title: Personalized therapeutic strategies for patients with retinitis pigmentosa.
abstract: BACKGROUND
  Retinitis pigmentosa (RP) encompasses many different hereditary retinal degenerations that are caused by a vast array of different gene mutations and have highly variable disease presentations and severities. This heterogeneity poses a significant therapeutic challenge, although an answer may eventually be found through two recent innovations: induced pluripotent stem cells (iPSCs) and clustered regularly interspaced short palindromic repeats (CRISPR)/Cas genome editing.
  
  
  METHODS
  This review discusses the wide-ranging applications of iPSCs and CRISPR-including disease modelling, diagnostics and therapeutics - with an ultimate view towards understanding how these two technologies can come together to address disease heterogeneity and orphan genes in a novel personalized medicine platform. An extensive literature search was conducted in PubMed and Google Scholar, with a particular focus on high-impact research published within the last 1 - 2 years and centered broadly on the subjects of retinal gene therapy, iPSC-derived outer retina cells, stem cell transplantation and CRISPR/Cas gene editing.
  
  
  CONCLUSIONS
  For the retinal pigment epithelium, autologous transplantation of gene-corrected grafts derived from iPSCs may well be technically feasible in the near future. Photoreceptor transplantation faces more significant unresolved technical challenges but remains an achievable, if more distant, goal given the rapid pace of advancements in the field.
authors.affiliation: Columbia University, College of Physicians and Surgeons , 50 Haven Ave, Box #123, Bard Hall, New York, NY 10032 , USA.
authors.forename: Andrew
authors.initials: A
authors.lastname: Zheng
authors.forename: Yao
authors.initials: Y
authors.lastname: Li
authors.forename: Stephen H
authors.initials: SH
authors.lastname: Tsang
mesh_terms: D064112:Clustered Regularly Interspaced Short Palindromic Repeats / Q000235:genetics; D015316:Genetic Therapy / Q000379:methods* / Q000639:trends; D006801:Humans; D057026:Induced Pluripotent Stem Cells / Q000637:transplantation*; D057285:Precision Medicine / Q000379:methods* / Q000639:trends; D055213:Retinal Pigment Epithelium / Q000473:pathology; D012174:Retinitis Pigmentosa / Q000175:diagnosis / Q000235:genetics* / Q000628:therapy*; D033581:Stem Cell Transplantation / Q000379:methods* / Q000639:trends
keywords: clustered regularly interspaced short palindromic repeats; gene therapy; induced pluripotent stem cells; retinal degenerations