title: Stem cells for investigation and treatment of inherited retinal disease.
abstract: Vision is the most important human sense. It facilitates every major activity of daily living ranging from basic communication, mobility and independence to an appreciation of art and nature. 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 some type of drug, gene or cell-based transplantation approach. In this review, we will discuss the current literature pertaining to retinal transplantation. We will focus on the use of induced pluripotent stem cells for interrogation of disease pathophysiology, analysis of drug and gene therapeutics and as a source of autologous cells for cell replacement.
authors.affiliation: Stephen A. Wynn Institute for Vision Research and.
authors.forename: Budd A
authors.initials: BA
authors.lastname: Tucker
authors.affiliation: Stephen A. Wynn Institute for Vision Research and.
authors.forename: Robert F
authors.initials: RF
authors.lastname: Mullins
authors.affiliation: Stephen A. Wynn Institute for Vision Research and Howard Hughes Medical Institute, Department of Ophthalmology and Visual Science, Carver College of Medicine, University of Iowa, Iowa city, IA, USA edwin-stone@uiowa.edu.
authors.forename: Edwin M
authors.initials: EM
authors.lastname: Stone
mesh_terms: D000818:Animals; D064112:Clustered Regularly Interspaced Short Palindromic Repeats / Q000235:genetics; D004195:Disease Models, Animal; D016678:Genome; D006801:Humans; D057026:Induced Pluripotent Stem Cells / Q000637:transplantation*; D008268:Macular Degeneration / Q000628:therapy*; D012162:Retinal Degeneration / Q000628:therapy*; D012174:Retinitis Pigmentosa / Q000628:therapy*; D033581:Stem Cell Transplantation