Gene Editing Nanomedicines to Correct Pathogenic Mutations in Retinal Pigmented Epithelium

Bioengineering advances have now made it feasible for precision gene editing directly in patient tissues with the goal of not only treatment, but of a cure. In this project, the priority is genetic disorders that inflict the eye, specifically the retinal pigmented epithelium (RPE) that surrounds the retina.

Existing state-of-the-art approaches to edit cells within human tissues have significant limitations both with respect to safety and efficacy. The nanomedicine approach of this project is anticipated to overcome these limitations and will set the foundation for a new paradigm in developing genomic medicine.

This project will use advances in biomaterials to generate nonviral, synthetic nanocarriers of CRISPR-Cas9 gene editing machinery for targeted delivery to the RPE. Such research would generally expand the types of tissues that could be edited and hence the spectrum of disease where genomic medicine could have an impact. This hypothesis will be tested to precisely edit pathogenic point mutations in vivo within transgenic mouse disease models and in patient-derived, induced pluripotent stem cell (iPSC)-based disease models.

Principal Investigator

• Krishanu Saha
  Assistant Professor of Biomedical Engineering at the Wisconsin Institute for Discovery
  

Co-Principal Investigators

• Shaoqin Sarah Gong
  Professor of Biomedical Engineering at the Wisconsin Institute for Discovery
• Bikash Pattnaik
  Assistant Professor of Pediatrics
• David Gamm
  Associate Professor of Ophthalmology and Visual Sciences and Director of the McPherson Eye Research Institute