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Updated: Jul 4, 2025
Directed Induction of Retinal Organoids from Human Pluripotent Stem Cells
Published on: April 21, 2021
Enriching new transplantable RGC-like cells from retinal organoids for RGC replacement therapy
1State Key Laboratory of Ophthalmology, Zhongshan Ophthalmic Center, Sun Yat-sen University, Guangdong Provincial Key Laboratory of Ophthalmology and Visual Science, Guangzhou, 510060, China.
Abstract:
Optic neuropathies, such as glaucoma, are due to progressive retinal ganglion cells (RGCs) degeneration, result in irreversible vision loss. The promising RGCs replacement therapy for restoring vision are impeded by insufficient RGC-like cells sources. The present work was enriched one new type RGC-like cells using two surface markers CD184 and CD171 from human induced pluripotent stem cells (hiPSCs) by FACS sorting firstly. These new kind cells have well proliferation ability and possessed passage tolerance in vitro 2D or 3D spheroids culture, which kept expressing Pax6, Brn3b and βIII-Tubulin and so on. The transplanted CD184+CD171+ RGC-like cells could survive and integrate into the normal and optic nerve crush (ONC) mice retina, especially they were more inclined to across the optic nerve head and extend to the damaged optic nerve. These data support the feasible application for cell replacement therapy in RGC degenerative diseases, as well as help to develop new commercial cells sorting reagents and establish good manufacturing practice (GMP) grade RGC-like donor cells for further clinical application.
Insights
Researchers identified a new type of retinal ganglion cells (RGCs) from human induced pluripotent stem cells. These RGC-like cells show promise for cell replacement therapy in vision loss diseases.
Area of Science:
- Ophthalmology
- Stem Cell Biology
- Regenerative Medicine
Background:
- Optic neuropathies, including glaucoma, cause irreversible vision loss due to progressive retinal ganglion cell (RGC) degeneration.
- Current RGC replacement therapies are limited by a lack of suitable RGC-like cell sources.
Purpose of the Study:
- To identify and characterize a novel population of RGC-like cells derived from human induced pluripotent stem cells (hiPSCs).
- To evaluate the therapeutic potential of these cells for RGC degenerative diseases.
Main Methods:
- Isolation of RGC-like cells from hiPSCs using fluorescence-activated cell sorting (FACS) based on CD184 and CD171 surface markers.
- In vitro characterization of cell proliferation, passage tolerance, and expression of key RGC markers (Pax6, Brn3b, βIII-Tubulin).
- In vivo transplantation of RGC-like cells into normal and optic nerve crush (ONC) mouse retinas.
Main Results:
- A novel population of RGC-like cells, CD184+CD171+, was successfully isolated from hiPSCs.
- These cells demonstrated robust proliferation, passage tolerance in 2D and 3D cultures, and maintained RGC-specific gene expression.
- Transplanted cells survived, integrated into mouse retinas, and showed a propensity to migrate across the optic nerve head towards the damaged optic nerve.
Conclusions:
- The identified CD184+CD171+ RGC-like cells represent a viable source for cell replacement therapy in RGC degenerative diseases.
- These findings support the development of new cell sorting reagents and Good Manufacturing Practice (GMP)-grade RGC-like donor cells for clinical applications.
