Year 4/NCE

Currently, there is no effective treatment to improve vision in patients suffering from blindness (retinal degeneration, RD). Alleviation of these diseases requires both new retinal cells including photoreceptors (the light-sensing cells in the back of the eye) and supporting retinal pigment epithelium (RPE). Our goal is to alleviate blindness to fulfill this need by transplants of stem cell derived retinal sheets from a renewable source. Human embryonic stem cells (hESCs) can be coaxed to “self-assemble” into early stages of eye development and develop into 3D retina organoids (ROs) resembling the normal retina. The hypothesis of this project is that hESC differentiated into immature retinal tissue can improve visual responses in two immunodeficient rat models of RD that do not reject human cells.  In the no-cost extension (4th year) of our research, the analysis of experiments in the two different immunodeficient RD rat models was continued and completed.

Our data about visual improvement, transplant differentiation and connectivity in the model with fast RD have been submitted to a scientific journal (review is in progress). These experiments were repeated in the second rat model of RD – with a slower rate of RD, at a disease stage when photoreceptors can no longer be rescued by RPE transplants. Data in the RCS model show that transplants improved visual acuity and visual responses in the brain at 6-10 months after transplantation.

This work was presented at scientific meetings. One paper and two reviews have been published, one is submitted and several manuscripts are close to being submitted. The laboratory has attracted many students who are interested to learn about retinal development and vision restoration. The work has led to new collaborations and an invitation to organize a retina organoid symposium at an International Scientific meeting in 2018.