Journal of Gene Medicine European Society of Gene Therapy (ESGT) Young Investigator Award 2005 will be presented at the 13 th annual ESGT meeting in Prague on November 1, 2005. The recipient, Aurélie Goyenvalle, won the award for her presentation ''Highly efficient exon-skipping and sustained correction of muscular dystrophy using an Adeno-Associated Viral vector'', given at the 12 th annual ESGT meeting in Tampere, Finland in November 2004. Aurélie Goyenvalle obtained a Masters Degree in Virology from the University of Paris VII and the Pasteur Institute in Paris. She became interested in gene therapy early on in her studies, being particularly drawn to the application of viruses within the field. In 2001, she began work on her PhD in the Duchenne Muscular Dystrophy (DMD) Laboratory at Genethon.
During her time at Genethon, she has developed an exon-skipping gene therapy strategy for DMD using adeno-associated virus vectors (AAV) encoding chimeric U7snRNA carrying antisense sequences against a selected exon of the dystrophin gene. When first tested in the mdx mouse model of DMD, the approach was shown to produce an almost complete restoration of dystrophin expression in muscles treated with an adeno-associated virus vector specific for exon 23 (Goyenvalle et al., 2004).
Aurélie is currently involved in the development of AAV and lentiviral vectors harbouring chimeric U7 snRNA carrying antisense sequences against exon 51 of the human dystrophin gene. Skipping this exon would restore a functional quasi-dystrophin in a significant subset of DMD patients. To date, the efficiency with which these vectors restore dystrophin expression has been tested and confirmed in myoblasts from patients.
Further information on the presentation given by Aurélie at the 12 th ESGT meeting in Tampere is provided below: