title: Patient-specific induced pluripotent stem cells (iPSCs) for the study and treatment of retinal degenerative diseases.
abstract: Vision is the sense that we use to navigate the world around us. Thus it is not surprising that blindness is one of people's most feared maladies. Heritable diseases of the retina, such as age-related macular degeneration and retinitis pigmentosa, are the leading cause of blindness in the developed world, collectively affecting as many as one-third of all people over the age of 75, to some degree. For decades, scientists have dreamed of preventing vision loss or of restoring the vision of patients affected with retinal degeneration through drug therapy, gene augmentation or a cell-based transplantation approach. In this review we will discuss the use of the induced pluripotent stem cell technology to model and develop various treatment modalities for the treatment of inherited retinal degenerative disease. We will focus on the use of iPSCs for interrogation of disease pathophysiology, analysis of drug and gene therapeutics and as a source of autologous cells for cell transplantation and replacement.
authors.affiliation: Stephen A. Wynn Institute for Vision Research, Department of Ophthalmology and Visual Sciences, University of Iowa, Iowa City, IA, USA.
authors.forename: Luke A
authors.initials: LA
authors.lastname: Wiley
authors.affiliation: Stephen A. Wynn Institute for Vision Research, Department of Ophthalmology and Visual Sciences, University of Iowa, Iowa City, IA, USA.
authors.forename: Erin R
authors.initials: ER
authors.lastname: Burnight
authors.affiliation: Stephen A. Wynn Institute for Vision Research, Department of Ophthalmology and Visual Sciences, University of Iowa, Iowa City, IA, USA.
authors.forename: Allison E
authors.initials: AE
authors.lastname: Songstad
authors.affiliation: Stephen A. Wynn Institute for Vision Research, Department of Ophthalmology and Visual Sciences, University of Iowa, Iowa City, IA, USA.
authors.forename: Arlene V
authors.initials: AV
authors.lastname: Drack
authors.affiliation: Stephen A. Wynn Institute for Vision Research, Department of Ophthalmology and Visual Sciences, University of Iowa, Iowa City, IA, USA.
authors.forename: Robert F
authors.initials: RF
authors.lastname: Mullins
authors.affiliation: Stephen A. Wynn Institute for Vision Research, Department of Ophthalmology and Visual Sciences, University of Iowa, Iowa City, IA, USA; Howard Hughes Medical Institute, University of Iowa, Iowa City, IA, USA.
authors.forename: Edwin M
authors.initials: EM
authors.lastname: Stone
authors.affiliation: Stephen A. Wynn Institute for Vision Research, Department of Ophthalmology and Visual Sciences, University of Iowa, Iowa City, IA, USA. Electronic address: budd-tucker@uiowa.edu.
authors.forename: Budd A
authors.initials: BA
authors.lastname: Tucker
mesh_terms: D000818:Animals; D004195:Disease Models, Animal; D006801:Humans; D057026:Induced Pluripotent Stem Cells / Q000637:transplantation*; D009154:Mutation; D012162:Retinal Degeneration / Q000235:genetics / Q000628:therapy*
keywords: CRISPR-based genome editing; Cell transplantation; Gene therapy; Induced pluripotent stem cells; Retinal degeneration