Stem Cell Therapy for Inherited Retinal Disease and ARMD

Stem cells hold the promise of being able to restore vision for many types of retinal degenerative diseases, including inherited retinal diseases like retinitis pigmentosa and age-related macular degeneration. However, there are still many challenges such as low
survival and integration of transplanted cells.

In a new study funded through a FBC Restore Vision 20/20 grant, Dr. William Beltran (University of Pennsylvania) and Dr. David Gamm (University of Wisconsin) have overcome some of these significant challenges, moving this research one step closer to a potential clinical trial.

Published in the journal Stem Cell Reports, the researchers developed a robust transplantation methodology which they tested in the eyes of dogs. This involved developing a new way to inject cells under the retina and an immunosuppression regimen to prevent rejection of transplanted cells. Following this procedure, the team was excited to see that transplanted cells not only survived in the retina but also appeared to form connections with other remaining cells. This last step is crucial if new transplanted photoreceptors are going to be able to pass light signals on to other cells in the retina and finally to the brain to form images.

This FBC-funded project is ongoing, and the next stage is to continue to optimize the procedure and test if transplanted cells restore some vision in the animal model.

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