Stem Cell Transplantation Therapy for Retinal Degenerative Diseases
In the past decade, progress in the research on human embryonic stem cells (hESCs) and induced pluripotent stem cells (iPSCs) has provided the solid basis to derive retinal pigment epithelium, photoreceptors, and ganglion cells from hESCs/iPSCs for transp
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Stem Cell Transplantation Therapy for Retinal Degenerative Diseases Fabin Han and Guotong Xu
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Introduction
Retinal degenerations such as age-related macular degeneration (AMD), macular dystrophy, and retinitis pigmentosa (RP) are the major causes of the aging blindness, because of the neurodegeneration of the photoreceptors and their supportive retinal pigment epithelium (RPE) in the retina. The most common retinal degenerative disease is AMD, of which about 80–90% are dry AMD caused by eventual degeneration of RPEs and photoreceptors. The other 10–20% are wet AMD caused by abnormal neovascularization and subsequent fibrosis in the retina (MacLaren and Pearson 2007). Another common retinal degeneration is Stargardt disease (STGD), which is an autosomal recessive macular dystrophy in that the affected patients have early disease onset in the age of about 5–15 years. Most patients with STGD are affected by genetic
F. Han (*) The Institute for Translational Medicine, Shandong University/Affiliated Second Hospital, Jinan, Shandong, China The Institute for Tissue Engineering and Regenerative Medicine, Liaocheng University/Liaocheng People’s Hospital, Liaocheng, Shandong, China e-mail: [email protected] G. Xu School of Medicine/Department of Ophthalmology, Tongji University, Shanghai, China East China Stem Cell Bank/Tongji Stem Cell Bank, Tongji University School of Medicine, Shanghai, China
mutations in the ABCA4 gene to accumulate the highly toxic A2E, leading to degeneration of RPE and subsequent dysfunction of photoreceptors. Mutations in BEST1 cause retinal disorders of bestrophinopathies, which mostly affect the functions of neural circuit pathways in retina. The most common macular dystrophies of bestrophinopathies are macular dystrophy (BVMD) and autosomal recessive bestrophinopathy (ARB) (Guziewicz et al. 2017; Tanna et al. 2017). In these retinal degenerative diseases, initial RPE lesions eventually lead to subsequent degenerations of photoreceptors to cause blindness. Since retinal degenerative diseases are mainly caused by progressive loss of RPE cells, photoreceptors, ganglion cells, or microvascular cells (endothelial and pericytes) in the retina, stem cell replacement therapy will provide great perspective for patients with RDD (Li et al. 2016b). The purpose of stem cell therapy for retinal degeneration is to use transplanted stem cells to replace the degenerated retinal cells in the retina for functional restoration of the vision. In the past decade, a lot of the research has extensively been conducted to replace degenerated neural cells in the retina with different stem cells such as hESCs, iPSCs, fetal neural stem cells, and adult bone marrow–derived mesenchymal stem cells (MSCs) (Ramsden et al. 2013). Because of their pluripotency, hESCs and iPSCs have been efficiently induced to generate functional RPE (hESC/iPSC-RPE) or photoreceptors in vitro.
# Springer Nature Singapore Pte Ltd. 2020 F. Han, P. Lu (eds.), Stem Cell-based Therapy for Neurodegenerative Diseases, Advances in Experimental Medicine a
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