Neural Regeneration Research ›› 2021, Vol. 16 ›› Issue (9): 1783-1785.doi: 10.4103/1673-5374.306076

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The therapeutic potential of nerve growth factor combined with blood-brain barrier modulation by focused ultrasound for neurodegenerative disorders 

Kristiana Xhima*, Isabelle Aubert*    

  1. Department of Laboratory Medicine and Pathobiology, University of Toronto, Toronto, ON, Canada (Xhima K, Aubert I)
    Hurvitz Brain Sciences Research Program, Sunnybrook Research Institute, Toronto, ON, Canada (Xhima K, Aubert I)
    Biological Sciences, Sunnybrook Research Institute, Toronto, ON, Canada (Xhima K, Aubert I)
  • Online:2021-09-15 Published:2021-02-05
  • Contact: Kristiana Xhima, PhD, kristiana.xhima@mail.utoronto.ca; Isabelle Aubert, PhD, isabelle.aubert@utoronto.ca.
  • Supported by:
    We thank Hang Yu Lin for figure illustration. 

    This work was supported by the Canadian Institutes of Health Research (grant FRN 137064 to IA), the FDC Foundation, the WB Family Foundation, Gerald and Carla Connor, the Weston Brain Institute (TR130117 to IA); a Frederick Banting and Charles Best Canada Graduate Scholarship (GSD 152271 to KX). This research was undertaken, in part, from funding through the Canada Research Chairs program (to IA). 

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Abstract: Nerve growth factor (NGF) is a neurotrophic factor critical for cholinergic neuronal survival, phenotypic maintenance and plasticity in the mammalian brain. NGF has been implicated in the pathogenesis of neurodegenerative disorders, with direct administration of NGF into the brain capable of facilitating neuroprotection and repair. To date, NGF has been proposed as a potential therapy for Alzheimer’s disease (AD) and Parkinson’s disease due to its regenerative effects on cholinergic neurons located in the basal forebrain and striatum, respectively. In other neurodegenerative diseases, including progressive supranuclear palsy, amyotrophic lateral sclerosis (ALS), vascular dementia, Lewy body dementia and frontotemporal lobar dementia, emerging evidence has revealed the impact of cholinergic dysfunction on clinical outcomes, thereby supporting the therapeutic potential of NGF. In this perspective, we will review the current challenges of NGF-related therapy for clinical use and address the relevance of magnetic resonance imaging (MRI)-guided focused ultrasound (MRIgFUS)-induced blood-brain barrier (BBB) permeability enhancement for the delivery of NGF-related compounds and brain repair.