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https://doi.org/10.1186/s13287-021-02539-8
Title: | cGMP-grade human iPSC-derived retinal photoreceptor precursor cells rescue cone photoreceptor damage in non-human primates | Authors: | SWATHI LINGAM LIU ZENGPING Yang, Binxia Wong, Wendy BHAV HARSHAD PARIKH Ong, Jun Yi Goh, Debbie Wong, Daniel Soo Lin Tan, Queenie Shu Woon Tan, Gavin SW Holder, Graham E REGHA KAKKAD VELUCHAMY AMUTHA BARATHI Hunziker, Walter LINGAM GOPAL ZENG XIANMIN SU XINYI |
Keywords: | Science & Technology Life Sciences & Biomedicine Cell & Tissue Engineering Cell Biology Medicine, Research & Experimental Research & Experimental Medicine Cell therapy Induced pluripotent stem cells Non-human primates Photoreceptor precursors OPTICAL COHERENCE TOMOGRAPHY PROGENITOR CELLS GENE-THERAPY TRANSPLANTATION GENERATION EPITHELIUM DELIVERY SHEETS TISSUE MODEL |
Issue Date: | 19-Aug-2021 | Publisher: | BMC | Citation: | SWATHI LINGAM, LIU ZENGPING, Yang, Binxia, Wong, Wendy, BHAV HARSHAD PARIKH, Ong, Jun Yi, Goh, Debbie, Wong, Daniel Soo Lin, Tan, Queenie Shu Woon, Tan, Gavin SW, Holder, Graham E, REGHA KAKKAD, VELUCHAMY AMUTHA BARATHI, Hunziker, Walter, LINGAM GOPAL, ZENG XIANMIN, SU XINYI (2021-08-19). cGMP-grade human iPSC-derived retinal photoreceptor precursor cells rescue cone photoreceptor damage in non-human primates. STEM CELL RESEARCH & THERAPY 12 (1). ScholarBank@NUS Repository. https://doi.org/10.1186/s13287-021-02539-8 | Abstract: | Background: Retinal regenerative therapies hold great promise for the treatment of inherited retinal degenerations (IRDs). Studies in preclinical lower mammal models of IRDs have suggested visual improvement following retinal photoreceptor precursors transplantation, but there is limited evidence on the ability of these transplants to rescue retinal damage in higher mammals. The purpose of this study was to evaluate the therapeutic potential of photoreceptor precursors derived from clinically compliant induced pluripotent stem cells (iPSCs). Methods: Photoreceptor precursors were sub-retinally transplanted into non-human primates (Macaca fascicularis). The cells were transplanted both in naïve and cobalt chloride-induced retinal degeneration models who had been receiving systemic immunosuppression for one week prior to the procedure. Optical coherence tomography, fundus autofluorescence imaging, electroretinography, ex vivo histology and immunofluorescence staining were used to evaluate retinal structure, function and survival of transplanted cells. Results: There were no adverse effects of iPSC-derived photoreceptor precursors on retinal structure or function in naïve NHP models, indicating good biocompatibility. In addition, photoreceptor precursors injected into cobalt chloride-induced retinal degeneration NHP models demonstrated an ability both to survive and to mature into cone photoreceptors at 3 months post-transplant. Optical coherence tomography showed restoration of retinal ellipsoid zone post-transplantation. Conclusions: These findings demonstrate the safety and therapeutic potential of clinically compliant iPSC-derived photoreceptor precursors as a cell replacement source for future clinical trials. | Source Title: | STEM CELL RESEARCH & THERAPY | URI: | https://scholarbank.nus.edu.sg/handle/10635/206729 | ISSN: | 1757-6512 | DOI: | 10.1186/s13287-021-02539-8 |
Appears in Collections: | Staff Publications Elements |
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cGMP-grade human iPSC-derived retinal photoreceptor precursor cells rescue cone photoreceptor damage in non-human primates.pdf | Published version | 2.72 MB | Adobe PDF | OPEN | Published | View/Download |
cGMP-grade human iPSC-derived retinal photoreceptor precursor cells rescue cone photoreceptor damage in non-human primates.pdf | 2.72 MB | Adobe PDF | CLOSED (no policy) | None |
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