Department of Ophthalmology and Visual Sciences
University of Iowa, Institute for Vision Research Center
Iowa City, Iowa

BASIC RESEARCH PROJECT
Development of mutation-independent gene therapy approaches for CEP290-LCA

Research Interests
Mutations in the CEP290 gene are a leading cause of Leber congenital amaurosis (LCA), a hereditary retinal dystrophy that causes severe vision loss in early childhood. Although gene therapy has shown promise for treating inherited retinal degenerations, CEP290LCA remains as a challenging target because of the gene’s large size and diverse mutations found throughout the gene. Our interest is aimed at overcoming the limitation of current gene therapy approaches and developing a novel strategy to treat CEP290-LCA by developing a mutation-independent, generic gene therapy vector for the gene.  Successful completion of this study will not only move us forward to the cure of CEP290-LCA but also provide a framework for the development of gene therapy vectors targeting other large gene associated genetic diseases.