Research in the Stem Cell and Retinal Regeneration Program at the Gavin Herbert Eye Institute at UC Irvine is directed toward the development of novel therapies for retinal degenerative diseases, specifically retinitis pigmentosa (RP) and age related macular degeneration (AMD). The primary approach being pursued is the transplantation of human retinal progenitor cells (hRPCs), a stem-like cell found in the immature retina. These cells can be isolated from tissue and propagated in culture.

Studies in animal models of retinal disease have shown that RPCs are capable of replacing retinal cell types, while also rescuing host cells that would otherwise die as a result of the disease process. Another important characteristic of these cells is that they do not tend to create an immune reaction when transplanted to unrelated recipients within the same species. RPCs are well tolerated as allografts to the retina and do not require immune suppression.

Current efforts are directed at preclinical studies in which clinical grade human cells are grown and tested for use in future trials, pending FDA approval. Work in the Klassen laboratory is currently investigating the molecular mechanisms underlying therapeutic efficacy. Active translational projects being carried out with collaborating laboratories at Cedars Sinai Medical Center, UCSB and UC Davis include proof of concept studies, GMP cell manufacturing, and in vivo toxicology. The work is progressing rapidly towards the clinic and has the active support of a Disease Team Therapy Development Award from the California Institute of Regenerative Medicine (CIRM).

Posted April 2013