Gene-modified bone marrow-derived stem cells: an attractive gene delivery system in inherited retinal disorders

Abstract

Objective: The issues of BMSC plasticity in the ocular system are reviewed. The therapeutic benefit of BMSC per se and gene-modified BMSC (as a vehicle for gene therapy) in inherited retinal disorders is discussed. Result: Recently, it was convincingly demonstrated that subpopulation of BMSC could restore the retinal function and structure by promoting/preserving the retinal vascularization rather than differentiating to retinal neurons/glia. In animal models of brain disorders, such as Parkinson s disease, BMSCs has been demonstrated as a promising vehicle for the delivery of therapeutic genes. Although little is known about the therapeutic potential of gene-modified BMSC in the ocular system, long-term engraftment and stable gene expression of gene-modified BMSCs have been shown in rodent retinas. Conclusion: The experimental evidences published over the past decade imply a possibility to use BMSC as a gene delivery system which can be simply transplanted and provide a stable long-term gene expression in the retina.