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Mimoun Azzouz "Viral Vectors: Gene Therapy and Research Application in Neurodegenerative Diseases"

Abstract :

Vectors based on adeno-associated and lentiviruses are opening up new approaches for the treatment of human neurodegenerative diseases.
They have been found to efficiently deliver genes into many cell types of the nervous system and in particular neurons, both in vitro and in vivo and the resulting expression is long-term, stable and non-toxic. These vectors have been refined making them safe for clinical applications.

This seminar will give an overview of the development of vectors from AAV and the non-primate lentivirus, Equine Infectious Anaemia virus (EIAV). Their application for gene transfer in the nervous system for therapeutic strategies in animal models of human neurodegenerative diseases such as Parkinson's disease, ALS and SMA will be discussed. In addition we have used these vectors to investigate mechanisms and signaling pathways in ALS and SMA. These features make vectors ideal for developing gene therapies and for investigating gene function in target validation and drug discovery studies. 

Selected publications

Ralph et al., (2005) Nature Medicine 11:429-33.
Azzouz et al. (2004) Nature 429: 413-417.
Azzouz et al.(2004) J.Clin. Invest 114 : 1726-1731.
Kirby et al. Brain 134(Pt 2):506-17 (2011).
Valori et al. Science Translational Medicine 2, 35ra42 (2010).
Ning et al. Human Molecular Genetics, 19(16):3159-68 (2010)

Scientific focus :

Professor Azzouz graduated in Biology and Neuroscience from the University of Rabat in 1993. He obtained a Master in Neuroscience with 1st Class Honours from the University of Marseille in 1994. In 1997 he was awarded a PhD in Neuropharmacology at the University Louis Pasteur in Strasbourg. He then worked as post doctoral scientist at the Gene Therapy Center in Lausanne, Switzerland from 1997 to 2000. He was recruited in 2000 by Oxford BioMedica Ltd as Senior Scientist then appointed as Director of Neurobiology in 2003. He was also a visiting scientist at Oxford University between 2000 and 2005. In 2006 he was appointed Professor of Translational Neuroscience at the University of Sheffield.

Nathalie Sans