Funded Research Projects


AAV9-SMN Gene Therapy for SMA

Principal investigator(s):
Dr. Martine Barkats
INSERM (France)
192 764€
Start Year:
2 years
SMA Europe


Dr. Martine Barkats

Q. What are the steps needed to take SMN gene therapy from mouse models into human clinical trials?

A. The project is ongoing. Dr. Barkats and her team recently discovered that a gene transfer vehicle, derived from a non-pathogenic virus called AAV9, was able to transfer genes to the brain and the spinal cord (CNS) despite the presence of the protective barrier to the CNS called the blood brain barrier. They subsequently showed that using this method to transfer the SMN1 gene to the spinal cord dramatically increased the life-span of a mouse model of SMA. The development of a clinical project based on this method of SMN delivery is ongoing as a collaboration between the Institute of Myology and Généthon (France). The team is going to pursue this research by optimizing the gene transfer vehicle and its route of delivery in order to improve the therapeutic efect of this promising gene therapy approach for SMA.