Engineered BDNF producing cells as a potential treatment for neurologic disease

Peter Deng, Johnathon D. Anderson, Abigail S. Yu, Geralyn Annett, Kyle D. Fink, Jan A. Nolta

Research output: Contribution to journalReview articlepeer-review

23 Scopus citations


ABSTRACT: Introduction: Brain-derived neurotrophic factor (BDNF) has been implicated in wide range of neurological diseases and injury. This neurotrophic factor is vital for neuronal health, survival, and synaptic connectivity. Many therapies focus on the restoration or enhancement of BDNF following injury or disease progression. Areascovered: The present review will focus on the mechanisms in which BDNF exerts its beneficial functioning, current BDNF therapies, issues and potential solutions for delivery of neurotrophic factors to the central nervous system, and other disease indications that may benefit from overexpression or restoration of BDNF. Expertopinion: Due to the role of BDNF in neuronal development, maturation, and health, BDNF is implicated in numerous neurological diseases making it a prime therapeutic agent. Numerous studies have shown the therapeutic potential of BDNF in a number of neurodegenerative disease models and in acute CNS injury, however clinical translation has fallen short due to issues in delivering this molecule. The use of MSC as a delivery platform for BDNF holds great promise for clinical advancement of neurotrophic factor restoration. The ease with which MSC can be engineered opens the door to the possibility of using this cell-based delivery system to advance a BDNF therapy to the clinic.

Original languageEnglish (US)
Pages (from-to)1025-1033
Number of pages9
JournalExpert Opinion on Biological Therapy
Issue number8
StatePublished - Aug 2 2016


  • BDNF
  • Gene Therapy
  • Huntington’s disease
  • Mesenchymal Stem Cell
  • Neurodegeneration

ASJC Scopus subject areas

  • Pharmacology
  • Drug Discovery
  • Clinical Biochemistry


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