VEGF delivery with retrogradely transported lentivector prolongs survival in a mouse ALS model

By M. Azzouz, G. S. Ralph, E. Storkebaum, Lucy E. Walmsley, K. Mitrophanous, S. Kingsman, Peter Carmeliet, N. Mazarakis. Nature, 2004

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Current hypotheses for the underlying biology of amyotrophic lateral sclerosis (2009, background): “A neuroprotective role for VEGF is supported by studies demonstrating delayed motor neuron disease onset and progression in mutant SOD1 models with genetic overexpression of VEGF,(34) intracerebroventricular administration of VEGF,(35) or intramuscular delivery of VEGF-expressing lentiviral vectors.”
Intravascular AAV9 preferentially targets neonatal-neurons and adult-astrocytes in CNS (2008, background): “Recent work in rodent models of SMA and ALS have demonstrated the potential of gene delivery using viruses that undergo retrograde transport after intramuscular injection, and this approach is currently being explored for clinical trial”
Neurovascular pathways to neurodegeneration in Alzheimer’s disease and other disorders (2011, background): “or intramuscular administration of a VEGF-expressing lentiviral vector that is transported retrogradely to motor neurons in SOD1 G93A mic”