1. Oxford BioMedica plc, The Oxford Science Park, Medawar Centre, Oxford OX4 4GA, UK
2. Center for Transgene Technology and Gene Therapy, Flanders Interuniversity Institute for Biotechnology, University of Leuven, Campus Gasthuisberg, Herestraat 49, B-3000 Leuven, Belgium
3. Present address: Oxxon Pharmaccines, Florey House, 3 Robert Robinson Avenue, Oxford Science Park, Oxford, OX4 4GP, UK

Correspondence to: Mimoun Azzouz¹Nicholas D. Mazarakis¹ Email: m.azzouz@oxfordbiomedica.co.uk
Email: n.mazarakis@oxfordbiomedica.co.uk

Abstract

Amyotrophic lateral sclerosis (ALS) causes adult-onset, progressive motor neuron degeneration in the brain and spinal cord, resulting in paralysis and death three to five years after onset in most patients¹. ALS is still incurable, in part because its complex aetiology remains insufficiently understood. Recent reports have indicated that reduced levels of vascular endothelial growth factor (VEGF), which is essential in angiogenesis and has also been implicated in neuroprotection^{2, 3, 4}, predispose mice and humans to ALS^{5, 6}. However, the therapeutic potential of VEGF for the treatment of ALS has not previously been assessed. Here we report that a single injection of a VEGF-expressing lentiviral vector into various muscles delayed onset and slowed progression of ALS in mice engineered to overexpress the gene coding for the mutated G93A form of the superoxide dismutase-1 (SOD1^G93A) (refs 7–10), even when treatment was only initiated at the onset of paralysis. VEGF treatment increased the life expectancy of ALS mice by 30 per cent without causing toxic side effects, thereby achieving one of the most effective therapies reported in the field so far.

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