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Related Concept Videos

Induced Pluripotent Stem Cells01:13

Induced Pluripotent Stem Cells

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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...
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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.
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Stem cell research aims to find ways to use stem cells to regenerate and repair cellular damage. Over time, most adult cells undergo the wear and tear of aging and lose their ability to divide and repair themselves. Stem cells do not display a particular morphology or function. Adult stem cells, which exist as a small subset of cells in most tissues, keep dividing and can differentiate into a number of specialized cells generally formed by that tissue. These cells enable the body to renew and...
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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.
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Embryonic stem (ES) cells are undifferentiated pluripotent cells, meaning they can produce any cell type in the body. This gives them tremendous potential in science and medicine since they can generate specific cell types for use in research or to replace body cells lost due to damage or disease.
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Related Experiment Video

Updated: Oct 19, 2025

Retinal Organoid Induction System for Derivation of 3D Retinal Tissues from Human Pluripotent Stem Cells
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Pluripotent stem cell therapy for retinal diseases.

Ishrat Ahmed1, Robert J Johnston2, Mandeep S Singh1

  • 1Wilmer Eye Institute, Johns Hopkins University School of Medicine, Baltimore, MD, USA.

Annals of Translational Medicine
|September 17, 2021
PubMed
Summary

Pluripotent stem cells (PSCs) show promise for treating retinal diseases like age-related macular degeneration. Clinical trials demonstrate the safety and feasibility of transplanting PSC-derived retinal pigment epithelium (RPE) cells.

Keywords:
Retinitis pigmentosa (RP)age-related macular degeneration (AMD)embryonic stem cell (ESC)induced pluripotent stem cell (iPSC)retinal organoid

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Area of Science:

  • Regenerative Medicine
  • Ophthalmology
  • Stem Cell Biology

Background:

  • Pluripotent stem cells (PSCs), including human embryonic stem cells (hESCs) and induced pluripotent stem cells (iPSCs), are explored for therapeutic applications.
  • Retinal degenerative diseases involve functional loss or damage of retinal cells, creating a need for novel treatments.
  • The retina's accessibility and advanced imaging make it a suitable model for testing stem cell therapies.

Purpose of the Study:

  • To review preclinical studies and clinical trials on the use of PSCs for retinal diseases.
  • To evaluate the safety and feasibility of PSC-derived cell transplantation in treating retinal disorders.
  • To highlight emerging technologies enhancing PSC-based retinal therapies.

Main Methods:

  • Review of preclinical research in animal models of retinal diseases.
  • Analysis of data from phase I/II clinical trials involving PSC-derived retinal pigment epithelium (RPE) transplantation.
  • Examination of ongoing clinical trials for age-related macular degeneration (AMD), Stargardt disease (STGD1), and retinitis pigmentosa (RP).

Main Results:

  • Preclinical studies show improved visual outcomes with PSC-derived photoreceptor or RPE cell transplantation.
  • Phase I/II clinical trials indicate safety and feasibility of PSC-derived RPE transplantation for AMD and STGD1.
  • Multiple ongoing clinical trials are investigating PSC-derived cells for various retinal conditions.

Conclusions:

  • PSC-based transplantation is a promising therapeutic strategy for retinal degenerative diseases.
  • Current clinical trials demonstrate the safety and potential efficacy of PSC-derived RPE cells.
  • Future advancements in gene editing, organoid transplantation, and imaging will further enhance PSC therapies for vision restoration.