Year 3/NCE

Inherited retinal degenerations result in visual loss in patients early in life. Retinitis Pigmentosa, a form of genetic disorder leading to vision loss, affects thousands of patients in US and in the state of California. The second most common gene whose mutation results in retinitis pigmentosa is RP1. How mutation in the gene result in loss of vision is not completely clear and we are working of gaining a better understanding of this by creating a 3D artificial retina in a dish. This involves using stem-cell derived eye cells grown on patterned artificial scaffolds. Thanks to the funding from CIRM, we have optimized out bioengineered scaffold design to closely match that of the retina in the human eye. In addition, we have been able to show that the stem cell generated from patient cells can be directed to form the various cells in the retina including the light sensing cells called the photoreceptor cells. These cells were then allowed to self-assemble in bioengineered 3D scaffolds also with the supporting RPE layer. This process allowed for alignment of the cells as if they are inside the eye generating a new structured artificial retina. We are using this model system to study eye diseases and we hope this will provide us with a novel tool to visualize disease processes. The artificial 3D retina has the potential for use in identifying new way to target retinal degenerations.