A New Model of a Human Retina in a Dish to Study AMD

Maria Valeria Canto-Soler, PhD
University of Colorado (Denver, CO)
Year Awarded:
2016
Grant Duration:
July 1, 2016 to June 30, 2019
Disease:
Macular Degeneration
Award Amount:
$160,000
Grant Reference ID:
M2016119
Award Type:
Standard
Award Region:
US Northwestern

The Helen Juanita Reed Award for Macular Degeneration Research.

Maria Valeria Canto-Soler, PhD

A Novel 3D Human Mini-Retina Model for Early AMD

Summary

Our studies aim at uncovering the mechanisms that trigger age-related macular degeneration (AMD), with the purpose of contributing to the development of therapeutic treatments for this condition. Within this context, our goal is to develop the first “human retina in a dish” model derived from stem cells that would allow us to recreate the cellular physical and functional interactions that occur in the retina of a patient suffering from early stages of AMD. This human AMD model will provide a unique biological system to investigate the initial triggers leading to this disease and to develop treatments to stop its progress.

Details

Our ability to find effective treatments for AMD is critically limited by the lack of understanding of the early mechanisms that trigger this disease. To overcome this problem, we are developing a “human retina in a dish” model derived from stem cells with the purpose of recreating the disease mechanisms that occur in the retina of a patient suffering from early stages of AMD.

Abnormal interactions between the light-sensitive cells of the retina, the photoreceptors, and the underlying retinal pigment epithelial cells, commonly referred as RPE, are at the center of the mechanisms that trigger AMD, but the specific characteristics of these abnormal interactions remain mostly unknown. In order to gain a better understanding of these interactions it is necessary to develop a human model capable of mimicking what occurs in the diseased retina and that can be studied in the laboratory.

To achieve that goal, we are using a specific type of stem cells called induced pluripotent stem cells, also referred as iPS cells. Human iPS cells are generated from adult cells, for example, skin or blood cells, obtained through a routine clinical biopsy. In the laboratory, these cells are then forced to express a group of specific genes that instruct them to “go back in time” and acquire a “pluripotent stem cell” status. These cells can then be re-directed to differentiate into any cell type of the adult body and used to study specific diseases and to develop therapeutic treatments. Using human iPS cells, we have recently succeeded in generating three-dimensional human retinal tissue containing functional photoreceptors and associated RPE cells. Building upon this success, we are now further improving the system in order to efficiently recreate the physical and functional interactions that occur between photoreceptors and RPE cells in normal conditions, as well as at the early stages of AMD. We expect that this AMD human model will provide a unique biological system to investigate the initial triggers leading to this disease and to develop treatments to stop its progress.

About the Researcher

Maria Valeria Canto-Soler, PhD, is an assistant professor of ophthalmology, and director of the Retinal Degenerations Research Center, at the Wilmer Eye Institute, Johns Hopkins University School of Medicine. Dr. Canto-Soler completed her PhD in Biomedical Sciences at Austral University in Buenos Aires, Argentina and a postdoctoral fellowship at the Wilmer Eye Institute, Johns Hopkins University, Baltimore.

Her research focuses on two main, highly complementary areas: the mechanisms that control retinal progenitor cell differentiation and the use of human induced pluripotent stem cell (iPSC) technology to model normal and diseased conditions of the retina. Within this framework, her lab has recently established a strategy to direct hiPSC to differentiate into three-dimensional light-sensitive retinal tissue in vitro; she and her team are now using these retinal organoids to study the mechanisms underlying retinal degenerative diseases such as age-related macular degeneration (AMD), and to develop therapeutic treatments for these conditions.

Her work has been supported by the National Institutes of Health, the Maryland Stem Cell Research Fund, Research to Prevent Blindness, and the Falk Medical Research Trust, among others. She has received several national and international awards including the Alcon Research Institute Young Investigator Award, and the William & Mary Greve Special Scholar Award from Research to Prevent Blindness. She has also recently been named Outstanding Personality by the Council of the Autonomous City of Buenos Aires, Argentina, in recognition to her scientific contributions.

Personal Story

At the time I was starting my own independent research lab, I decided I wanted my research program to have an important translational component in order to contribute in a significant way to finding treatments for blinding diseases. As I became more and more aware of the impact of age-related macular degeneration (AMD) on the lives of people affected by this disease and their families, I decided to make every possible effort to develop a specific line of research focused on AMD. The award from the BrightFocus foundation is making it possible to begin to realize this goal. It is because of this that I want to express my most wholehearted gratitude to all BrightFocus donors that so generously support the work of those of us who are working day after day in trying to find cures for this debilitating disease.

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