Successful Cell Transplantation Gives Hope for New Glaucoma Treatment

Learn about the first successful attempt to transplant lab-grown retinal ganglion cells (RGCs) into the eyes of mice to find a cure for glaucoma.

What: Researchers have reported the first successful attempt to transplant lab-grown retinal ganglion cells (RGCs) derived from induced pluripotent stem cells (iPSCs) into the eyes of mice. Based on this work in animal models, they estimate that humans with late-stage glaucoma could begin receiving vision-restoring transplants in the next 10 years.

Where: Oswald J, et al. Transplantation of miPSC/mESC-Derived Retinal Ganglion Cells Into Healthy and Glaucomatous Retinas. Molecular Therapy: Methods & Clinical Development, June, 2021.

BrightFocus Connection: This research was supported by a 2020 BrightFocus National Glaucoma Research grant awarded to Petr Baranov, MD, PhD, the study’s lead author, at the Schepens Eye Research Institute at Harvard Medical School (see link to grant profile below).

Why It Is Important: A collaborative team of researchers have made the first successful attempt to transplant lab-grown RGCs derived from stem cells into the eyes of mice. RGCs are the nerve cells that transmit visual information from the eye to the brain. When a critical number of these cells becomes destroyed by glaucoma, vision loss results.

Interestingly, they found that the transplanted donor cells, which were grown in petri dishes, integrated themselves into the diseased retinas of recipient mice and survived there for up to a year. Induced pluripotent stem cells (iPSCs) are specially cultured stem cells obtained from adult human cells that can be programmed to become one of many cell types – in this case, RGCs.

Moreover, researchers re-isolated the transplanted donor cells from the host retina both one day and one week after transplant and studied their gene transcription by a technique called single-cell RNA sequencing. The results showed that, once transplanted, the donor cells were able to express markers that indicated that they can connect and communicate with other retinal nerve cells in the host tissue. Identifying these markers will help researchers better understand ways to make the transplanted cells function properly and improve vision.

According to the authors, practical applications of RGC transplantation in humans depends on the availability of a robust, scalable source of human RGCs for transplant. And because the eye rarely rejects transplanted cells, transplant candidates would not need to receive RGCs grown from their own stem cells. Instead, human RGCs could be grown from iPSCs provided by a universal donor and stored frozen in a cell bank to be thawed and transplanted when needed.