Open AccessTranslating Induced Pluripotent Stem Cells from Bench to Bedside: Application to Retinal Diseases
Author(s) -
Alona O Cramer,
Robert E. MacLaren
Publication year - 2013
Publication title -
current gene therapy
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 0.704
H-Index - 71
eISSN - 1875-5631
pISSN - 1566-5232
DOI - 10.2174/1566523211313020008
Subject(s) - induced pluripotent stem cell , reprogramming , transplantation , retina , clinical trial , neuroscience , stem cell , retinal , medicine , retinal degeneration , biology , bioinformatics , embryonic stem cell , cell , ophthalmology , surgery , microbiology and biotechnology , biochemistry , genetics , gene
Induced pluripotent stem cells (iPSc) are a scientific and medical frontier. Application of reprogrammed somatic cells for clinical trials is in its dawn period; advances in research with animal and human iPSc are paving the way for retinal therapies with the ongoing development of safe animal cell transplantation studies and characterization of patient- specific and disease-specific human iPSc. The retina is an optimal model for investigation of neural regeneration; amongst other advantageous attributes, it is the most accessible part of the CNS for surgery and outcome monitoring. A recent clinical trial showing a degree of visual restoration via a subretinal electronic prosthesis implies that even a severely degenerate retina may have the capacity for repair after cell replacement through potential plasticity of the visual system. Successful differentiation of neural retina from iPSc and the recent generation of an optic cup from human ESc invitro increase the feasibility of generating an expandable and clinically suitable source of cells for human clinical trials. In this review we shall present recent studies that have propelled the field forward and discuss challenges in utilizing iPS cell derived retinal cells as reliable models for clinical therapies and as a source for clinical cell transplantation treatment for patients suffering from genetic retinal disease.