Preclinical Studies of Induced Pluripotent Stem Cell-Derived Astrocyte Transplantation in ALS

Abstract

We have made substantial progress in obtaining skin biopsies with subsequent fibroblast cultures of a number of sporadic ALS, familial ALS, and control subjects in the last year. We have created numerous iPS cell lines from these patient samples and have characterized their long-term differentiation into astroglia. We now have data from the in vivo transplantation of these astroglial progenitors into rat spinal cords. We have characterized several control and 1 ALS cell line with regard to their survival, differentiation, migration, and phenotypic effects on the host animals. Our data demonstrate that there are differences amongst the cell lines with regard to these properties but we have not appreciated obvious differences with regard to ALS cells when compared with control astroglial cells. Finally, we have demonstrated, in preparation for our therapeutic approaches, that these glial progenitor cells continue to mature into astrocytes following transplantation but that their survival is significantly reduced when compared with previous studies using human fetally-derived glial restricted precursors.

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Document Details

Document Type
Technical Report
Publication Date
Oct 01, 2013
Accession Number
ADA590384

Entities

People

  • Hongjun Song
  • Nicholas J Maragakis

Organizations

  • Johns Hopkins University

Tags

DTIC Thesaurus Topics

  • Antigens
  • Astrocytes
  • Cell Line
  • Cell Physiological Processes
  • Cells
  • Data Analysis
  • Diseases And Disorders
  • Gene Expression
  • Genetics
  • Intercellular Junctions
  • Nervous System
  • Neurodegeneration
  • Neurodegenerative Diseases
  • Neurons
  • Spinal Cord
  • Stem Cells
  • Therapy

Fields of Study

  • Biology

Readers

  • Immunology and Pathology

Technology Areas

  • Biotechnology