Sheffield Institute for
Translational Neuroscience

Professor Mimoun Azzouz

Chair of Translational Neuroscience

ERC Advanced Fellow

Director of Research & Innovation

Deputy Head Neurology Unit

Research Interests

Professor Azzouz has a long-standing interest in developing gene therapy approaches for neurodegenerative diseases. Azzouz’ team utilises viral based gene transfer systems both for research and gene therapy applications. Such viral systems have included lentiviruses and adeno-associated vectors. His research focuses on developing new therapeutic strategies for monogenic neuromuscular disorders (e.g. SMA) and other motor neuron diseases (e.g. ALS). He also collaborates with other groups looking at new experimental approaches to treatment of Parkinson’s disease. Members of the team are also investigating molecular pathways and signalling of motor neuron death associated with ALS and SMA.




Current Projects

Recent research data has provided promising proof of concept for Resagen, a potential gene therapy product for patients with SMA. Applying this research could lead to a safe and effective treatment, but further studies are needed before starting clinical trials.

Using a modified virus as a vehicle for delivery, Resagen restores the missing survival motor neurone protein. It is given intravenously and crosses the blood-brain barrier to reach the affected cells in the spinal cord. Studies with mice have proved the concept - mice with the genetic mutation can survive for only two weeks, but after the therapy they can survive for over 300 days. The model can be translated to humans.

Resagen has received orphan drug status from the European Medicines Agency (EMA) and the team continues the pre-clinical development of Resagen with support from the Medical Research Council's Developmental Pathway Funding Scheme. The funding will help to develop a protocol for manufacture, as well as to conduct toxicity and safety studies paving the way towards clinical trials.Professor Azzouz hopes that with enough financial backing a Phase I clinical trial of Resagen will begin in the third quarter of 2014 to translate this therapy to human application in SMA patients.


Other research programmes:

1)      Design and development of CNS targeted vector system
2)      In vitro and in vivo disease modelling using viral vectors
3)      Biology of RNA binding proteins (e.g. SMN, Gle1)

  • Professor Pamela Shaw, The University of Sheffield, Sheffield, UK
  • Professor Michael Sendtner, Germany
  • Professor Nicola Woodroofe, Sheffield Hallam University, Sheffield, UK
  • Dr Jean-Marc Gallo, King's College London, UK
  • Dr. Guiseppe Battaglia, The University of Sheffield, UK.
  • Professor Ruth Luthi-Carter, University of Leicester
  • Professor Tom Gillingwater, Edinburgh University 
Principal Funding Sources
  • European Research Council (ERC Advanced Investigator Award)
  • Muscular Dystrophy Association (MDA), USA
  • Jennifer Trust for SMA, UK
  • MRC
  • Wellcome Trust
  • FightSMA, USA
  • AFM, France
  • NHS Trust
  • MS Society
  • SMA Europe/SMA Trust




Research Team

Senior Lecturer
  • Ke Ning
Post Doctorals 
  • Vera Lukashchuk
  • Jayanth Chandran
  • Saúl Herranz-Martín
  • Katherine Lewis
  • Kayleigh Iremonger
  • Tommaso Iannitti
Research technicians
  • Ian Coldicott
  • Ioannis Tsagakis
PhD students
  • Eva Karyka
  • Joseph Scarrott
  • Aziza R Alrafiah
  • Callum Walker
Project managers
  • Stacy Young
  • Evy De Leenheer


Professor Azzouz has an excellent publication track record. He published papers in high profile and prestigious journals such as Nature, Nature Medicine, Nature Neuroscience, Science Translational Medicine.


Selected publications

Nanou N, Higginbottom A, Valori C.F., Wyles M., Ning K., Shaw P, Azzouz M. Viral delivery of antioxidant genes as a therapeutic strategy in experimental models of amyotrophic lateral sclerosis. Molecular Therapy 21(8):1486-96. (2013) doi: 10.1038/mt.2013.115.

Kirby J., Ning K., Ferraiuolo L., Heath P.R., Ismail A., Kuo S-W., Cox L., Sharrack B., Wharton S.B., Ince P.G., Shaw PJ., Azzouz M. PTEN/Akt pathway linked to motor neuron survival in human SOD1-related amyotrophic lateral sclerosis. Brain, 134(Pt 2):506-17 (2011).

C. F. Valori, K. Ning, M. Wyles, R. J. Mead, A. J. Grierson, P. J. Shaw, M. Azzouz, Systemic delivery of scAAV9 expressing SMN prolongs survival in a model of spinal muscular atrophy . Sci.Transl. Med. 2, 35ra42 (2010).

Ning K, Drepper C, Valori C, Wyles M, Higginbottom A, Shaw P, Azzouz M*, Sendtner M*. PTEN depletion rescues axonal growth defect and improve survival in SMN-deficient motor neurons. Human Molecular Genetics, 19 (16):3159-68 (2010)(* joint senior authors).

Jarraya B, Drouot X, Brouillet E, Condé F, Azzouz M, Kingsman SM, Hantraye P, Mazarakis ND & Palfi S. Dopamine Gene Therapy for Parkinson´s Disease in a Nonhuman Primate Without Associated Dyskinesia. Sci. Transl Med. 1 (2):2ra4. (2009)

Wong LF,. Yip PK, Battaglia A, Grist J, Corcoran J, Maden M, Azzouz M, Kingsman SM, Kingsman AJ, Mazarakis ND and McMahon SB. Retinoic acid receptor β 2 promotes functional regeneration of sensory axons into the adult rat spinal cord. Nature Neuroscience, 9(2):243-250 (2006)

Ralph GS, Radcliffe PA, Bilsland L, Leroux MA, Greensmith L, Mitrophanous KA, Kingsman SM, Mazarakis ND, & Azzouz M
Silencing of mutant SOD1 using interfering RNA induces long term reversal of ALS in a transgenic mouse model. Nature Medicine, 11(4):429-33 (2005)

Ralph GS, Mazarakis ND & Azzouz M. Therapeutic gene silencing in neurodegenerative disease using interfering RNA. Journal of Molecular Medicine, 83(6):413-9 (2005)

Teng Q, Garrity-Moses M, Tanase D, Liu JK, Mazarakis ND, Azzouz M, & Boulis NM. Trophic activity of rabies G protein pseudotyped EIAV mediated IGF-I motor neuron gene transfer in vitro. Neurobiology of Disease, 20: 694-700 (2005)

Dowd E, Monville C, Torres E, Azzouz M, Mazarakis ND, Dunnett SB. The neurotrophic capabilty of letivector-mediated expression of GDNF: funtional assessment using a complex operant lateralised nose-poking task. EJN, 22: 2587-2595 (2005)

Azzouz M, Ralph GS , Storkebaum E, Walmsley L, Mitrophanous KA, Kingsman SM, Carmeliet P, & Mazarakis ND. VEGF delivery with retrogradely transported lentivector prolongs survival in a mouse ALS model. Nature, 429: 413-417 (2004).

Azzouz M, Le T, Walmsley L., Monani U., Wilkes F, Mitrophanous KA, Kingsman SM, Burghes A, & Mazarakis ND. LentiVector-Mediated SMN Replacement in a Mouse Model of spinal Muscular Atrophy. The Journal Clinical Investigation 114: 1726-1731 (2004).

Azzouz M, Martin-Rendon E, Barber RD, Mitrophanous KA, Carter EE, Rohll JB, Kingsman SM, Kingsman AJ, and Mazarakis ND. Multicistronic Lentiviral Vector-Mediated Striatal Gene Transfer of Aromatic L-Amino Acid Decarboxylase, Tyrosine Hydroxylase and GTP Cyclohydrolase I Induces Sustained Transgene Expression, Dopamine Production and Functional Improvement in a Rat Model of Parkinson´s Disease. The Journal of Neuroscience 22:10302-10312 (2002).

Mazarakis ND, Azzouz M, Rohll JB, Ellard FM, Olsen A , Carter EE, Barber R, O´Malley K, Kingsman SM, AJ Kingsman, and Mitrophanous KA. Rabies-G envelope pseudotyping of lentiviral vectors enables retrograde axonal transport and access to the nervous system after peripheral delivery. Human Molecular Genetics 10: 2109-2121 (2001).

Azzouz M, Hottinger A, Zurn A, Aebischer P, and Bueler H. Increased motoneuron survival and improved neuromuscular function in transgenic ALS mice after intraspinal injection of an adeno-associated virus encoding Bcl-2. Human Molecular Genetics 9: 801-811 (2000).

Professional Activities

  • Deputy Head of Academic Unit of Neurology, and Head of Gene Therapy
  • Director of Research & Innovation, Department of Neuroscience
  • ERC Advanced Investigator Award (2011)
  • MRC DPFS Award holder (2011)
  • Advisor for BioMarin Pharmaceutical Inc 2011-
  • Advisor for Oxford BioMedica Ltd. 2006-2008
  • Member of the Scientific Advisory Board, French Muscular Dystrophy Association 2008-
  • Member of Strategic Board, AFM 2010-
  • Panel Member of the Research Council of Norway 2011-
  • Panel member for MRC 2012-
  • Neuroscience Board Panel member, Germany 2013-
  • Member of the Editorial Board of Current Stem Cell Research & Therapy
  • Member of the Editorial Board of American Journal of Translational Research
  • Chairman of UK SMA Conference (November 2008)
  • Holder of several patents
  • Faculty of the American Society of Gene Therapy.
  • Faculty of the American Society of Neuroscience.

Biography & Awards

  • Jan 2006-present
    University of Sheffield
    Chair of Translational Neuroscience
  • 2003 – 2005
    Oxford Biomedica Ltd, Oxford
    Director of Neurobiology
  • 2000 – 2003
    Oxford Biomedica Ltd, Oxford
    Senior Scientist
  • 1997 - 2000
    Gene Therapy Centre, Lausanne, Switzerland
    Postdoctoral Position
  • 1993 - 1997
    University Louis Pasteur of Strasbourg, France
    Ph.D. in Neuropharmacology


Professor Azzouz has been successful in attracting an array of scientific awards including the prestigious ERC Advanced Investigator Award. This award is a top level EU ad hominem award acknowledging his pre-eminence in European biomedical research.

Professor Azzouz is advisor for pharmaceutical companies (BioMarin Pharmaceutical Inc, Oxford BioMedica Ltd). He is member of scientific advisory panel for various funding bodies such as the Medical Research Council (MRC), the French Muscular Dystrophy Association and The Research Council of Norway. He is also Founder and Board Director of ReSaGen Ltd.

Contact Details

Academic Neurology Unit
Department of Neuroscience
University of Sheffield
Room B31
385a Glossop Road
S10 2HQ

T: +44 (0)114 2222238

Secretary: Rebecca Brown
T: +44 (0)114 2222261