Related Experiment Video
Updated: Aug 5, 2026
07:43
Subretinal Transplantation of Human Embryonic Stem Cell-Derived Retinal Tissue in a Feline Large Animal Model
Published on: August 5, 2021
Stem Cell Therapy for Inherited Retinal Disorders: Rebuilding the Retina
1Wilmer Eye Institute, Johns Hopkins University, 600 North Wolfe Street, Baltimore, MD 21287, United States.
American Journal of Ophthalmology
|July 30, 2026
Summary
Stem cell therapies show promise for inherited retinal disorders (IRDs) by replacing damaged cells or providing neuroprotection. While safe administration is demonstrated in early trials, larger studies are needed to confirm efficacy in preventing vision loss.
Area of Science:
- Ophthalmology
- Regenerative Medicine
- Genetics
Background:
- Inherited retinal disorders (IRDs) cause progressive vision loss due to photoreceptor and retinal pigment epithelium (RPE) degeneration.
- Stem cell therapy offers a potential treatment strategy for IRDs.
- Current approaches focus on cell replacement or neuroprotection.
Purpose of the Study:
- To review the current status of stem cell therapies for inherited retinal disorders.
- To assess the safety and potential efficacy of stem cell transplantation in clinical trials.
Main Methods:
- Review of recent clinical trials involving stem cell administration for IRDs.
- Analysis of different routes of stem cell delivery: intravitreal, sub-retinal, and suprachoroidal injections.
- Evaluation of therapeutic strategies including cell replacement and neuroprotection.
Main Results:
- Stem cell therapies have been safely administered in early-phase clinical trials for IRDs.
- Demonstrated safety across various administration routes.
- Preliminary evidence suggests potential for vision restoration or preservation.
Conclusions:
- Stem cell therapy is a viable and safe approach for treating inherited retinal disorders.
- Further large-scale clinical studies are essential to establish the efficacy of these treatments.
- Stem cell therapies hold significant promise for preventing or reversing vision loss in IRD patients.
Related Concept Videos
01:22iPS Cell Differentiation
The ability of induced pluripotent stem cells or iPSCs to differentiate into most body cell types has stimulated repair and regenerative medicine research over the past few decades. iPSC-derived blood cells, hepatocytes, beta islet cells, cardiomyocytes, neurons, and other cell types can repair injuries or regenerate damaged tissue in diseases such as diabetes and neurodegenerative disorders.
01:13Induced Pluripotent Stem Cells
Stem cells are undifferentiated cells that divide and produce different types of cells. Ordinarily, cells that have differentiated into a specific cell type are post-mitotic—that is, they no longer divide. However, scientists have found a way to reprogram these mature cells so that they “de-differentiate” and return to an unspecialized, proliferative state. These cells are also pluripotent like embryonic stem cells—able to produce all cell types—and are therefore called induced pluripotent stem...
01:21Stem Cell Therapy for Tissue Regeneration
Stem cell therapy is a method used in regenerative medicine to repair and restore function to damaged tissues and organs. Stem cells have the potential to proliferate and differentiate into various tissue types, making them ideal candidates for tissue regeneration. For example, hematopoietic stem cell transplants are commonly used in blood cancer treatment to replenish damaged bone marrow and restore healthy blood cells.
Types of Stem Cells used in Stem Cell Therapy
The two main cell types that...
Types of Stem Cells used in Stem Cell Therapy
The two main cell types that...
00:50Unrenewable Cells
In humans, the photoreceptor cells of the eye and sensory hair cells of the ear lack stem cells. These cells are thus unrenewable and cannot be replaced when they are damaged or destroyed.
Photoreceptors
The retina is composed of several layers and contains specialized cells called photoreceptors. The photoreceptors (rods and cones) change their membrane potential when stimulated by light energy. There are two types of photoreceptors—rods and cones—which differ in the shape of their outer...
Photoreceptors
The retina is composed of several layers and contains specialized cells called photoreceptors. The photoreceptors (rods and cones) change their membrane potential when stimulated by light energy. There are two types of photoreceptors—rods and cones—which differ in the shape of their outer...