Roles for RPE-specific MicroRNAs in Age-related Retinal Diseases
Detailed Non-Technical Summary
The identification of AMD-related miRNAs will be important for improving diagnosis. miRNAs are stable molecules that can be readily introduced into cells or, alternatively, whose activity can be blocked. The information obtained in course of this study on their role can be utilized to develop approaches for the treatment of AMD. Furthermore, identification of miRNAs activity in RPE derived from ES cells could contribute to the design of cell-replacement therapy in the RPE.
To learn more about the miRNAs that are involved with AMD, Dr. Ashery-Padan’s group is using a unique set of mice in which miRNAs are not expressed during RPE development. Analyses of the appearance and function of cells and molecular changes in the genetic models have already revealed that miRNAs are indispensable for the full activity and survival of the RPE. Specific mutations in selected miRNAs are being produced and tested in mice and in RPE derived from stem cells to determine the specific roles of these developmental regulators in the physiology of the RPE.
Understanding the mechanisms by which RPE cell fate is normally acquired and maintained, and the cross talk between the RPE and adjacent cell types of the choroid and retina, is pivotal for understanding RPE dysfunctions, and for design of future RPE cell-replacement therapies. This study, supported by the BrightFocus foundation, combines functional in vivo studies with stem cell technologies aimed at deciphering the roles of miRNAs in RPE function and in the development of AMD. The team’s discoveries on miRNA’s function during normal development of the RPE may be applicable for future development of cell-based therapies for retinal degenerations, like AMD.