title: Personalized Medicine: Cell and Gene Therapy Based on Patient-Specific iPSC-Derived Retinal Pigment Epithelium Cells.
abstract: Interest in generating human induced pluripotent stem (iPS) cells for stem cell modeling of diseases has overtaken that of patient-specific human embryonic stem cells due to the ethical, technical, and political concerns associated with the latter. In ophthalmology, researchers are currently using iPS cells to explore various applications, including: (1) modeling of retinal diseases using patient-specific iPS cells; (2) autologous transplantation of differentiated retinal cells that undergo gene correction at the iPS cell stage via gene editing tools (e.g., CRISPR/Cas9, TALENs and ZFNs); and (3) autologous transplantation of patient-specific iPS-derived retinal cells treated with gene therapy. In this review, we will discuss the uses of patient-specific iPS cells for differentiating into retinal pigment epithelium (RPE) cells, uncovering disease pathophysiology, and developing new treatments such as gene therapy and cell replacement therapy via autologous transplantation.
authors.affiliation: Department of Ophthalmology, Columbia University Medical Center, Columbia University, 160 Fort Washington Ave, Research Annex, Room 513, 10032, New York, NY, USA. yl2635@columbia.edu.
authors.forename: Yao
authors.initials: Y
authors.lastname: Li
authors.affiliation: Department of Ophthalmology, Columbia University Medical Center, Columbia University, 160 Fort Washington Ave, Research Annex, Room 513, 10032, New York, NY, USA. lc2988@columbia.edu.
authors.forename: Lawrence
authors.initials: L
authors.lastname: Chan
authors.affiliation: Columbia University College of Physicians and Surgeons, 100 Haven Ave, Apt 14B, 10032, New York, NY, USA. hvn2102@columbia.edu.
authors.forename: Huy V
authors.initials: HV
authors.lastname: Nguyen
authors.affiliation: New York Presbyterian Hospital/Columbia University Medical Center, 10032, New York, NY, USA. sht2@columbia.edu.|Department of Pathology and Cell Biology, Department of Ophthalmology, Edward Harkness Eye Institute, Columbia University, 160 Fort Washington Avenue, Research Annex, Room 509B, 10032, New York, NY, USA. sht2@columbia.edu.
authors.forename: Stephen H
authors.initials: SH
authors.lastname: Tsang
mesh_terms: D000818:Animals; D002454:Cell Differentiation / Q000235:genetics; D064987:Cell- and Tissue-Based Therapy / Q000379:methods* / Q000639:trends; D015316:Genetic Therapy / Q000379:methods* / Q000639:trends; D006801:Humans; D057026:Induced Pluripotent Stem Cells / Q000166:cytology* / Q000637:transplantation; D057285:Precision Medicine / Q000379:methods* / Q000639:trends; D012162:Retinal Degeneration / Q000235:genetics / Q000628:therapy; D055213:Retinal Pigment Epithelium / Q000166:cytology*; D014182:Transplantation, Autologous
keywords: Cell therapy; Disease modeling; Gene correction; Gene therapy; RPE; Sub-retinal transplantation; iPS