| Descripción: |
Background: In spite of the therapeutic potential in several experimental models of inherited retinal degeneration, gene replacement therapy remains limited by technical/surgical difficulties of therapeutic gene delivery, and it seems to be effective only in young/neonatal animals. Bone marrow-derived stem cell (BMSC)-based therapy has been proposed as an alternative for treatment of retinal disorders. BMSC is a rich, inexhaustible source of potentially autologous adult stem cells. Despite numerous experimental evidences of their plasticity and therapeutic potential, the possibility that BMSC can populate the retina and differentiate into functionally retinal neurons and/or glia remains controversial.
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 Parkinsons 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. |
