Pluripotent Stem Cells
LCA Patient-Derived IPS Cells
WARF: P250286US01
Inventors: Bikash Pattnaik
The Invention
A UW-Madison researcher has access to LCA 16 patient derived IPS cells. These cells will be differentiated into retinal pigment epithelial cells. The RPE cells will be dosed with escalating amount of the gene therapy vehicle to determine the necessary dose to correct the underlying genetic defect.