Professor Mimoun Azzouz

Azzouz has great drive and passion to employ his scientific skills for the ultimate benefit of patients and families suffering from some of the most devastating diseases in medicine.

Azzouz achievements went beyond therapy development. Indeed, he also made significant discoveries related to the mechanisms of diseases in ALS/MND and SMA. For example, The recent discovery of the mechanism underpinning genome instability and neural cell death caused by C9orf72 expansion in ALS [Nature Neuroscience 20(9):1225-1235. (2017)] and role of PTEN in ALS and SMA [Human Molecular Genetics, 19(16):3159-68 (2010); Brain, 134(Pt 2):506-17 (2011)].

Major research interests

• Use of viral based gene transfer systems for research and therapy applications.
• Design and development of CNS targeted gene therapy vector systems
• In vitro and in vivo modelling of ALS/MND and SMA using viral vectors
• Identification of novel gene therapeutics to neurodegeneration in motor neuron diseases and HSP.
• Development of various gene therapy concepts: neuroprotection (GDNF, VEGF), gene silencing (SOD1), gene replacement (SMN) and approaches based on CRISPR editing (C9orf72).
• Clinical development to gene therapeutics: GLP regulatory safety studies and GMP manufacturing
• Biology of RNA binding proteins (e.g. SMN, Gle1).
• Elucidation of molecular mechanisms linked to DNA instability in ALS/MND, SMA and Dementia.
• Process innovation to establish novel processes for manufacturing of viral vectors.
• Manufacturing of clinical vectors for phase 1/2 human clinical trials. 

Collaborations