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.
