Macular Degeneration Stem Cell Treatment

Macular Degeneration and Stem Cell Therapy

What is Macular Degeneration?

Macular Degeneration and Stem Cell Therapy

Macular Degeneration and Stem Cell Therapy

Macular Degeneration or Age Related Macular Degeneration (AMD,ARMD) is a eyesight condition which mostly affects older people. AMD results in a loss of vision in the center of the visual field (the macula) because of damage or wear to the retina.

AMD can occur in either a wet or dry types. AMD is a major cause of visual impairment in people of 50 years age or more. AMD can make it difficult or impossible to read or to be able to recognize faces, although enough peripheral vision can remain to allow normal daily life.
Although some macular dystrophies that younger people get are referred to as macular degeneration, the term generally refers to age-related macular degeneration.


Stemming vision loss with stem cells.

J Clin Invest. 2010 Sep 1;120(9):3012-21

Authors: Marchetti V, Krohne TU, Friedlander DF, Friedlander M

Dramatic advances in the field of stem cell research have raised the possibility of using these cells to treat a variety of diseases. The eye is an excellent target organ for such cell-based therapeutics due to its ready accessibility, the prevalence of vasculo- and neurodegenerative diseases affecting vision, and the availability of animal models to demonstrate proof of concept. In fact, stem cell therapies have already been applied to the treatment of disease affecting the ocular surface, leading to preservation of vision. Diseases in the back of the eye, such as macular degeneration, diabetic retinopathy, and inherited retinal degenerations, present greater challenges, but rapidly emerging stem cell technologies hold the promise of autologous grafts to stabilize vision loss through cellular replacement or paracrine rescue effects.

PMID: 20811157 [PubMed - indexed for MEDLINE]

Related Articles Stem Cells for Retinal Disease: A Perspective on the Promise and Perils. Am J Ophthalmol. 2017 Jul;179:32-38 Authors: Rao RC, Dedania VS, Johnson MW Abstract PURPOSE: To summarize key concepts, as well as early safety and efficacy signals from clinical trials, for stem/progenitor cell-based interventions for retinal disease. DESIGN: Interpretive essay. METHODS: Review and synthesis of selected recent reports of stem/progenitor cell-based approaches for retinal disease, with interpretation and perspective. RESULTS: Stem/progenitor cell-based interventions represent a novel class of potential therapies for retinal diseases, such as age-related macular degeneration and inherited retinal dystrophies, aoong others. Sources include pluripotent stem cells and fetal and postnatal tissues. Two mechanisms of "rescue" have been proposed: regenerative or trophic. Although pluripotent and fetal sourced-cell types have been tested in preclinical animal models of retinal disease, many postnatal stem/progenitor cell populations currently in trial do not have preclinical safety or efficacy data. Some early-phase trials of cell therapies suggest acceptable safety profiles. Other reports, involving some types of autologous, nonocular cell sources, have been linked to severe, blinding complications. Larger trials will be needed to determine short-term and long-term safety and efficacy of these cell-based interventions. CONCLUSIONS: Stem/progenitor cell-based interventions have the potential to address blinding retinal diseases that affect hundreds of millions worldwide. Yet no Food and Drug Administration-approved stem cell therapies for retinal disease exist. Although some early-phase trial data are promising, reports of blinding complications from cell interventions remain troubling. It is paramount to apply a strong level of scientific rigor toward a well-planned, step-wise sequence of preclinical and clinical studies, to determine whether this class of potential therapies will be safe and effective for individuals with retinal diseases. PMID: 28428049 [PubMed - indexed for MEDLINE]
Related Articles Stem Cell Derived Retinal Pigment Epithelium: The Role of Pigmentation as Maturation Marker and Gene Expression Profile Comparison with Human Endogenous Retinal Pigment Epithelium. Stem Cell Rev. 2017 Jul 21;: Authors: Bennis A, Jacobs JG, Catsburg LAE, Ten Brink JB, Koster C, Schlingemann RO, van Meurs J, Gorgels TGMF, Moerland PD, Heine VM, Bergen AA Abstract In age-related macular degeneration (AMD) the retinal pigment epithelium (RPE) deteriorates, leading to photoreceptor decay and severe vision loss. New therapeutic strategies aim at RPE replacement by transplantation of pluripotent stem cell (PSC)-derived RPE. Several protocols to generate RPE have been developed where appearance of pigmentation is commonly used as indicator of RPE differentiation and maturation. It is, however, unclear how different pigmentation stages reflect developmental stages and functionality of PSC-derived RPE cells. We generated human embryonic stem cell-derived RPE (hESC-RPE) cells and investigated their gene expression profiles at early pigmentation (EP) and late pigmentation (LP) stages. In addition, we compared the hESC-RPE samples with human endogenous RPE. We used a common reference design microarray (44 K). Our analysis showed that maturing hESC-RPE, upon acquiring pigmentation, expresses markers specific for human RPE. Interestingly, our analysis revealed that EP and LP hESC-RPE do not differ much in gene expression. Our data further showed that pigmented hESC-RPE has a significant lower expression than human endogenous RPE in the visual cycle and oxidative stress pathways. In contrast, we observed a significantly higher expression of pathways related to the process adhesion-to-polarity model that is typical of developing epithelial cells. We conclude that, in vitro, the first appearance of pigmentation hallmarks differentiated RPE. However, further increase in pigmentation does not result in much significant gene expression changes and does not add important RPE functionalities. Consequently, our results suggest that the time span for obtaining differentiated hESC-RPE cells, that are suitable for transplantation, may be greatly reduced. PMID: 28730556 [PubMed - as supplied by publisher]

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