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Advances in reprogramming-based study of neurologic disorders

Academic Article
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Overview

authors

  • Nityanandam, A.
  • Baldwin, Kristin

publication date

  • June 2015

journal

  • Stem Cells and Development  Journal

abstract

  • The technology to convert adult human non-neural cells into neural lineages, through induced pluripotent stem cells (iPSCs), somatic cell nuclear transfer, and direct lineage reprogramming or transdifferentiation has progressed tremendously in recent years. Reprogramming-based approaches aimed at manipulating cellular identity have enormous potential for disease modeling, high-throughput drug screening, cell therapy, and personalized medicine. Human iPSC (hiPSC)-based cellular disease models have provided proof of principle evidence of the validity of this system. However, several challenges remain before patient-specific neurons produced by reprogramming can provide reliable insights into disease mechanisms or be efficiently applied to drug discovery and transplantation therapy. This review will first discuss limitations of currently available reprogramming-based methods in faithfully and reproducibly recapitulating disease pathology. Specifically, we will address issues such as culture heterogeneity, interline and inter-individual variability, and limitations of two-dimensional differentiation paradigms. Second, we will assess recent progress and the future prospects of reprogramming-based neurologic disease modeling. This includes three-dimensional disease modeling, advances in reprogramming technology, prescreening of hiPSCs and creating isogenic disease models using gene editing.

subject areas

  • Animals
  • Cell Transplantation
  • Cellular Reprogramming
  • Genetic Therapy
  • Humans
  • Induced Pluripotent Stem Cells
  • Neurodegenerative Diseases
  • Regenerative Medicine
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Identity

PubMed Central ID

  • PMC4440984

International Standard Serial Number (ISSN)

  • 1547-3287

Digital Object Identifier (DOI)

  • 10.1089/scd.2015.0044

PubMed ID

  • 25749371
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Additional Document Info

start page

  • 1265

end page

  • 1283

volume

  • 24

issue

  • 11

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