Abstract
Stem cell and cell reprogramming technology represent a rapidly growing field in regenerative medicine. A number of novel neural reprogramming methods have been established, using pluripotent stem cells (PSCs) or direct reprogramming, to efficiently derive specific neuronal cell types for therapeutic applications. Both in vitro and in vivo cellular reprogramming provide diverse therapeutic pathways for modeling neurological diseases and injury repair. In particular, the retina has emerged as a promising target for clinical application of regenerative medicine. Herein, we review the potential of neuronal reprogramming to develop regenerative strategy, with a particular focus on treating retinal degenerative diseases and discuss future directions and challenges in the field.
Highlights
The vertebrate retina is a multi-layer tissue at the back of the eye that is responsible for visual function
Two major retinal degenerative disorders are age-related: glaucoma, which is caused by degeneration of the optic nerve; and age-related Macular Degeneration (AMD), which is characterized by degeneration of retinal pigment epithelium (RPE) and photoreceptors
Technological advances in cell reprogramming technology over the past few years have opened up new opportunities for regenerative medicine targeting the central nervous system and the retina
Summary
The vertebrate retina is a multi-layer tissue at the back of the eye that is responsible for visual function. Two major retinal degenerative disorders are age-related: glaucoma, which is caused by degeneration of the optic nerve; and age-related Macular Degeneration (AMD), which is characterized by degeneration of retinal pigment epithelium (RPE) and photoreceptors These two retinal dystrophies combine to affect 250–280 million patients worldwide [5,6]. Retinitis pigmentosa causes irreversible vision loss due to the degeneration of photoreceptors, while Leber’s hereditary optic neuropathy is an inherited mitochondrial disorder that results in degeneration of RGCs. Dominant optic atrophy is another common IRD inherited in an autosomal dominant pattern with high penetrance, which leads to visual defects by damaging RGCs and the optic nerve. Current advances in cell reprogramming technology allow the conversion of one cell type into another, providing an attractive strategy to generate retinal neurons to repair the retina. These iPSC disease models could be used for testing therapeutic interventions, such as candidate or novel drugs and neuroprotective compounds [17]
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