Professor Mimoun Azzouz

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
m.azzouz@sheffield.ac.uk
+44 114 222 2238
+44 114 222 0654 (Administrator: Laura Haslam)
Room B31, Sheffield Institute for Translational Neuroscience (SITraN)

Full contact details

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

Mimoun Azzouz is currently Professor of Translational Neuroscience and Director of Gene Therapy Innovation & Manufacturing Centre (GTIMC) at the University of Sheffield. His pioneering work, which has already produced major breakthroughs, led to real translational impact into major therapeutic advances for human neurodegenerative diseases. 

Azzouz leadership has been recognized by several prestigious awards, e.g. ERC Advanced Investigator, ERC Proof-of-Concept, IMI ARDAT (). He successfully attracted a unique combination of substantial funding from public, private, charity and local authority to establish GTIMC, a centre which includes provision of a state-of-the art manufacturing facility for gene therapy products to treat patients. 

Azzouz is member of Panels/Boards of funding bodies, Scientific Advisory Board memberships, Evaluation Committees, keynote and plenary lectures at established international meetings/institutions. He is inventor on several patents and is co-founder of 2 start-ups companies: BlackfinBio and Crucible Therapeutics.

Research interests

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

InstitutionCountry
Asphalion S.L. (ASP)ES
Commissariat A L’Energie Atomique (CEA)FR
Centro de Neurociências e Biologia Celular (CNC)PT
Genethon (GNT)FR
Genosafe SAS (GNS)FR
Instituto de Biologia Experimental e Tecnológica (iBET)PT
Institut du Cerveau et de la Moelle Epiniere (ICM)FR
Institut National De La Sante et La Recherche Medicale (INSERM)FR
Lund University (LU)SE
Lysogene (LSG)FR
Hannover Medical School (MHH)DE
Mimetas BV (MIM)NL
Pharmacoidea (PHI)HU
SynVaccine Ltd (SVC)IL
Takis Biotech (Takis)IT
The Chancellor Masters And Scholars-University of Cambridge (UC)UK
The Rosalind Franklin Institute (RFI)UK
University of Liverpool (ULIV)UK
University of Heidelberg (UKHD)DE
University College London (UCL)UK
University of Eastern Finland (UEF)FI
The Chancellor Masters And Scholars-University of Oxford (UOXF)UK
Pfizer (PFE)UK
Bayer (BAY)DE
Janssen Pharmaceutica NV (J&J)BE
Lonza AG (LAG)CH
Novartis (NVS)CH
Novo Nordisk (NOVO)DK
Sanofi-Aventis (SARD)FR
Spark Therapeutics, Inc (SPK)US
Takeda Pharmaceuticals International AG (TPIZ)CH
Viscofan SA (VSA)ES 
Astellas Pharma Europe BV (ASTEL)NL
Publications

Show: Featured publications All publications

Journal articles

  • Mann CJ, Giblin J, Braun M, Schmid F, Sørensen MR, Caferra P, Hudák A, Letoha T, Coderch N, Hickling TP & Azzouz M (2025) . Cell Reports Medicine, 102422-102422.
  • 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).
  • 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 (2024) . EMBO Molecular Medicine, 16(11), 2882-2917.
  • 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, 32(10), 3220-3259.
  • Giovannelli I, Higginbottom A, Kirby J, Azzouz M & Shaw PJ (2023) . Nature Reviews Neurology, 19(1), 39-52.
  • 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 Science Alliance, 5(9).
  • 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).
  • 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.
  • Walker C, Herranz-Martin S, Karyka E, Liao C, Lewis K, Elsayed W, Lukashchuk V, Chiang SC, Ray S, Mulcahy PJ , Jurga M et al (2017) . Nature Neuroscience, 20(9), 1225-1235.
  • 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.
  • Lukashchuk V, Lewis K, Coldicott I, Grierson A & Azzouz M (2016) . Molecular Therapy — Methods & Clinical Development, 2016(3).
  • 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.
  • 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, Ralph GS, Storkebaum E, Walmsley LE, Mitrophanous KA, Kingsman SM, Carmeliet P & Mazarakis ND (2004) . Nature, 429(6990), 413-417.

All publications

Journal articles

  • Mann CJ, Giblin J, Braun M, Schmid F, Sørensen MR, Caferra P, Hudák A, Letoha T, Coderch N, Hickling TP & Azzouz M (2025) . Cell Reports Medicine, 102422-102422.
  • Kocsy K, Wilkinson H, Felix-Ilemhenbhio F, Bax B, Van Agtmael T, Azzouz M & Majid A (2025) . Gene Therapy.
  • Felix-Ilemhenbhio F, Kocsy K, Azzouz M & Majid A (2025) . Brain Research, 1863, 149754-149754.
  • 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).
  • 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 (2024) . EMBO Molecular Medicine, 16(11), 2882-2917.
  • 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, 32(10), 3220-3259.
  • 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, 32(6), 2035-2035.
  • 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, 32(6), 2034-2034.
  • Castelli LM, Lin Y-H, Sanchez-Martinez A, Gül A, Imran KM, Higginbottom A, Márkus NM, Upadhyay SK, Martins AW, Cooper-Knock J , Montmasson C et al (2023) . Science Translational Medicine, 15(685).
  • Hudák A, Roach M, Pusztai D, Pettkó-Szandtner A, Letoha A, Szilák L, Azzouz M & Letoha T (2023) . International Journal of Molecular Sciences, 24(4).
  • Almaghrabi S, Lovewell T, Azzouz M & Tazi Ahnini R (2023) . BioMed Research International, 2023, 1-12.
  • 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).
  • Giovannelli I, Higginbottom A, Kirby J, Azzouz M & Shaw PJ (2023) . Nature Reviews Neurology, 19(1), 39-52.
  • 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).
  • 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.
  • 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).
  • 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 Science Alliance, 5(9).
  • 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, 8(3), 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, 31(16), 2693-2710.
  • Marrone L, Marchi PM & Azzouz M (2022) . Expert Opinion on Biological Therapy, 22(9), 1163-1176.
  • Marchi PM, Marrone L & Azzouz M (2022) . Trends in Molecular Medicine, 28(1), 79-80.
  • Benson BC, Shaw PJ, Azzouz M, Highley JR & Hautbergue GM (2021) . Frontiers in Neuroscience, 15.
  • Winburn I, Schulz M, LaRosa G & Azzouz M (2021) . Haemophilia, 27(5).
  • 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).
  • Cicardi ME, Marrone L, Azzouz M & Trotti D (2021) . The EMBO Journal, 40(10).
  • 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 (2021) . Aging Cell, 20(1).
  • 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, 8(10), 379-392.
  • 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.
  • Walker C, Herranz-Martin S, Karyka E, Liao C, Lewis K, Elsayed W, Lukashchuk V, Chiang SC, Ray S, Mulcahy PJ , Jurga M et al (2017) . Nature Neuroscience, 20(9), 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 and Mechanisms.
  • 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 MJ, Higginbottom A, Hautbergue GM, Cooper-Knock J, Mulcahy PJ, De Vos KJ, Renton AE, Pliner H, Calvo A, Chio A , Traynor BJ et al (2017) . Human Molecular Genetics, 26(6), 1133-1145.
  • Powis RA, Karyka E, Boyd P, Come J, Jones RA, Zheng Y, Szunyogova E, Groen EJN, Hunter G, Thomson D , Wishart TM et al (2016) . JCI Insight, 1(11).
  • Alsomali N, Seytanoglu A, Valori C, 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 K, Coldicott I, Grierson A & Azzouz M (2016) . Molecular Therapy — Methods & Clinical Development, 2016(3).
  • Sharp PS, Shaw K, Boorman L, Harris S, Kennerley AJ, Azzouz M & Berwick J (2015) . SCIENTIFIC REPORTS, 5.
  • Sharp P, Shaw K, Boorman L, Harris S, Kennerley AJ, Azzouz M & Berwick J (2015) . Scientific Reports, 5.
  • Tian X, Nyberg S, Sharp PS, Madsen J, Daneshpour N, Armes SP, Berwick J, Azzouz M, Shaw P, Abbott NJ & Battaglia G (2015) . Scientific Reports, 5.
  • Lovewell TRJ, McDonagh AJ, Messenger AG, Azzouz M & Tazi-Ahnini R (2015) . PLoS ONE, 10(5).
  • Scarrott JM, Herranz-Martín S, Alrafiah AR, Shaw PJ & Azzouz M (2015) . Expert Opinion on Biological Therapy.
  • 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.
  • Bennett EJ, Mead RJ, Azzouz M, Shaw PJ & Grierson AJ (2014) . PLOS ONE, 9(9).
  • 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(2), 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.
  • 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.
  • 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.
  • 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.
  • Dowd E, Monville C, Torres EM, Wong L-F, Azzouz M, Mazarakis ND & Dunnett SB (2005) . Eur J Neurosci, 22(10), 2587-2595.
  • 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.
  • 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.
  • Ralph GS, Mazarakis ND & Azzouz M (2005) . J Mol Med (Berl), 83(6), 413-419.
  • 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.
  • 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.
  • Azzouz M, Kingsman SM & Mazarakis ND (2004) . J Gene Med, 6(9), 951-962.
  • 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 LF, Azzouz M, Walmsley LE, Askham Z, Wilkes FJ, Mitrophanous KA, Kingsman SM & Mazarakis ND (2004) . MOL THER, 9(5), 765-765.
  • 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) . 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) . 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, 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, 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, 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.
  • 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.
  • 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.
  • Crooks L, Cooper-Knock J, Heath PR, Bouhouche A, Fahime EE, Azzouz M, Bakri Y, Adnaoui M, Ibrahimi A & Tazi-Ahnini R () .

Book 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) , Personalised Medicine (pp. 241-257).

Conference proceedings

  • 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)
  • Chandran J, Sharp P, Collins M & Azzouz M (2016) . Human Gene Therapy, Vol. 27(7) (pp A14-A14). London, UK, 15 June 2016 - 15 June 2016.
  • Iannitti T, Scarrot JM, Coldicott IRP, Kaspar BK, Ferraiuolo L, Shaw PJ & Azzouz M (2016) . Human Gene Therapy, Vol. 27(7) (pp A12-A12). University College London Institute of Child Health
  • 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)
  • Azzouz M (2013) SMN replacement gene therapy for spinal muscular atrophy: clinical development. HUMAN GENE THERAPY, Vol. 24(5) (pp A10-A10)
  • 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)
  • 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)
  • 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 (2022) , 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 (2021) , 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 (2021) , 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 (2021) , Cold Spring Harbor Laboratory.
Research group

SITraN 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
  • Rachel Waller, ARDAT Project Manager
  • Chris Webster, SITraN Fellow
  • Daniel Hollard, PhD Student
  • Zih-Liang Yang, Research Technician

GTIMC Team

Professional activities and memberships
  • 2006-2008   Advisor for Oxford BioMedica plc.
  • 2008-2013   Member of the Scientific Advisory Board, Muscular Dystrophy Association (F)
  • 2010-2016   Member of Strategic Board, AFM (F)
  • 2010               Member of the Evaluation Committee: Genethon, Paris, France
  • 2012-2016   Panel member for the UK Medical Research Council (MRC DPFS)
  • 2013-2014   Neuroscience Panel Member, German Ministry of Education and Research
  • 2011-2012   Advisor for BioMarin Pharmaceutical Inc
  • 2012-2013   Advisor for QBRI, Qatar Foundation, Qatar
  • 2014-2016   Advisor for Spherium biomed
  • 2013-2015   Panel member, Health Research Board (HRB), Dublin (Ireland)
  • 2016-2019   Board member of British Society for Gene & Cell Therapy
  • 2017-             Scientific Advisor for Beat Batten Foundation: 
  • 2017-             Scientific Advisor for CureAP4
  • 2017-             Scientific Advisor for Maddi Foundation
  • 2018               Chair Evaluation Committee, NCMM, Research Council of Norway, Oslo, Norway
  • 2018-2020   Telocyte Scientific Advisor Board (SAB)
  • 2018-             Member of the LifeArc Philanthropic Fund Panel
  • 2018-             Panel member of Irish Research Council, Advanced Laureate Awards Scheme
  • 2019               Expert Panel – Innovative Medicine Initiative (IMI)
  • 2020-             Member of the UKRI Future Leaders Fellowship Panel College
  • 2020-             NC3Rs CRACK IT Challenge 32 Transgene Track - Challenge Panel
  • 2021-             Scientific Advisory Board, Alcyone Therapeutics
  • 2021- 2024   Chair, NHSA Northern Network for Advanced Therapies
  • 2021               MHRA Pharmacovigilance Expert Advisory Group to advice on medicine development
  • 2021-2025    Chair of UKRI MRC/LifeArc National IHfGT process Innovation Committee
  • 2021               REF21 sub-panel UOA 4, Psychology, Psychiatry and Neuroscience
  • 2022-              Panel member, Caixa Foundation, Barcelona, Spain
  • 2023               MHRA Pharmacovigilance Expert Advisory Group
  • 2025-             Member of the ERC Advanced Awards Panel
  • 2025               Franklin Institute Bower Award Selection Committee
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”

Links

Administrator: Laura Haslam