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Induced Pluripotent Stem Cells Hold Promise for Treating Eye Diseases Including Macular Dystrophy

A multi-country team of researchers led by UC San Diego biologists has found that induced pluripotent stem cells (iPSCs) derived from a patient’s own cells can be turned into various types of functional cells to treat various diseases, but these specialized cells differ in their potential to provoke immune response despite being transplanted to the same patient they were collected from. Retinal pigment epithelium cells that can be used to treat eye diseases are among those that are not rejected, as the study published in the journal Cell Stem Cell shows.

In 2011 Yang Xu, a biology professor at UC San Diego who led the current study, and his colleagues discovered that specialized cells developed from induced pluripotent stem cells can cause the immune system to reject them in the same individual they are derived from. This happens due to abnormal gene expression in the transplanted cells.

This has been the major obstacle to the safe use of induced pluripotent stem cells since the time they were discovered. At the same time, these cells are of particular interest to the researchers developing of stem cell therapies as they are not derived from embryos and thus are not subject to various restrictions.

The researchers used mice to study effects of transplantation of cells derived from induced pluripotent stem cells. However, as mouse immune system is quite different to human one, they developed a humanized laboratory mouse – i.e. a mouse with a functional human immune system able to provide an immune response to cells derived from induced pluripotent stem cells from the same individual. This enabled them to evaluate the human immune responses to various specialized cells used in the research.

The researchers tested immune reactions to a variety of cell types derived from human iPSCs and found out that, for example, smooth muscle cells were strongly rejected by the humanized mice immune systems, while retinal pigment epithelial cells were tolerated well. This differential immunogenicity was due to abnormal quantities of immunogenic antigens produced in smooth muscle cells, which was not the case for retinal pigment epithelium cells.

The finding gives hope for patients with macular degeneration – the main cause of blindness and visual impairment in older adults. 


Fetal stem cells treatment results depend on: disease's severity, age of the patient, adherence for the medications and regime. Treatment results, presented on this site, are individual for each clinical case.