Can Neural Stem Cells Slow Retinal Degeneration and Preserve Vision?

The progressive degeneration of the retina, responsible for hereditary diseases such as retinitis pigmentosa, leads to irreversible vision loss by destroying photoreceptors and pigment epithelial cells. Despite advances in gene therapy targeting specific mutations, the genetic diversity of these conditions necessitates the development of universal therapeutic strategies. A promising approach involves the use of human neural stem cells, which can protect remaining photoreceptors and preserve visual function.

A recent study explored the long-term fate of these grafted cells in an animal model of retinal degeneration. The results show that neural stem cells, once injected, differentiate mainly into astroglial-like cells. They act by secreting trophic factors, modulating local metabolism, reducing oxidative stress and inflammation, and remodeling the extracellular matrix that supports the retina’s structure. These combined mechanisms help slow the loss of photoreceptors and maintain residual visual activity.

However, the effectiveness of this protection decreases over time. The interactions between the grafted cells and the host retinal cells weaken, limiting long-term benefits. Among the factors secreted by stem cells, the MANF protein plays a key role: it protects photoreceptors, regulates the immune response, and limits apoptosis. Its action, along with that of other molecules such as MYDGF, MDK, and PTN, is essential for supporting the survival of retinal cells in a degenerative environment.

Researchers also observed that retinal glial cells, such as Müller cells and microglia, have their activity altered by the presence of stem cells. These changes help stabilize the retinal structure and reduce harmful inflammatory reactions. Yet, as the disease progresses, these protective effects diminish, highlighting the importance of improving the retinal environment to extend therapeutic benefits.

These findings pave the way for combined approaches, associating stem cell transplantation with treatments specifically targeting the retinal environment. The goal is to enhance photoreceptor survival and maintain functional vision for as long as possible. The challenge remains to understand how to optimize these interactions for lasting protection against blindness.

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Official Study Source

DOI: https://doi.org/10.1038/s41467-026-69776-4

Title: Dynamic transcriptomic remodeling in grafted human neural progenitor cells uncovers mechanisms for vision preservation in a rat model of retinitis pigmentosa

Journal: Nature Communications

Publisher: Springer Science and Business Media LLC

Authors: Saba Shahin; Shaughn Bell; Bin Lu; Somanshu Banerjee; Vivek Swarup; Hui Xu; Jason Chetsawang; Stephany Ramirez; Jorge S. Alfaro; Alexander Laperle; Soshana Svendsen; Clive N. Svendsen; Shaomei Wang