One of the limits of gene therapy for spinal amyotrophy (SMA) lies in the difficulty in transporting the therapeutic gene as far as the motoneurons of the spinal cord. In fact, the central nervous system (brain and spinal cord) is separated from the blood circulation by the haemato-encephalic barrier, which is very difficult to cross. Martine Barkats and her team (Institute of Myology) have shown – for the first time in cat models – that an AAV (adeno-associated virus) vector is capable of passing from the blood circulation to the spinal cord. These encouraging results should be confirmed by more extensive studies.