Mitochondrial Neurogastrointestinal Encephalomyopathy Caused by Thymidine Phosphorylase Enzyme Deficiency: From Pathogenesis to Emerging Therapeutic Options

Rana Yadak*, Peter Sillevis Smitt, Marike W. van Gisbergen, Niek P. van Til, Irenaeus F. M. de Coo*

*Corresponding author for this work

Research output: Contribution to journalArticleAcademicpeer-review

27 Citations (Web of Science)
Original languageEnglish
Article number31
Number of pages14
JournalFrontiers in Cellular Neuroscience
Volume11
DOIs
Publication statusPublished - 15 Feb 2017

Keywords

  • mitochondrial neurogastrointestinal encephalomyopathy
  • MNGIE
  • thymidine phosphorylase
  • metabolic disease
  • HSCT
  • HSCGT
  • lentiviral vector
  • CELL GENE-THERAPY
  • CHRONIC GRANULOMATOUS-DISEASE
  • LONG-TERM SAFETY
  • LENTIVIRAL VECTOR
  • MNGIE SYNDROME
  • DNA DEPLETION
  • REPLACEMENT THERAPY
  • HEMATOPOIETIC-CELLS
  • MURINE MODEL
  • BONE-MARROW

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