Please use this identifier to cite or link to this item: 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
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