Generating Retinal Ganglion Cells in a Dish to Study and Treat Glaucoma

Xiuqian Mu, MD, PhD
University at Buffalo, The State University of New York (Buffalo, NY)
Year Awarded:
Grant Duration:
July 1, 2016 to June 30, 2018
Award Amount:
Grant Reference ID:
Award Type:
Award Region:
US Northeastern
Xiuqian Mu, MD, PhD

Generation of Retinal Ganglion Cells by Reprograming


Glaucoma is a disease that causes vision loss and blindness in millions of people. This proposal aims to improve existing procedures and establish new ones to generate retinal ganglion cells, the cells affected in glaucoma, in a petri dish. The cells thus produced will be used to study the reasons causing glaucoma, to screen for drugs to treat it, and to develop new therapeutic strategies.


The goal of this proposal is to improve existing and to establish new procedures for efficient generation of retinal ganglion cells (RGCs), the major cell type affected in one of the most common eye diseases, glaucoma. Our experimental design is based on our recent finding that two genes are essential and sufficient for RGC differentiation during development. We will develop procedures in which these two genes, along with others if necessary, are activated in induced pluripotent stem cells  or fibroblasts (ie, cells that were themselves grown from adult human cell samples) so that these cells can be directed to differentiate into RGCs. If successful, the RGCs thus generated can be used to model the disease in vitro, to screen for drugs that can prevent their death, and to develop treatment of the disease by cell replacement.

About the Researcher

Xiuqian Mu, MD, PhD, is currently an associate professor of Ophthalmology and Biochemistry at the Jacobs School of Medicine and Biomedical Sciences, State University of New York at Buffalo (SUNY-Buffalo). Dr. Mu obtained his MD from Qingdao Medical College (now Qingdao University School of Medicine) located in Qingdao, China, and PhD in biochemistry and molecular biology from Peking Union Medical College located in Beijing. He subsequently received training in molecular biology and developmental biology at the National Institutes of Health and The University of Texas MD Anderson Cancer Center. He started his own lab at SUNY-Buffalo in 2008. Dr. Mu’s major research interests are the molecular and genetic mechanisms underlying the generation of the diverse cell types in the retina. He has made major contributions to our understanding of the gene regulatory network controlling the formation of retinal ganglion cells. His current research focuses are on both retinal cell fate determination and in vitro differentiation of retinal neurons for therapeutic purposes.

Personal Story

Since I was young, I have always been curious about the natural world and eager to learn how its inner workings drive various natural processes. Therefore, becoming a developmental biologist and study how our body forms during embryogenesis came naturally, and the body part I chose to study is the eye, or to be more precise, the retina.

The retina fascinates me since its seemingly simple but elegant structure allows us to see the various aspect of the visual world. The function of the retina is achieved by the many different retinal cell types. Whereas this by itself is fascinating, there is another side to the retina. Defects or damages to the retina can lead to devastating vision loss and even blindness. Therefore, bridging the basic research of retinal development with relevant retinal diseases became a logical and necessary transition in my research.

Research on retinal development has reached the point where we can design new strategies to study and treat retinal diseases. We propose to use what we learned about the process of retinal ganglion cell (RGC) differentiation during development and develop more efficient procedures to produce these cells in a petri dish. The key finding we rely on, which was made by our lab and published recently, is that just two transcription factors (proteins that turn genes on and off) are sufficient for the generation of RGCs during embryo development. We therefore reasoned that these two factors can be used in vitro to generate RGCs from stem cells. Obviously this will be different from the normal developmental process, and we need to figure out the right conditions to achieve the goal, which is what we will do with this grant. Our ultimate goal is to use the generated RGCs to model the disease in vitro, to screen for drugs that can prevent their death, and to directly treat the disease by cell replacement.

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