Grant Award Details
To generate CRISPR-Cas9 gene-edited retinal reporter PSCs to develop systematic and quantifiable methods to improve retinal differentiation.
Grant Application Details
- Microenvironment based optimization of retinal induction using CRISPR-CAS9 reporter pluripotent stem cells as an expandable source of retinal progenitors and photoreceptors.
To increase the efficiency of generating pure retinal progenitor cultures for use in transplantation and to probe general aspects of retinal development.
Our methods could increase the efficiency of obtaining transplantable patient specific induced pluripotent stem cell derived retinal cells for the treatment of blindness through cell replacement.
Major Proposed Activities
- Make stem cell based early retinal reporters as tools for optimization.
- Optimize cell differentiation, focusing on hypoxia and diffusible factors.
California is already a leader in retinal stem cell research with great emphasis placed on transplantation of fetal retinal progenitors. These cells show great promise as a short term tool for cell replacement. The ultimate goal will be to use a patients own cells for cell replacement and for that to happen PSC technology needs to be further developed. Our work will bolster the work of other California scientists by providing them with new and improved methods for obtaining transplantable cells.