Azzouz, Mimoun, Professor

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

m.azzouz@sheffield.ac.uk
+44 114 222 2238

+44 114 222 0654 (Administrator: Laura Haslam)

Room B31, 91̽�� Institute for Translational Neuroscience (SITraN)

Full contact details

Professor Mimoun Azzouz
Neuroscience, School of Medicine and Population Health
Room B31
91̽�� Institute for Translational Neuroscience (SITraN)
385a Glossop Road
91̽��
S10 2HQ

Profile

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 91̽�� 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)

Collaborations

, 91̽��, 91̽��, UK
Professor Michael Sendtner, Germany
Professor Nicola Woodroofe, 91̽�� Halam University, 91̽��, UK
Dr Jean-Marc Gallo, King's College London, UK
Dr. Guiseppe Battaglia, 91̽��, 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

Publications

Journal articles

Webster CP, Hall B, Crossley OM, Dauletalina D, King M, Lin Y-H, Castelli LM, Yang Z-L, Coldicott I, Kyrgiou-Balli E , Higginbottom A et al (2025) . Life Science Alliance, 8(2), e202402757-e202402757.
Vrellaku B, Sethw Hassan I, Howitt R, Webster CP, Harriss E, McBlane F, Betts C, Schettini J, Lion M, Mindur JE , Duerr M et al (2024) . Molecular Therapy.
Javed H, Menon SA, Al-Mansoori KM, Al-Wandi A, Majbour NK, Ardah MT, Varghese S, Vaikath NN, Haque ME, Azzouz M & El-Agnaf OMA (2024) . Molecular Therapy.
Javed H, Menon SA, Al-Mansoori KM, Al-Wandi A, Majbour NK, Ardah MT, Varghese S, Vaikath NN, Haque ME, Azzouz M & El-Agnaf OMA (2024) . Molecular Therapy.
Castelli LM, Lin Y-H, Sanchez-Martinez A, Gül A, Mohd Imran K, Higginbottom A, Upadhyay SK, Márkus NM, Rua Martins R, Cooper-Knock J , Montmasson C et al (2023) . Science Translational Medicine, 15(685).
Almaghrabi S, Lovewell T, Azzouz M & Tazi Ahnini R (2023) . BioMed Research International, 2023.
Scarrott JM, Alves-Cruzeiro J, Marchi PM, Webster CP, Yang Z-L, Karyka E, Marroccella R, Coldicott I, Thomas H & Azzouz M (2023) . Brain Communications, 5(1).
Moll T, Odon V, Harvey C, Collins MO, Peden A, Franklin J, Graves E, Marshall JNG, dos Santos Souza C, Zhang S , Castelli L et al (2023) . Life Science Alliance, 6(1), e202201449-e202201449.
Bauer CS, Webster CP, Shaw AC, Kok JR, Castelli LM, Lin Y-H, Smith EF, Illanes-Álvarez F, Higginbottom A, Shaw PJ , Azzouz M et al (2022) . Frontiers in Cellular Neuroscience, 16.
Marchi PM, Marrone L, Brasseur L, Coens A, Webster CP, Bousset L, Destro M, Smith EF, Walther CG, Alfred V , Marroccella R et al (2022) . Life Sci Alliance, 5(9).
Han Y, King M, Tikhomirov E, Barasa P, Souza CDS, Lindh J, Baltriukiene D, Ferraiuolo L, Azzouz M, Gullo MR & Kozlova EN (2022) . International Journal of Molecular Sciences, 23(10).
Schmidt NA, Giblin J, MacLachlan TK, Dandapat S, Veres G, Reimer TA, Schulz M, Hatzmann E, Dahy A, LaRosa G , Azzouz M et al (2022) . Cell and Gene Therapy Insights, 08(03), 377-394.
Karyka E, Berrueta Ramirez N, Webster CP, Marchi PM, Graves EJ, Godena VK, Marrone L, Bhargava A, Ray S, Ning K , Crane H et al (2022) . Life Science Alliance, 5(8).
Marrone L, Marchi PM, Webster CP, Marroccella R, Coldicott I, Reynolds S, Alves-Cruzeiro J, Yang Z-L, Higginbottom A, Khundadze M , Shaw PJ et al (2022) . Human Molecular Genetics.
Marrone L, Marchi PM & Azzouz M (2022) . Expert Opinion on Biological Therapy.
Castelli LM, Cutillo L, Souza CDS, Sanchez-Martinez A, Granata I, Lin Y-H, Myszczynska MA, Heath PR, Livesey MR, Ning K , Azzouz M et al (2021) . Molecular Neurodegeneration, 16(1).
Marchi PM, Marrone L & Azzouz M (2021) . Trends in Molecular Medicine.
Gatto N, Dos Santos Souza C, Shaw AC, Bell SM, Myszczynska MA, Powers S, Meyer K, Castelli LM, Karyka E, Mortiboys H , Azzouz M et al (2020) . Aging Cell.
Crooks L, Cooper-Knock J, Heath PR, Bouhouche A, Elfahime M, Azzouz M, Bakri Y, Adnaoui M, Ibrahimi A, Amzazi S & Tazi-Ahnini R (2020) . BMC Genetics, 21(1).
Franklin JP, Azzouz M & Shaw PJ (2020) . Expert Opinion on Orphan Drugs.
Almaghrabi S, Azzouz M & Tazi Ahnini R (2020) . Clinical & Translational Immunology, 9.
Behne R, Teinert J, Wimmer M, D’Amore A, Davies AK, Scarrott JM, Eberhardt K, Brechmann B, Chen IP-F, Buttermore ED , Barrett L et al (2020) . Human Molecular Genetics, 29(2), 320-334.
Alves-Cruzeiro J, Webster CP & Azzouz M (2019) . Proceedings of the National Academy of Sciences, 116(37), 18162-18164.
Iannitti T, Scarrott J, Likhite S, Coldicott IRP, Lewis KE, Heath PR, Higginbottom A, Myszczynska MA, Milo M, Hautbergue GM , Meyer K et al (2018) . Molecular Therapy : Nucleic Acids, 12, 75-88.
Lovewell T, McDonagh A, Messenger A, Azzouz M & Tazi-Ahnini R (2018) . Frontiers in Immunology, 9.
Alrafiah A, Karyka E, Coldicott I, Iremonger K, Lewis K, Ning K & Azzouz M (2018) . Molecular Therapy - Methods and Clinical Development, 9, 81-89.
Castelli LM, Lin YH, Ferraiuolo L, Sanchez-Martinez A, Ning KE, Azzouz M, Whitworth A, Shaw PJ & Hautbergue GM (2018) . Therapeutic Targets for Neurological Diseases, 4.
Chandran JS, Sharp PS, Karyka E, Aves-Cruzeiro JMDC, Coldicott I, Castelli L, Hautbergue G, Collins MO & Azzouz M (2017) . Scientific Reports, 7(1).
Walker C, Herranz-Martin S, Karyka E, Liao C, Lewis K, Lukashchuk V, Chiang S-C, Ray S, Mulcahy PJ, Jurga M , Tsagakis I et al (2017) . Nature Neuroscience, 20, 1225-1235.
Herranz-Martin S, Chandran J, Lewis K, Mulcahy P, Higginbottom A, Walker C, Valenzuela IM-PY, Jones RA, Coldicott I, Iannitti T , Akaaboune M et al (2017) . Disease Models & Mechanisms, 10, 859-868.
Hautbergue GM, Castelli LM, Ferraiuolo L, Sanchez-Martinez A, Cooper-Knock J, Higginbottom A, Lin YH, Bauer CS, Dodd JE, myszczynska MA , Alam SM et al (2017) . Nature Communications, 8.
Stopford M, Higginbottom A, Hautbergue , Cooper-Knock , Mulcahy , De Vos K, Renton A, Pliner H, Calvo A, Chio A , Traynor B et al (2017) . Human Molecular Genetics, 26(6), 1133-1145.
Azzouz M (2016) . JCI Insight, 1(11).
Seytanoglu A, Alsomali NI, Valori CF, McGown A, Kim HR, Ning K, Ramesh T, Sharrack B, Wood JD & Azzouz M (2016) . Neuroscience, 322, 287-297.
(2016) . Molecular Therapy, 24(4), 843-843.
Javed H, Menon SA, Al-Mansoori KM, Al-Wandi A, Majbour NK, Ardah MT, Varghese S, Vaikath NN, Haque ME, Azzouz M & El-Agnaf OMA (2016) . Molecular Therapy, 24(4), 746-758.
Lukashchuk V, Lewis KE, Coldicott I, Grierson AJ & Azzouz M (2016) . Molecular Therapy - Methods and Clinical Development, 3.
Sharp PS, Shaw K, Boorman L, Harris S, Kennerley A, Azzouz M & Berwick J (2015) . Scientific Reports, 5.
Tian X, Nyberg S, S. Sharp P, Madsen J, Daneshpour N, Armes SP, Berwick J, Azzouz M, Shaw P, Abbott NJ & Battaglia G (2015) . Scientific Reports, 5, 11990-11990.
Scarrott JM, Herranz-Martín S, Alrafiah AR, Shaw PJ & Azzouz M (2015) . Expert Opinion on Biological Therapy, 15(7), 935-947.
Little D, Valori CF, Mutsaers CA, Bennett EJ, Wyles M, Sharrack B, Shaw PJ, Gillingwater TH, Azzouz M & Ning K (2015) . Molecular Therapy, 23(2), 270-277.
Mulcahy PJ, Binny C, Muszynski B, Karyka E & Azzouz M (2015) , 1-22.
Sharp PS, Shaw K, Boorman L, Harris S, Kennerley AJ, Azzouz M & Berwick J (2015) . Scientific Reports, 5(1).
Bennett EJ, Mead RJ, Azzouz M, Shaw PJ & Grierson AJ (2014) . PLoS ONE, 9(9), e107918-e107918.
Mulcahy PJ, Iremonger K, Karyka E, Herranz-Martín S, Shum K-T, Tam JKV & Azzouz M (2014) . Hum Gene Ther, 25(7), 575-586.
Tam JKV, Karyka E & Azzouz M (2014) . Expert Opinion on Orphan Drugs, 2(5), 465-476.
Yang D-J, Wang X-L, Ismail A, Ashman CJ, Valori CF, Wang G, Gao S, Higginbottom A, Ince PG, Azzouz M , Xu J et al (2014) . Cell Death Dis, 5, e1096.
Nanou A, Higginbottom A, Valori CF, Wyles M, Ning K, Shaw P & Azzouz M (2013) . Mol Ther, 21(8), 1486-1496.
Azzouz M & Binny C (2013) . Journal of Microscopy and Ultrastructure, 1(3), 63-63.
Wang L, Chierico L, Little D, Patikarnmonthon N, Yang Z, Azzouz M, Madsen J, Armes SP & Battaglia G (2012) . Angewandte Chemie, 124(44), 11284-11287.
Wang L, Chierico L, Little D, Patikarnmonthon N, Yang Z, Azzouz M, Madsen J, Armes SP & Battaglia G (2012) . Angew Chem Int Ed Engl, 51(44), 11122-11125.
Ismail A, Ning K, Al-Hayani A, Sharrack B & Azzouz M (2012) . TRANSLATIONAL NEUROSCIENCE, 3(2), 132-142.
Ning K, Drepper C, Valori CF, Wyles M, Ismail A, Higginbottom A, Herrmann T, Shaw PJ, Sendtner M & Azzouz M (2011) PTEN signalling in motor neuron disease (ALS & SMA). HUMAN GENE THERAPY, 22(10), A64-A64.
Ismail A, Ning K, Sharrack B & Azzouz M (2011) Impact of PTEN modulation on motor neuron survival in experimental models of motor neuron disease. HUMAN GENE THERAPY, 22(10), A62-A62.
Little D, Ning K, Nanou A, Wang LG, Canton I, Battaglia G & Azzouz M (2011) Polymersome mediated gene therapy for spinal muscular atrophy. HUMAN GENE THERAPY, 22(10), A83-A84.
Azzouz M (2011) Current Advances in Gene Therapy for Spinal Muscular Atrophy. HUMAN GENE THERAPY, 22(10), A19-A19.
Nanou A, Higginbottom A, Wyles M, Ning K, Shaw P & Azzouz M (2011) Gene therapy approaches to evaluate neuroprotection in experimental models of Amyotrophic Lateral Sclerosis. HUMAN GENE THERAPY, 22(10), A64-A64.
Mead RJ, Bennett EJ, Kennerley AJ, Sharp P, Sunyach C, Kasher P, Berwick J, Pettmann B, Battaglia G, Azzouz M , Grierson A et al (2011) . PLoS One, 6(8), e23244.
Kirby J, Ning K, Ferraiuolo L, Heath PR, Ismail A, Kuo S-W, Valori CF, Cox L, Sharrack B, Wharton SB , Ince PG et al (2011) . Brain, 134(Pt 2), 506-517.
Valori CF, Ning K, Wyles M, Mead RJ, Grierson AJ, Shaw PJ & Azzouz M (2010) . Sci Transl Med, 2(35), 35ra42.
Ning K, Drepper C, Valori CF, Ahsan M, Wyles M, Higginbottom A, Herrmann T, Shaw P, Azzouz M & Sendtner M (2010) . Hum Mol Genet, 19(16), 3159-3168.
Jarraya B, Boulet S, Ralph GS, Jan C, Bonvento G, Azzouz M, Miskin JE, Shin M, Delzescaux T, Drouot X , Hérard A-S et al (2009) . Sci Transl Med, 1(2), 2ra4.
Nanou A & Azzouz M (2009) . Prog Brain Res, 175, 187-200.
Valori CF, Ning K, Wyles M & Azzouz M (2008) . Curr Gene Ther, 8(6), 406-418.
Azzouz M (2006) . Biochim Biophys Acta, 1762(11-12), 1122-1127.
Wong L-F, Yip PK, Battaglia A, Grist J, Corcoran J, Maden M, Azzouz M, Kingsman SM, Kingsman AJ, Mazarakis ND & McMahon SB (2006) . Nat Neurosci, 9(2), 243-250.
Ralph GS, Binley K, Wong L-F, Azzouz M & Mazarakis ND (2006) . Clin Sci (Lond), 110(1), 37-46.
Teng Q, Garrity-Moses M, Federici T, Tanase D, Liu JK, Mazarakis ND, Azzouz M, Walmsley LE, Carlton E & Boulis NM (2005) . Neurobiol Dis, 20(3), 694-700.
Dowd E, Monville C, Torres EM, Wong L-F, Azzouz M, Mazarakis ND & Dunnett SB (2005) . Eur J Neurosci, 22(10), 2587-2595.
Ralph GS, Mazarakis ND & Azzouz M (2005) . J Mol Med (Berl), 83(6), 413-419.
Ralph GS, Radcliffe PA, Day DM, Carthy JM, Leroux MA, Lee DCP, Wong L-F, Bilsland LG, Greensmith L, Kingsman SM , Mitrophanous KA et al (2005) . Nat Med, 11(4), 429-433.
Azzouz M, Le T, Ralph GS, Walmsley L, Monani UR, Lee DCP, Wilkes F, Mitrophanous KA, Kingsman SM, Burghes AHM & Mazarakis ND (2004) . J Clin Invest, 114(12), 1726-1731.
Azzouz M & Mazarakis N (2004) . Curr Gene Ther, 4(3), 277-286.
Azzouz M, Kingsman SM & Mazarakis ND (2004) . J Gene Med, 6(9), 951-962.
Guillot S, Azzouz M, Déglon N, Zurn A & Aebischer P (2004) . Neurobiol Dis, 16(1), 139-149.
Azzouz M, Ralph GS, Storkebaum E, Walmsley LE, Mitrophanous KA, Kingsman SM, Carmeliet P & Mazarakis ND (2004) . Nature, 429(6990), 413-417.
Wong LF, Azzouz M, Walmsley LE, Askham Z, Wilkes FJ, Mitrophanous KA, Kingsman SM & Mazarakis ND (2004) . MOL THER, 9(5), 765-765.
Azzouz M, Ralph S, Wong L-F, Day D, Askham Z, Barber RD, Mitrophanous KA, Kingsman SM & Mazarakis ND (2004) . Neuroreport, 15(6), 985-990.
Wong L-F, Azzouz M, Walmsley LE, Askham Z, Wilkes FJ, Mitrophanous KA, Kingsman SM & Mazarakis ND (2004) . Mol Ther, 9(1), 101-111.
Azzouz M, Martin-Rendon E, Barber RD, Mitrophanous KA, Carter EE, Rohll JB, Kingsman SM, Kingsman AJ & Mazarakis ND (2002) 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.. J Neurosci, 22(23), 10302-10312.
Martin-Rendon E, Azzouz M & Mazarakis ND (2001) Lentiviral vectors for the treatment of neurodegenerative diseases.. Curr Opin Mol Ther, 3(5), 476-481.
Mazarakis ND, Azzouz M, Rohll JB, Ellard FM, Wilkes FJ, Olsen AL, Carter EE, Barber RD, Baban DF, Kingsman SM , Kingsman AJ et al (2001) . Hum Mol Genet, 10(19), 2109-2121.
Hottinger AF, Azzouz M, Déglon N, Aebischer P & Zurn AD (2000) Complete and long-term rescue of lesioned adult motoneurons by lentiviral-mediated expression of glial cell line-derived neurotrophic factor in the facial nucleus.. J Neurosci, 20(15), 5587-5593.
Azzouz M, Hottinger A, Paterna JC, Zurn AD, Aebischer P & Büeler H (2000) . Hum Mol Genet, 9(5), 803-811.
Azzouz M, Deglon N, Zurn A & Aebischer P (2000) Gene transfer to the mouse spinal cord using a lentiviral vector. EUR J NEUROSCI, 12, 228-228.
Azzouz M, Poindron P, Guettier S, Leclerc N, Andres C, Warter JM & Borg J (2000) Prevention of mutant SOD1 motoneuron degeneration by copper chelators in vitro.. J Neurobiol, 42(1), 49-55.
Azzouz M, Krezel W, Dollé P, Vodouhe C, Warter JM, Poindron P & Borg J (1999) . Neuroreport, 10(5), 1013-1018.
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.. Muscle Nerve, 20(1), 45-51.
Kennel PF, Fonteneau P, Martin E, Schmidt JM, Azzouz M, Borg J, Guenet JL, Schmalbruch H, Warter JM & Poindron P (1996) . Neurobiol Dis, 3(2), 137-147.
Azzouz M, Kenel PF, Warter J-M , Poindron P & Borg J (1996) . Exp Neurol, 138(2), 189-197.
Borg J, Azzouz M, Leclerc N, Gurney M, Warter JM & Poindron P (1996) Biphasic progression of motor unit dysfunction in an animal model of ALS. J NEUROCHEM, 66, S112-S112.
Wiseman JP, Scarrott JM, Alves-Cruzeiro J, Saffari A, Böger C, Karyka E, Dawes E, Davies AK, Marchi PM, Graves E , Fernandes F et al () . EMBO Molecular Medicine.
Hudák A, Roach M, Pusztai D, Pettkó-Szandtner A, Letoha A, Szilák L, Azzouz M & Letoha T () . International Journal of Molecular Sciences, 24(4), 3141-3141.
Giovannelli I, Higginbottom A, Kirby J, Azzouz M & Shaw PJ () . Nature Reviews Neurology.
Winburn I, Schulz M, LaRosa G & Azzouz M () . Haemophilia.
Cicardi ME, Marrone L, Azzouz M & Trotti D () . The EMBO Journal.
Crooks L, Cooper-Knock J, Heath PR, Bouhouche A, Fahime EE, Azzouz M, Bakri Y, Adnaoui M, Ibrahimi A & Tazi-Ahnini R () .
Lovewell TRJ, McDonagh AJ, Messenger AG, Azzouz M & Tazi-Ahnini R () . PLoS ONE, 10(5).
Benson BC, Shaw PJ, Azzouz M, Highley JR & Hautbergue GM () . Frontiers in Neuroscience, 15.

Chapters

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
Chandran JS, Scarrott JM, Shaw PJ & Azzouz M (2017) , Advances in Experimental Medicine and Biology (pp. 241-257). Springer International Publishing

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)
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)
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)
Wiseman J, Cruzeiro J, Scarrott J, Karyka E, Marchi P, Webster CP, Yang Z, Coldicott I, Shaw PJ, Ferraiuolo L & Azzouz M (2022) Gene Replacement Therapy for Spastic Paraplegia 47. HUMAN GENE THERAPY, Vol. 33(23-24) (pp A26-A27)
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)
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)
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)
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)
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)
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)
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)
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)
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)
Woodroofe MN, Sinagra M, Bunning R, Bolton C & Azzouz M (2013) EFFECT OF SILENCING ADAM17 EXPRESSION BY AN ADENOVIRAL VECTOR-MEDIATED RNA INTERFERENCE APPROACH IN CHRONIC RELAPSING EXPERIMENTAL AUTOIMMUNE ENCEPHALOMYELITIS.. GLIA, Vol. 61 (pp S43-S43)
Azzouz M (2013) SMN replacement gene therapy for spinal muscular atrophy: clinical development. HUMAN GENE THERAPY, Vol. 24(5) (pp A10-A10)
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)
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)
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)
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)
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)
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)
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)
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)
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)
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)
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)
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)
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)
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)
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)
Azzouz M (2006) Treatment of familial ALS using Lentiviral-mediated silencing of mutant SOD1. NEUROMUSCULAR DISORDERS, Vol. 16 (pp S53-S54)
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)
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)
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)
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)
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)
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)
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)
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)

Patents

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

Datasets

Gatto N, Souza CDS, Bell S, Shaw A, Myszczynska M, Powers S, Meyer K, Heath P, Castelli L, Karyka E , Mortiboys H et al .

Preprints

Moll T, Odon V, Harvey C, Collins MO, Peden A, Franklin J, Graves E, Marshall JNG, Santos Souza CD, Zhang S , Azzouz M et al () , Cold Spring Harbor Laboratory.
Marchi PM, Marrone L, Brasseur L, Bousset L, Webster CP, Destro M, Smith EF, Walther CG, Alfred V, Marroccella R , Robinson D et al () , Cold Spring Harbor Laboratory.
Castelli LM, Sanchez-Martinez A, Lin Y-H, Upadhyay SK, Higginbottom A, Cooper-Knock J, Gül A, Walton A, Montmasson C, Cohen R , Bauer CS et al () , Cold Spring Harbor Laboratory.
Castelli LM, Cutillo L, Souza CDS, Sanchez-Martinez A, Granata I, Myszczynska MA, Heath PR, Livesey MR, Ning K, Azzouz M , Shaw PJ et al () , Cold Spring Harbor Laboratory.

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”

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School of Medicine and Population Health