The MitoPark mouse provides a valuable model to examine the benefit of dopamine cell replacement therapy, since this animal model of mitochondrial dysfunction closely mimics the progressive degeneration of dopamine neurons in Parkinson's disease (Beal, 2010; Galter et al., 2010), and presents a much less variable phenotype than that seen with toxin models.
Recent remarkable progress in stem cell research has brought great optimism and offered the possibility to use them for developmental biology studies, disease modelling, cell replacement therapy, and tissue engineering in regenerative medicine [ 5, 10– 13].
Stem cells possess huge importance in developmental biology, disease modelling, cell replacement therapy, and tissue engineering in regenerative medicine because they have the remarkable potential for self-renewal and to differentiate into almost all the cell types in the human body.
Emerging studies of human pluripotent stem cells (hPSCs) raise new prospects for neurodegenerative disease modeling and cell replacement therapies.
In addition, most cell replacement protocols evaluated in animal models of Parkinson's disease have utilized post-mitotic, differentiated dopamine neurons as grafting material.
To address the importance of inflammation and immunosuppression for cell replacement therapy in the AAV-based model, we performed a second set of experiments.
Human pluripotent stem cells (hPSCs), namely, embryonic stem cells (ESCs) and induced pluripotent stem cells (iPSCs), with their ability of indefinite self-renewal and capability to differentiate into cell types derivatives of all three germ layers, represent a powerful research tool in developmental biology, for drug screening, disease modelling, and potentially cell replacement therapy.
Our findings demonstrate for the first time that transplantation of flow-sorted Crx-positive embryonic-stage cells generates new cone photoreceptors in two models of LCA and present a useful model for further investigation towards cone cell replacement therapy.
These cells known as induced pluripotent stem cells (iPSCs) provide a promising source of patient specific cells for cell replacement therapies as well as in vitro models for a variety of genetic diseases [79] [81].
These qualities make hPSCs potential sources of large quantities of any cell type that can then be used for research and clinical applications, including disease modeling, drug screening, and cell replacement therapies for degenerative disease.
