He, Y., Zhang, Y., Liu, X., Ghazaryan, E., Li, Y., Xie, J., & Su, G. (2014). Recent advances of stem cell therapy for retinitis pigmentosa. International journal of molecular sciences, 15(8), 14456–14474. https://doi.org/10.3390/ijms150814456
Abstract
Retinitis pigmentosa (RP) is a group of inherited retinal disorders characterized by progressive loss of photoreceptors and eventually leads to retina degeneration and atrophy. Until now, the exact pathogenesis and etiology of this disease has not been clear, and many approaches for RP therapies have been carried out in animals and in clinical trials. In recent years, stem cell transplantation-based attempts made some progress, especially the transplantation of bone marrow-derived mesenchymal stem cells (BMSCs). This review will provide an overview of stem cell-based treatment of RP and its main problems, to provide evidence for the safety and feasibility for further clinical treatment.
Introduction
Retinitis pigmentosa (RP) is a group of inherited retinal disorders characterized by progressive loss of photoreceptors and eventually leads to retina degeneration and atrophy. New approaches for RP therapies include: cell transplantation therapy, gene therapy, cytokine therapy, nutrition therapy, and hyperbaric oxygen therapy. Present therapies for RP are restricted in their efficacy or safety, for example: maintenance of long-term efficacy using a single injection of cytokines is difficult, but there is a risk of infection after repeated intra-vitreous injections in cytokine therapy. Gene therapy has been shown to improve visual function in inherited retinal disease. RP is a hereditary cause of blindness, which has four main modes of inheritance: Autosomal dominant RP (ADRP), autosomal recessive RP (ARRP), X-linked RP (XLRP) and dihybrid inheritance, also mitochondrial genetic and non-genetic forms. Thomas and colleagues injected rAAV2-VMD2-hMERTK vector into the sub-retinal space of RCS rats and SD rats; it showed improvement of visual function in RCS, they also performed a series of safety studies in normal SD rats, and demonstrated that no local or systemic toxicity was detected after either dose of vector delivery and no indication of vector spread outside the treated eye. This group also prompted a phase I clinical trial in which rAAV2-VMD2-hMERTK vector were injected into the sub-retinal space of patients with retinal disease due to MERTK mutations.
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