Gene Therapy For Neurodegeneration

Researchers from INSERM in France and Rhone-Poulenc Rorer subsidiary RPRGencell have demonstrated, for the first time, that an in vivo gene therapy can improve lifespan in a genetical model of disease. The work is published in Nature Medicine (April).

The researchers used an adenovirus vector to deliver two nerve growth factors, ciliary neurotrophic factor and neurotrophin-3, to muscle cells in mice with a form of motor neuron disease. The mice exhibited a 50% increase in lifespan, reduced loss of motor axons and improved neuromuscular function, as measured by electromyography.

Neurotrophic factors have been pursued as treatments for motor neuron diseases for some time, but have been limited by the need to inject them, as well as their toxicity and poor bioavailability. This new gene therapy approach goes a long way towards circumventing these drawbacks, particularly as relatively long-term expression of the factors followed intramuscular injection in this study. Local delivery mimics more closely the physiological situation.