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      Personalized Medicine: Cell and Gene Therapy Based on Patient-Specific iPSC-Derived Retinal Pigment Epithelium Cells.

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          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.

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          Author and article information

          Journal
          Adv. Exp. Med. Biol.
          Advances in experimental medicine and biology
          Springer Nature
          0065-2598
          0065-2598
          2016
          : 854
          Affiliations
          [1 ] 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.
          [2 ] 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.
          [3 ] Columbia University College of Physicians and Surgeons, 100 Haven Ave, Apt 14B, 10032, New York, NY, USA. hvn2102@columbia.edu.
          [4 ] New York Presbyterian Hospital/Columbia University Medical Center, 10032, New York, NY, USA. sht2@columbia.edu.
          [5 ] 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.
          Article
          10.1007/978-3-319-17121-0_73
          26427458

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