Identification and characterization of human retinal stem cells capable of retinal regeneration

Good news!

"Human neural retinal stem-like cells (hNRSCs) found in fetal retinas and organoids show promise for retinal regeneration and vision restoration."

From the editor's summary and abstract:

"Editor’s summary

Cell-based therapies could potentially reverse retinal degeneration but require access to cells with high differentiation and safety profiles. Here, Liu et al. identified a distinct population of human neural retinal stem-like cells (hNRSCs) that reside in the ciliary marginal zone (CMZ) of fetal retinas and exhibit transcription profiles, indicating high differentiation and self-renewal potential. The authors found a similar cell type in a CMZ-like region of human retinal organoids (hROs). Transplanted hRO-derived hNRSCs differentiated into various retinal cell types and improved visual function in a mouse model of retinitis pigmentosa. hNRSCs could provide a useful and replenishable resource for translational research and therapeutic applications. ...

Abstract

Human retinal stem cells hold great promise in regenerative medicine, yet their existence and characteristics remain elusive.

Here, we performed single-cell multiomics and spatial transcriptomics of human fetal retinas and uncovered a cell subpopulation, human neural retinal stem-like cells (hNRSCs), distinct from retinal pigment epithelium stem-like cells and traditional retinal progenitor cells. We found that these hNRSCs reside in the peripheral retina in the ciliary marginal zone, exhibiting substantial self-renewal and differentiation potential.

We conducted single-cell and spatial transcriptomic analyses of human retinal organoids (hROs) and revealed that hROs contain a population of hNRSCs with similar transcriptional profiles and developmental trajectories to hNRSCs in the fetal retina potentially capable of regenerating all retinal cells.

Furthermore, we identified crucial transcription factors, such as MECOM, governing hNRSC commitment to neural retinogenesis and regulating repair processes in hROs. 

hRO-derived hNRSCs transplanted into the rd10 mouse model of retinitis pigmentosa differentiated and were integrated into the retina, alleviated retinal degeneration, and improved visual function.

Overall, our work identifies and characterizes a distinct category of retinal stem cells from human retinas, underscoring their regenerative potential and promise for transplantation therapy."

Contents | Science Translational Medicine 17, 791

Identification and characterization of human retinal stem cells capable of retinal regeneration (no public access) [This paper was apparently first published back in December 2023]

Identification and characterization of human retinal stem cells capable of retinal regeneration (open access, ResearchGate)