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)
+44 114 222 2238
Full contact details
Neuroscience, School of Medicine and Population Health
Sheffield Institute for Translational Neuroscience (SITraN)
385a Glossop Road
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.
- 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
- A cell-penetrant peptide blocking C9ORF72 -repeat RNA nuclear export reduces the neurotoxic effects of dipeptide repeat proteins. Science Translational Medicine, 15(685).
- Characterisation of APS-1 experimental models is crucial for development of novel therapies. BioMed Research International, 2023.
- Ap4b1-knockout mouse model of hereditary spastic paraplegia type 47 displays motor dysfunction, aberrant brain morphology and ATG9A mislocalization. Brain Communications, 5(1).
- Low expression of EXOSC2 protects against clinical COVID-19 and impedes SARS-CoV-2 replication. Life Science Alliance, 6(1), e202201449-e202201449.
- Loss of TMEM106B exacerbates C9ALS/FTD DPR pathology by disrupting autophagosome maturation. Frontiers in Cellular Neuroscience, 16.
- C9ORF72-derived poly-GA DPRs undergo endocytic uptake in iAstrocytes and spread to motor neurons.. Life Sci Alliance, 5(9).
- Towards 3D bioprinted spinal cord organoids. International Journal of Molecular Sciences, 23(10). View this article in WRRO
- Current global regulatory landscape for biodistribution & shedding assessment of rAAV gene therapies & recommendations of the IMI ARDAT consortium on future directions. Cell and Gene Therapy Insights, 08(03), 377-394.
- SMN-deficient cells exhibit increased ribosomal DNA damage. Life Science Alliance, 5(8).
- SPG15 protein deficits are at the crossroads between lysosomal abnormalities, altered lipid metabolism and synaptic dysfunction. Human Molecular Genetics.
- Circumventing the packaging limit of AAV-mediated gene replacement therapy for neurological disorders. Expert Opinion on Biological Therapy.
- SRSF1-dependent inhibition of C9ORF72-repeat RNA nuclear export : genome-wide mechanisms for neuroprotection in amyotrophic lateral sclerosis. Molecular Neurodegeneration, 16(1). View this article in WRRO
- Delivery of therapeutic AAV9 vectors via cisterna magna to treat neurological disorders. Trends in Molecular Medicine.
- Directly converted astrocytes retain the ageing features of the donor fibroblasts and elucidate the astrocytic contribution to human CNS health and disease. Aging Cell. View this article in WRRO
- Identification of single nucleotide variants in the Moroccan population by whole-genome sequencing. BMC Genetics, 21(1).
- SOD1-targeting therapies for neurodegenerative diseases : a review of current findings and future potential. Expert Opinion on Orphan Drugs. View this article in WRRO
- AAV9‐mediated AIRE gene delivery clears circulating antibodies and tissue T‐cell infiltration in a mouse model of autoimmune polyglandular syndrome type‐1. Clinical & Translational Immunology, 9. View this article in WRRO
- Adaptor protein complex 4 deficiency: a paradigm of childhood-onset hereditary spastic paraplegia caused by defective protein trafficking. Human Molecular Genetics, 29(2), 320-334.
- The hybrid AAVP tool gets an upgrade. Proceedings of the National Academy of Sciences, 116(37), 18162-18164. View this article in WRRO
- Translating SOD1 Gene Silencing toward the Clinic: A Highly Efficacious, Off-Target-free, and Biomarker-Supported Strategy for fALS. Molecular Therapy : Nucleic Acids, 12, 75-88. View this article in WRRO
- Meta-analysis of autoimmune regulator-regulated genes in human and murine models: a novel human model provides insights on the role of autoimmune regulator in regulating STAT1 and STAT1-regulated genes. Frontiers in Immunology, 9. View this article in WRRO
- Plastin 3 promotes motor neuron axonal growth and extends survival in a mouse model of spinal muscular atrophy. Molecular Therapy - Methods and Clinical Development, 9, 81-89. View this article in WRRO
- SRSF1-dependent nuclear export of C9ORF72 repeat transcripts: targeting toxic gain-of-functions induced by protein sequestration as a selective therapeutic strategy for neuroprotection. Therapeutic Targets for Neurological Diseases, 4. View this article in WRRO
- Site Specific Modification of Adeno-Associated Virus Enables Both Fluorescent Imaging of Viral Particles and Characterization of the Capsid Interactome. Scientific Reports, 7(1). View this article in WRRO
- C9orf72 Expansion Disrupts ATM-mediated Chromosomal Break Repair. Nature Neuroscience, 20, 1225-1235. View this article in WRRO
- Viral delivery of C9ORF72 hexanucleotide repeat expansions in mice lead to repeat length dependent neuropathology and behavioral deficits.. Disease Models & Mechanisms, 10, 859-868. View this article in WRRO
- SRSF1-dependent nuclear export inhibition of C9ORF72 repeat transcripts prevents neurodegeneration and associated motor deficits. Nature Communications, 8. View this article in WRRO
- C9ORF72 hexanucleotide repeat exerts toxicity in a stable, inducible motor neuronal cell model, which is rescued by partial depletion of Pten. Human Molecular Genetics, 26(6), 1133-1145. View this article in WRRO
- Systemic restoration of UBA1 ameliorates disease in spinal muscular atrophy. JCI Insight, 1(11). View this article in WRRO
- Deficiency in the mRNA export mediator Gle1 impairs Schwann cell development in the zebrafish embryo. Neuroscience, 322, 287-297. View this article in WRRO
- Corrigendum to “Development of Nonviral Vectors Targeting the Brain as a Therapeutic Approach For Parkinson's Disease and Other Brain Disorders”. Molecular Therapy, 24(4), 843-843.
- Development of Nonviral Vectors Targeting the Brain as a Therapeutic Approach For Parkinson’s Disease and Other Brain Disorders. Molecular Therapy, 24(4), 746-758.
- AAV9-mediated central nervous system–targeted gene delivery via cisterna magna route in mice. Molecular Therapy - Methods and Clinical Development, 3. View this article in WRRO
- Comparison of stimulus-evoked cerebral hemodynamics in the awake mouse and under a novel anesthetic regime. Scientific Reports, 5. View this article in WRRO
- LRP-1-mediated intracellular antibody delivery to the Central Nervous System. Scientific Reports, 5, 11990-11990. View this article in WRRO
- Current developments in gene therapy for amyotrophic lateral sclerosis. Expert Opinion on Biological Therapy, 15(7), 935-947. View this article in WRRO
- PTEN Depletion Decreases Disease Severity and Modestly Prolongs Survival in a Mouse Model of Spinal Muscular Atrophy. Molecular Therapy, 23(2), 270-277. View this article in WRRO
- Adeno-Associated Vectors for Gene Delivery to the Nervous System, 1-22.
- Erratum: Corrigendum: Comparison of stimulus-evoked cerebral hemodynamics in the awake mouse and under a novel anesthetic regime. Scientific Reports, 5(1).
- Early Detection of Motor Dysfunction in the SOD1G93A Mouse Model of Amyotrophic Lateral Sclerosis (ALS) Using Home Cage Running Wheels. PLoS ONE, 9(9), e107918-e107918. View this article in WRRO
- Gene therapy: a promising approach to treating spinal muscular atrophy.. Hum Gene Ther, 25(7), 575-586.
- Current and investigational treatments for spinal muscular atrophy. Expert Opinion on Orphan Drugs, 2(5), 465-476.
- PTEN regulates AMPA receptor-mediated cell viability in iPS-derived motor neurons.. Cell Death Dis, 5, e1096. View this article in WRRO
- Viral delivery of antioxidant genes as a therapeutic strategy in experimental models of amyotrophic lateral sclerosis.. Mol Ther, 21(8), 1486-1496. View this article in WRRO
- Microscopy: Progress and prospect. Journal of Microscopy and Ultrastructure, 1(3), 63-63.
- Encapsulation of Biomacromolecules within Polymersomes by Electroporation. Angewandte Chemie, 124(44), 11284-11287.
- Encapsulation of biomacromolecules within polymersomes by electroporation.. Angew Chem Int Ed Engl, 51(44), 11122-11125.
- PTEN: A molecular target for neurodegenerative disorders. TRANSLATIONAL NEUROSCIENCE, 3(2), 132-142.
- PTEN signalling in motor neuron disease (ALS & SMA). HUMAN GENE THERAPY, 22(10), A64-A64.
- Impact of PTEN modulation on motor neuron survival in experimental models of motor neuron disease. HUMAN GENE THERAPY, 22(10), A62-A62.
- Polymersome mediated gene therapy for spinal muscular atrophy. HUMAN GENE THERAPY, 22(10), A83-A84.
- Current Advances in Gene Therapy for Spinal Muscular Atrophy. HUMAN GENE THERAPY, 22(10), A19-A19.
- Gene therapy approaches to evaluate neuroprotection in experimental models of Amyotrophic Lateral Sclerosis. HUMAN GENE THERAPY, 22(10), A64-A64.
- Optimised and rapid pre-clinical screening in the SOD1(G93A) transgenic mouse model of amyotrophic lateral sclerosis (ALS).. PLoS One, 6(8), e23244. View this article in WRRO
- Phosphatase and tensin homologue/protein kinase B pathway linked to motor neuron survival in human superoxide dismutase 1-related amyotrophic lateral sclerosis.. Brain, 134(Pt 2), 506-517. View this article in WRRO
- Systemic delivery of scAAV9 expressing SMN prolongs survival in a model of spinal muscular atrophy.. Sci Transl Med, 2(35), 35ra42.
- PTEN depletion rescues axonal growth defect and improves survival in SMN-deficient motor neurons.. Hum Mol Genet, 19(16), 3159-3168.
- Dopamine gene therapy for Parkinson's disease in a nonhuman primate without associated dyskinesia.. Sci Transl Med, 1(2), 2ra4.
- Gene therapy for neurodegenerative diseases based on lentiviral vectors.. Prog Brain Res, 175, 187-200.
- Development and applications of non-HIV-based lentiviral vectors in neurological disorders.. Curr Gene Ther, 8(6), 406-418.
- Gene Therapy for ALS: progress and prospects.. Biochim Biophys Acta, 1762(11-12), 1122-1127.
- Retinoic acid receptor beta2 promotes functional regeneration of sensory axons in the spinal cord.. Nat Neurosci, 9(2), 243-250.
- Gene therapy for neurodegenerative and ocular diseases using lentiviral vectors.. Clin Sci (Lond), 110(1), 37-46.
- Trophic activity of Rabies G protein-pseudotyped equine infectious anemia viral vector mediated IGF-I motor neuron gene transfer in vitro.. Neurobiol Dis, 20(3), 694-700.
- Lentivector-mediated delivery of GDNF protects complex motor functions relevant to human Parkinsonism in a rat lesion model.. Eur J Neurosci, 22(10), 2587-2595.
- Therapeutic gene silencing in neurological disorders, using interfering RNA.. J Mol Med (Berl), 83(6), 413-419.
- Silencing mutant SOD1 using RNAi protects against neurodegeneration and extends survival in an ALS model.. Nat Med, 11(4), 429-433.
- Lentivector-mediated SMN replacement in a mouse model of spinal muscular atrophy.. J Clin Invest, 114(12), 1726-1731.
- Non-primate EIAV-based lentiviral vectors as gene delivery system for motor neuron diseases.. Curr Gene Ther, 4(3), 277-286.
- Lentiviral vectors for treating and modeling human CNS disorders.. J Gene Med, 6(9), 951-962.
- Local GDNF expression mediated by lentiviral vector protects facial nerve motoneurons but not spinal motoneurons in SOD1(G93A) transgenic mice.. Neurobiol Dis, 16(1), 139-149.
- VEGF delivery with retrogradely transported lentivector prolongs survival in a mouse ALS model.. Nature, 429(6990), 413-417.
- Transduction patterns of pseudotyped lentiviral vectors in the nervous system (vol 9, pg 101, 2003). MOL THER, 9(5), 765-765.
- Neuroprotection in a rat Parkinson model by GDNF gene therapy using EIAV vector.. Neuroreport, 15(6), 985-990.
- Transduction patterns of pseudotyped lentiviral vectors in the nervous system.. Mol Ther, 9(1), 101-111.
- 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.
- Lentiviral vectors for the treatment of neurodegenerative diseases.. Curr Opin Mol Ther, 3(5), 476-481.
- Rabies virus glycoprotein pseudotyping of lentiviral vectors enables retrograde axonal transport and access to the nervous system after peripheral delivery.. Hum Mol Genet, 10(19), 2109-2121.
- 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.
- Increased motoneuron survival and improved neuromuscular function in transgenic ALS mice after intraspinal injection of an adeno-associated virus encoding Bcl-2.. Hum Mol Genet, 9(5), 803-811.
- Gene transfer to the mouse spinal cord using a lentiviral vector. EUR J NEUROSCI, 12, 228-228.
- Prevention of mutant SOD1 motoneuron degeneration by copper chelators in vitro.. J Neurobiol, 42(1), 49-55.
- Compensatory mechanism of motor defect in SOD1 transgenic mice by overactivation of striatal cholinergic neurons.. Neuroreport, 10(5), 1013-1018.
- Progressive motor neuron impairment in an animal model of familial amyotrophic lateral sclerosis.. Muscle Nerve, 20(1), 45-51.
- Electromyographical and motor performance studies in the pmn mouse model of neurodegenerative disease.. Neurobiol Dis, 3(2), 137-147.
- Enhancement of mouse sciatic nerve regeneration by the long chain fatty alcohol, N-Hexacosanol.. Exp Neurol, 138(2), 189-197.
- Biphasic progression of motor unit dysfunction in an animal model of ALS. J NEUROCHEM, 66, S112-S112.
- Syndecan-4 Mediates the Cellular Entry of Adeno-Associated Virus 9. International Journal of Molecular Sciences, 24(4), 3141-3141.
- Prospects for gene replacement therapies in amyotrophic lateral sclerosis. Nature Reviews Neurology.
- Reply to: Gene therapy to cure haemophilia: Is robust scientific inquiry the missing factor?. Haemophilia.
- Proteostatic imbalance and protein spreading in amyotrophic lateral sclerosis. The EMBO Journal.
- Identification of single nucleotide variants in the Moroccan population by whole-genome sequencing.
- The AIRE -230Y Polymorphism Affects AIRE Transcriptional Activity: Potential Influence on AIRE Function in the Thymus. PLoS ONE, 10(5). View this article in WRRO
- Proteinopathies as Hallmarks of Impaired Gene Expression, Proteostasis and Mitochondrial Function in Amyotrophic Lateral Sclerosis. Frontiers in Neuroscience, 15.
- Gene Therapy in the Nervous System: Failures and Successes In El-Khamisy S (Ed.), Personalised Medicine Lessons from Neurodegeneration to Cancer Springer
- 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
Conference proceedings papers
- Identification and Validation of Target Receptors for AAV9. MOLECULAR THERAPY, Vol. 30(4) (pp 426-426)
- Identification and validation of target receptors for AAV9. HUMAN GENE THERAPY, Vol. 33(23-24) (pp A56-A56)
- Gene-based therapeutics for C9ALS/FTD. HUMAN GENE THERAPY, Vol. 33(23-24) (pp A91-A91)
- Gene editing as a potential therapeutic approach for ALS/FTD-associated with expanded C9ORF72. HUMAN GENE THERAPY, Vol. 30(8) (pp A22-A22)
- View this article in WRRO 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 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 Site specific labelling of adeno-associated virus identifies targets for enhancing viral transduction efficiency. HUMAN GENE THERAPY, Vol. 27(7) (pp A14-A14)
- Experimental modelling of ALS by AAV-mediated in vivo modulation of the C9ORF72 gene. HUMAN GENE THERAPY, Vol. 27(7) (pp A16-A16)
- 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)
- Deficiency in Gle1, an mRNA export mediator, inhibits Schwann cell development in the zebrafish embryo. FEBS JOURNAL, Vol. 281 (pp 771-771)
- The mRNA exporter GLE1 is essential for embryonic development. FEBS Journal, Vol. 281(Suppl 1) (pp 644-645)
- Regulation of protein aggregation by Arfaptin2 in amyotrophic lateral sclerosis. HUMAN GENE THERAPY, Vol. 25(5) (pp A14-A14)
- 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)
- 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)
- SMN replacement gene therapy for spinal muscular atrophy: clinical development. HUMAN GENE THERAPY, Vol. 24(5) (pp A10-A10)
- 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)
- Efficient scAAV9-mediated delivery of SMN to motor neurons in neonatal and juvenile mice. HUMAN GENE THERAPY, Vol. 24(5) (pp A38-A38)
- 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)
- Systemic delivery of scAAV9 expressing SMN prolongs survival in a mouse model of SMA. HUMAN GENE THERAPY, Vol. 21(10) (pp 1424-1425)
- Current Advances in Gene Therapy for Spinal Muscular Atrophy. HUMAN GENE THERAPY, Vol. 21(10) (pp 1388-1389)
- 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)
- Oxidative Stress as Target for Neuroprotection in Experimental Models of Amyotrophic Lateral Sclerosis (ALS). NEUROLOGY, Vol. 74(9) (pp A436-A436)
- Reduced GLE1 Protein Levels Cause Axonal Growth Defects in Zebrafish Motor Neurons. NEUROLOGY, Vol. 74(9) (pp A441-A441)
- 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)
- Complete Rescue of SMA Mouse Model by Systemic Delivery of scAAV9 Mediating SMN Replacement. NEUROLOGY, Vol. 74(9) (pp A287-A287)
- A Phase I/II Trial for Parkinson's Disease Using a Lentiviral Vector (ProSavin (R)). MOLECULAR THERAPY, Vol. 17 (pp S197-S197)
- ProSavin: A gene therapy for Parkinson's Disease. HUMAN GENE THERAPY, Vol. 19(10) (pp 1060-1061)
- Optimizing EIAV vectors and delivery routes for ALS gene therapy. HUMAN GENE THERAPY, Vol. 19(4) (pp 405-405)
- 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)
- Optimizing EIAV vectors and delivery routes for ALS gene therapy. HUMAN GENE THERAPY, Vol. 18(10) (pp 1026-1027)
- Treatment of familial ALS using Lentiviral-mediated silencing of mutant SOD1. NEUROMUSCULAR DISORDERS, Vol. 16 (pp S53-S54)
- Targeted ablation of mutant SOD1 in ALS models using lentiviral mediated delivery of interfering RNA. JOURNAL OF NEUROCHEMISTRY, Vol. 90 (pp 59-59)
- Cervical spinal cord delivery of a lentiviral vector in SOD-1 transgenic mice. MOLECULAR THERAPY, Vol. 9 (pp S201-S201)
- EIAV-IGF-I gene transfer to motor neurons enhances axonal length in vitro. MOLECULAR THERAPY, Vol. 9 (pp S280-S280)
- Functional rescue of parkinsonian non-human primates by a dopamine producing multicistronic lentiviral vector. MOLECULAR THERAPY, Vol. 9 (pp S407-S407)
- The fetal approach: A novel therapy for the treatment of musculo-skeletal disease. MOLECULAR THERAPY, Vol. 9 (pp S91-S91)
- VEGF gene therapy with retrogradely transported lentivirus prolongs survival in mouse ALS model. MOLECULAR THERAPY, Vol. 9 (pp S200-S200)
- Lentiviral vector-mediated gene therapy for a mouse model of spinal muscular atrophy. MOLECULAR THERAPY, Vol. 7(5) (pp S248-S248)
- Progressive motor neuron impairment in an animal model of familial amyotrophic lateral sclerosis. NEUROCHEMISTRY (pp 485-490)
- Directly converted astrocytes retain the ageing features of the donor fibroblasts and elucidate the astrocytic contribution to human CNS health and disease..
- Low expression of EXOSC2 protects against clinical COVID-19 and impedes SARS-CoV-2 replication, Cold Spring Harbor Laboratory.
- C9orf72-derived poly-GA DPRs undergo endocytic uptake in iNPC-derived astrocytes and spread to motor neurons, Cold Spring Harbor Laboratory.
- A cell-penetrant peptide blocking C9ORF72-repeat RNA nuclear export suppresses neurodegeneration, Cold Spring Harbor Laboratory.
- Safety and efficacy of C9ORF72-repeat RNA nuclear export inhibition in amyotrophic lateral sclerosis, Cold Spring Harbor Laboratory.
- Research group
- 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”