title: Modeling retinal dystrophies using patient-derived induced pluripotent stem cells.
abstract: Retinal degenerative disease involving photoreceptor (PR) cell loss results in permanent vision loss and often blindness. Generation of induced pluripotent stem cell (iPSC)-derived retinal cells and tissues from individuals with retinal dystrophies is a relatively new and promising method for studying retinal degeneration mechanisms in vitro. Recent advancements in strategies to differentiate human iPSCs (hiPSCs) into 3D retinal eyecups with a strong resemblance to the mature retina raise the possibility that this system could offer a reliable model for translational drug studies. However, despite the potential benefits, there are challenges that remain to be overcome before stem-cell-derived retinal eyecups can be routinely used to model human retinal diseases. This chapter will discuss both the potential of these 3D eyecup approaches and the nature of some of the challenges that remain.
authors.affiliation: Department of Ophthalmology, The Johns Hopkins University School of Medicine, Smith Building Rm 3029, 400 N Broadway, Baltimore, MD, USA, kwahlin1@jhmi.edu.
authors.forename: Karl J
authors.initials: KJ
authors.lastname: Wahlin
authors.forename: Julien
authors.initials: J
authors.lastname: Maruotti
authors.forename: Donald J
authors.initials: DJ
authors.lastname: Zack
mesh_terms: D018929:Cell Culture Techniques; D055808:Drug Discovery; D006801:Humans; D020419:Photoreceptor Cells, Vertebrate / Q000166:cytology*; D039904:Pluripotent Stem Cells / Q000166:cytology*; D012162:Retinal Degeneration / Q000188:drug therapy / Q000235:genetics / Q000473:pathology*; D058499:Retinal Dystrophies / Q000188:drug therapy / Q000235:genetics / Q000473:pathology*; D055213:Retinal Pigment Epithelium / Q000166:cytology*