Personalized stem cell-based therapy for degenerative retinal diseases
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Personalized stem cell-based therapy for degenerative retinal diseases Huirong Li 1 & Ling Hou 1
# Springer Science+Business Media, LLC, part of Springer Nature 2020
Gene therapy is a feasible strategy to treat genetic diseases but to date is successful only in a minority of patients. Photoreceptors (rods and cones) are the main light-detecting cells in the neural retina, capable of transferring visual information via phototransduction into electrical signals. Loss of photoreceptors due to degeneration is a major threat to human health as progressive vision loss severely interferes with a person’s daily activities and leads to high socio-economic costs. Unfortunately, there are few options to effectively prevent photoreceptor degeneration or preserve photoreceptor survival in retinal degenerative diseases. Recently, proof-ofconcept findings by Barnea-Cramer et al. [1], published in Molecular Therapy, and by Anzalone et al. [2], published in Nature, show that in combination with gene editing and delivery, photoreceptor replacement therapy is a new potential approach for treating blindness caused by the loss of photoreceptors. For rod transplantation, primarily immature rod precursors have been reported to be optimal in both neonatal and adult mouse retina [3]. Transplanted precursors seem to incorporate into the outer nuclear layer (ONL) of the recipient retina and mature into photoreceptors, making synaptic contacts with host rod bipolar cells, responding to light stimulation and restoring scotopic vision [3, 4]. However, detailed analyses showed that the transplanted precursors do not truly integrate into the host ONL but rather restore functional proteins in host rods by donor-host cytoplasmic exchange [5, 6]. Recently, AAV-mediated gene transfer to Müller glia has led to de novo regeneration of rods that integrate well into the ONL and restore vision in mice with partial retinal degeneration [7]. In addition, increasing evidence shows that transplantation of rod
* Ling Hou [email protected] 1
Laboratory of Developmental Cell Biology and Disease School of Ophthalmology and Optometry and Eye Hospital State Key Laboratory of Ophthalmology, Optometry and Vision Science , Wenzhou Medical University , Wenzhou 325003, China
precursors can restore scotopic visual functions in mice with partial or even complete degeneration of the ONL [1, 3, 4]. Therefore, photoreceptor transplantation provides hope for clinical treatment of retinal degenerative diseases. Despite these promising advances, however, one should not disregard the fact that photoreceptor transplantation may also lead to immune-mediated rejection, notwithstanding the view that the retina is an immune-privileged site [8]. Evidently, to overcome immune rejection, one might consider an autologous source of photoreceptors for transplantation. In fact, patient-specific induced pluripotent stem cells (iPSCs) may provide such an autologous source of cells [9]. However, this strategy is hampered by high labor intensity, low efficiency, high costs and unpredictable m
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