Professor Mimoun Azzouz

Neuroscience, School of Medicine and Population Health

Chair of Translational Neuroscience

ERC Advanced Investigator

Director of Gene Therapy Innovation and Manufacturing Centre (GTIMC)

ARDAT Coordinator

Professor Mimoun Azzouz
Profile picture of Professor Mimoun Azzouz
+44 114 222 2238
+44 114 222 0654 (Administrator: Laura Haslam)

Full contact details

Professor Mimoun Azzouz
Neuroscience, School of Medicine and Population Health
Room B31
Sheffield Institute for Translational Neuroscience (SITraN)
385a Glossop Road
S10 2HQ

Professor Azzouz 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 plc as Senior Scientist then appointed as Director of Neurobiology in 2003. In 2006, he was invited to join the University of Sheffield and was appointed to the Chair of Translational Neuroscience. Azzouz is currently Deputy Head of Department, Research & Innovation.

His track record of translational research productivity is characterised by publications in top ranking scientific journals. He won prestigious awards including the ERC Advanced Investigator (2011) and ERC Proof-of-Concept (2017) Awards. These awards are top level EU ad hominem award acknowledging his pre-eminence in European biomedical research.

He is a member Panels/Boards for various funding bodies such as the Medical Research Council (Translational MRC Panel, UK), the French Muscular Dystrophy Association (AFM), the Health Research Board (HRB) of Ireland, Research Council of Norway, and the Neuroscience Panel, Germany.

He has been recently named as Board member of the British Society for Gene and Cell Therapy. He serves as Scientific Advisor for pharmaceutical companies and other organisations.

Research interests

Professor Azzouz has achieved international pre-eminence in the field of gene therapy applications and vector development for neurodegenerative diseases. His contribution has been outstanding over the last 15 years as evidenced by the quality of publications achieved.

He has achieved a very high international status in an important field. His original and pioneering work, which has already produced major breakthroughs in animal models of neurodegenerative diseases, will in the near future, translate into major therapeutic advances in the field of human neurodegenerative disease.

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)].

His exceptional achievements were recently recognised at the European level by winning the prestigious ERC Advanced Investigator and ERC PoC Awards designed for individuals with excellence in scientific research. These awards offer him a platform to cement his European leadership in the field of gene therapy and translational neuroscience.

Current projects

  • Utilisation 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 and testing of various gene therapy concepts: neuroprotection (GDNF, VEGF), gene silencing (SOD1), gene replacement (SMN, SPG15, SPG47) and approaches based on CRISPR modulation of expansions (C9orf72).
  • Clinical development to gene therapeutics: GLP regulatory safety studies and GMP manufacturing
  • Biology of RNA binding proteins (e.g. SMN, Gle1).
  • Elucidation of the molecular mechanisms linked to DNA instability in ALS/MND, SMA and Dementia.
  • Molecular pathways and signalling of motor neuron death (e.g. PTEN)


  • Professor Pamela Shaw, The University of Sheffield, Sheffield, UK
  • Professor Michael Sendtner, Germany
  • Professor Nicola Woodroofe, Sheffield Halam 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
  • Professor Henry Houlden, University College London, UK
  • Dr Jenny Hirst, University of Cambridge, UK

Journal articles


  • Chandran JS, Scarrott JM, Shaw P & Azzouz M (2017) Gene Therapy in the Nervous System: Failures and Successes In El-Khamisy S (Ed.), Personalised Medicine Lessons from Neurodegeneration to Cancer Springer RIS download Bibtex download
  • Chandran JS, Scarrott JM, Shaw PJ & Azzouz M (2017) Gene Therapy in the Nervous System: Failures and Successes, Advances in Experimental Medicine and Biology (pp. 241-257). Springer International Publishing View this article in WRRO RIS download Bibtex download

Conference proceedings papers

  • Roach M, Azzouz M & Webster C (2022) Identification and Validation of Target Receptors for AAV9. MOLECULAR THERAPY, Vol. 30(4) (pp 426-426) RIS download Bibtex download
  • Roach M, Webster C & Azzouz M (2022) Identification and validation of target receptors for AAV9. HUMAN GENE THERAPY, Vol. 33(23-24) (pp A56-A56) RIS download Bibtex download
  • Webster CP, Crossley OM, Yang ZL, Coldicott I, King MC, Souza CDS, Ferraiuolo L & Azzouz M (2022) Gene-based therapeutics for C9ALS/FTD. HUMAN GENE THERAPY, Vol. 33(23-24) (pp A91-A91) RIS download Bibtex download
  • Alves-Cruzeiro JM, Karyka E, Bauer C, Coldicott I, Simon S, Hautbergue GM, Webster C, Myszczynska M, Higginbottom A, Ferraiuolo L & Azzouz M (2019) Gene editing as a potential therapeutic approach for ALS/FTD-associated with expanded C9ORF72. HUMAN GENE THERAPY, Vol. 30(8) (pp A22-A22) RIS download Bibtex download
  • Lukashchuk V, Lewis K, Coldicott I, Grierson A & Azzouz M (2016) AAV9-mediated central nervous system-targeted gene delivery via cisterna magna route in mice. HUMAN GENE THERAPY, Vol. 27(7) (pp A11-A11) View this article in WRRO RIS download Bibtex download
  • Iannitti T, Scarrot JM, Coldicott IRP, Kaspar BK, Ferraiuolo L, Shaw PJ & Azzouz M (2016) Gene Therapy for Familial ALS Using AAV9 Mediated Silencing of Mutant SOD1. HUMAN GENE THERAPY, Vol. 27(7) (pp A12-A12) View this article in WRRO RIS download Bibtex download
  • Chandran J, Sharp P, Collins M & Azzouz M (2016) Site specific labelling of adeno-associated virus identifies targets for enhancing viral transduction efficiency. HUMAN GENE THERAPY, Vol. 27(7) (pp A14-A14) View this article in WRRO RIS download Bibtex download
  • Herranz-Martin S, Lewis KE, Coldicott I, Chandran JS, Lukashchuk V, Iannitti T, Shaw PJ & Azzouz M (2016) Experimental modelling of ALS by AAV-mediated in vivo modulation of the C9ORF72 gene. HUMAN GENE THERAPY, Vol. 27(7) (pp A16-A16) RIS download Bibtex download
  • Scarrott J, Ferraiuolo L, Heath P, Gelsthorpe C, Kaspar B, Shaw DP & Azzouz M (2016) Investigating the Specificity of RNAi Molecules in Human Gene Therapy for Superoxide Dismutase 1-linked Familial Amyotrophic Lateral Sclerosis. HUMAN GENE THERAPY, Vol. 27(7) (pp A16-A17) RIS download Bibtex download
  • Alsomali N, Seytanoglu A, Valori C, Kim HR, Ning K, Ramesh T, Sharrack B, Wood JD & Azzouz M (2014) Deficiency in Gle1, an mRNA export mediator, inhibits Schwann cell development in the zebrafish embryo. FEBS JOURNAL, Vol. 281 (pp 771-771) RIS download Bibtex download
  • Almansoori K, Valori C, Furley A, Chandran J, Wood J & Azzouz M (2014) The mRNA exporter GLE1 is essential for embryonic development. FEBS Journal, Vol. 281(Suppl 1) (pp 644-645) RIS download Bibtex download
  • Mohammedeid A, Ning K, Kong SC, Azzouz M & Grierson A (2014) Regulation of protein aggregation by Arfaptin2 in amyotrophic lateral sclerosis. HUMAN GENE THERAPY, Vol. 25(5) (pp A14-A14) RIS download Bibtex download
  • Lovewell TRJ, McDonagh AJ, Messenger AG, Maleki-Dizaji A, Azzouz M & Tazi-Ahnini R (2013) Meta-analysis of AIRE regulated gene expression microarray data reveals a network of transcriptional interactions mediated by intermediate nodes. IMMUNOLOGY, Vol. 140 (pp 58-58) RIS download Bibtex download
  • Azzouz M (2013) SMN replacement gene therapy for spinal muscular atrophy: clinical development. HUMAN GENE THERAPY, Vol. 24(5) (pp A10-A10) RIS download Bibtex download
  • Little D, Valori C, Wyles M, Shaw P, Azzouz M & Ning K (2013) Systemic delivery of scAAV9 expressing PTEN siRNA prolongs survival in a model of spinal muscular atrophy. HUMAN GENE THERAPY, Vol. 24(5) (pp A27-A27) RIS download Bibtex download
  • Binny C, Karyka E, Ning K, Bennett E, Ince P, Shaw P & Azzouz M (2013) Efficient scAAV9-mediated delivery of SMN to motor neurons in neonatal and juvenile mice. HUMAN GENE THERAPY, Vol. 24(5) (pp A38-A38) RIS download Bibtex download
  • Lovewell TRJ, Mcdonagh AJG, Abu-Duhier F, Azzouz M, Messenger AG & Tazi-Ahnini R (2013) The effect of AIRE-207 polymorphism on AIRE transcriptional activity highlights the potential role of AIRE in the pathogenesis of alopecia areata. JOURNAL OF INVESTIGATIVE DERMATOLOGY, Vol. 133(5) (pp 1395-1395) RIS download Bibtex download
  • Valori CF, Ning K, Wyles M, Mead RJ, Grierson AJ, Shaw PJ & Azzouz M (2010) Systemic delivery of scAAV9 expressing SMN prolongs survival in a mouse model of SMA. HUMAN GENE THERAPY, Vol. 21(10) (pp 1424-1425) RIS download Bibtex download
  • Azzouz M, Valori C, Ning K & Wyles M (2010) Current Advances in Gene Therapy for Spinal Muscular Atrophy. HUMAN GENE THERAPY, Vol. 21(10) (pp 1388-1389) RIS download Bibtex download
  • Jarraya B, Lepetit H, Ralph S, Miskin J, Gurruchaga JM, Fenelon G, Boulet S, Jan C, Bonvento G, Azzouz M , Brugiere P et al (2010) Gene Therapy in Parkinson Disease: From Preclinical Studies in Primates to a Phase I Clinical Trial. HUMAN GENE THERAPY, Vol. 21(6) (pp 764-765) RIS download Bibtex download
  • Nanou A, Higginbottom A, Valori C, Wyles M, Ning K, Shaw P & Azzouz M (2010) Oxidative Stress as Target for Neuroprotection in Experimental Models of Amyotrophic Lateral Sclerosis (ALS). NEUROLOGY, Vol. 74(9) (pp A436-A436) RIS download Bibtex download
  • Seytanoglu A, Valori C, Ramesh T, Sharrack B & Azzouz M (2010) Reduced GLE1 Protein Levels Cause Axonal Growth Defects in Zebrafish Motor Neurons. NEUROLOGY, Vol. 74(9) (pp A441-A441) RIS download Bibtex download
  • Ning K, Drepper C, Ismail A, Valori CF, Wyles M, Higginbottom A, Herrmann T, Shaw P, Sharrack B, Sendtner M & Azzouz M (2010) PTEN Depletion Rescues the beta-Actin Deficit in Axonal Growth Cones in Motoneurons from a Mouse Model of Spinal Muscular Atrophy. NEUROLOGY, Vol. 74(9) (pp A489-A489) RIS download Bibtex download
  • Valori C, Ning K, Wyles M & Azzouz M (2010) Complete Rescue of SMA Mouse Model by Systemic Delivery of scAAV9 Mediating SMN Replacement. NEUROLOGY, Vol. 74(9) (pp A287-A287) RIS download Bibtex download
  • Jarraya B, Ralph S, Lepetit H, Stratful E, Boulet S, Jan C, Bonvento G, Azzouz M, Miskin JE, Gurruchaga JM , Vinti M et al (2009) A Phase I/II Trial for Parkinson's Disease Using a Lentiviral Vector (ProSavin (R)). MOLECULAR THERAPY, Vol. 17 (pp S197-S197) RIS download Bibtex download
  • Jarraya B, Ralph S, Lepetit H, Boulet S, Jan C, Bonvento G, Azzouz M, Miskin J, Gurruchaga JM, Palfi S , Fenelon G et al (2008) ProSavin: A gene therapy for Parkinson's Disease. HUMAN GENE THERAPY, Vol. 19(10) (pp 1060-1061) RIS download Bibtex download
  • Goodhead LH, Ning K, Azzouz M, Kingsman SM, Mitrophanous KA & Ralph GS (2008) Optimizing EIAV vectors and delivery routes for ALS gene therapy. HUMAN GENE THERAPY, Vol. 19(4) (pp 405-405) RIS download Bibtex download
  • Jarraya B, Ralph S, Bonvento G, Shin M, Jan C, Delzescaux T, Drouot X, Herard AS, Brouillet E, Azzouz M , Conde F et al (2007) Lentiviral-mediated dopamine replacement mediates sustained correction of parkinsonian symptoms in an MPTP-lesioned NHP model. HUMAN GENE THERAPY, Vol. 18(10) (pp 1026-1026) RIS download Bibtex download
  • Goodhead L, Azzouz M, Kingsman SM, Mitrophanous KA & Ralph S (2007) Optimizing EIAV vectors and delivery routes for ALS gene therapy. HUMAN GENE THERAPY, Vol. 18(10) (pp 1026-1027) RIS download Bibtex download
  • Azzouz M (2006) Treatment of familial ALS using Lentiviral-mediated silencing of mutant SOD1. NEUROMUSCULAR DISORDERS, Vol. 16 (pp S53-S54) RIS download Bibtex download
  • Ralph S, Radcliffe PA, Bilsland L, Greensmith L, Mitrophanous KA, Mazarakis ND & Azzouz M (2004) Targeted ablation of mutant SOD1 in ALS models using lentiviral mediated delivery of interfering RNA. JOURNAL OF NEUROCHEMISTRY, Vol. 90 (pp 59-59) RIS download Bibtex download
  • Tanase D, Teng QS, Krishnaney AA, Liu JK, Garrity-Moses ME, Mazarakis N, Walmsley LE, Azzouz M & Boulis NM (2004) Cervical spinal cord delivery of a lentiviral vector in SOD-1 transgenic mice. MOLECULAR THERAPY, Vol. 9 (pp S201-S201) RIS download Bibtex download
  • Teng QS, Garrity-Moses M, Liu JK, Tanase D, Azzouz M, Walmsley L, Mazarakis ND & Boulis NM (2004) EIAV-IGF-I gene transfer to motor neurons enhances axonal length in vitro. MOLECULAR THERAPY, Vol. 9 (pp S280-S280) RIS download Bibtex download
  • Jarraya B, Azzouz M, Miskin J, Ralph SG, Wilkes F, Rohll J, Walmsley LE, Ellard F, Kingsman SM, Mitrophanous KA , Hantraye P et al (2004) Functional rescue of parkinsonian non-human primates by a dopamine producing multicistronic lentiviral vector. MOLECULAR THERAPY, Vol. 9 (pp S407-S407) RIS download Bibtex download
  • Themis M, Gregory LG, Waddington SN, Holder MV, Mitrophanous KA, Buckley SMK, Bigger BW, Ellard FE, Walmsley LE, Radcliff P , Mazarakis N et al (2004) The fetal approach: A novel therapy for the treatment of musculo-skeletal disease. MOLECULAR THERAPY, Vol. 9 (pp S91-S91) RIS download Bibtex download
  • Azzouz M, Le T, Ralph SG, Wilkes F, Burghes AH, Kingsman SM, Mitrophanous KA & Mazarakis ND (2004) VEGF gene therapy with retrogradely transported lentivirus prolongs survival in mouse ALS model. MOLECULAR THERAPY, Vol. 9 (pp S200-S200) RIS download Bibtex download
  • Azzouz M, Le TN, Waddington S, Walmsley L, Monani U, Wilkes FJ, Themis M, Mitrophanous KA, Burghes A & Mazarakis ND (2003) Lentiviral vector-mediated gene therapy for a mouse model of spinal muscular atrophy. MOLECULAR THERAPY, Vol. 7(5) (pp S248-S248) RIS download Bibtex download
  • Azzouz M, Leclerc N, Gurney M, Warter JM, Poindron P & Borg J (1997) Progressive motor neuron impairment in an animal model of familial amyotrophic lateral sclerosis. NEUROCHEMISTRY (pp 485-490) RIS download Bibtex download


  • Azzouz M, Scarrott J & Karyka E (2021) Gene Therapy Treatment. WO2021205028A1 Appl. 14 Oct 2021. RIS download Bibtex download



Research group

Research team

  • Evangelia Karyka, Postdoctoral Research Associate
  • Joseph Scarrott, Postdoctoral Research Associate
  • Michela Pulix, Postdoctoral Research Associate
  • Emily Graves, PhD Student
  • João Alves-Cruzeiro, PhD Student
  • Paolo Marchi, PhD Student
  • Louise Whiteley, Research Technician
  • Nesrin Gariballa, Research Attachment
Professional activities and memberships
  • 2006-08 Advisor for Oxford BioMedica plc
  • 2008-13 Member of the Scientific Advisory Board, Muscular Dystrophy Association (F)
  • 2010 Committee of assessors for Genethon, Paris, France
  • 2010-16 Member of Strategic Board, AFM (F)
  • 2011-12 Advisor for BioMarin Pharmaceutical Inc
  • 2012-14 Panel Member for the Research Council of Norway
  • 2012-16 Panel member for the UK Medical Research Council (DPFS, MRC)
  • 2012-13 Advisor for QBRI, Qatar Foundation, Qatar
  • 2012- Member of Research Excellence Framework 2014 (REF14) Committee, FMDH
  • 2013-14 Neuroscience Panel Member, German Ministry of Education and Research
  • 2013- Panel member, Health Research Board (HRB), Dublin (Ireland)
  • 2014- Wellcome Trust Virtual Panel member for Translational Research Funds
  • 2015-17 Advisor for Spherium biomed
  • 2016- Board member of British Society for Gene & Cell Therapy
  • 2017- Advisory Board, IBRO-MENA
  • 2017- Scientific Advisor for the Batten CLN7 trial (Beat Batten)
  • 2017- Scientific Advisor for CureSPG47
  • 2017- Assessor for Innovate UK
  • 2017- Scientific Advisory Board, Telocyte, USA
  • 2017- Scientific Advisor for Maddi Foundation
Honours and awards
  • 2017: ERC Proof-of-Concept Award
  • 2014: Chair and organiser of Fusion Conference, Cancun, Mexico
  • 2012: ERC Advanced Investigator Award
  • 2012: Nomination for The Shaw Prize 2012
  • 2011: MRC DPFS Award holder
  • 2008: Chairman of UK SMA Conference
  • 2006: Faculty of the American Society of Gene Therapy
  • 2006: Faculty of the American Society of Neuroscience
  • 2006: Approached for an advisory role to the UK Government Department for Environment, Food and Rural Affairs (DEFRA) for “Defra desk study to review environmental risks from research trials and marketing of Genetically Modified (GM) veterinary and human medicines”