the presence of a cone-rich and rodless central area in the primate retina called the fovea. This structure is part of the macula, which contains the highest density of rods and cones in the central region [2]. Biomolecules 2021, 11, 1135 and glaucoma, the rare forms of inherited retinal degenerations (IRDs) have presented unique challenges in management and treatment
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