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Author Details

Mimoun Azzouz
Sheffield Institute for Translational Neuroscience (SITraN), University of Sheffield
1996
76
31
Nevan J Krogan (CM4AI)
PMIDPaper TitleJournal TitlePublished Year
36481799Prospects for gene replacement therapies in amyotrophic lateral sclerosis.Nat Rev Neurol2023
36857431A cell-penetrant peptide blocking <i>C9ORF72</i>-repeat RNA nuclear export reduces the neurotoxic effects of dipeptide repeat proteins.Sci Transl Med2023
36685668Characterisation of APS-1 Experimental Models Is Crucial for Development of Novel Therapies.Biomed Res Int2023
36834552Syndecan-4 Mediates the Cellular Entry of Adeno-Associated Virus 9.Int J Mol Sci2023
36632189Ap4b1-knockout mouse model of hereditary spastic paraplegia type 47 displays motor dysfunction, aberrant brain morphology and ATG9A mislocalization.Brain Commun2023
34756547Delivery of therapeutic AAV9 vectors via cisterna magna to treat neurological disorders.Trends Mol Med2022
35440492SMN-deficient cells exhibit increased ribosomal DNA damage.Life Sci Alliance2022
35568435<i>C9ORF72</i>-derived poly-GA DPRs undergo endocytic uptake in iAstrocytes and spread to motor neurons.Life Sci Alliance2022
35628601Towards 3D Bioprinted Spinal Cord Organoids.Int J Mol Sci2022
36619668Loss of TMEM106B exacerbates C9ALS/FTD DPR pathology by disrupting autophagosome maturation.Front Cell Neurosci2022
36241425Low expression of EXOSC2 protects against clinical COVID-19 and impedes SARS-CoV-2 replication.Life Sci Alliance2022
35291294Low expression of EXOSC2 protects against clinical COVID-19 and impedes SARS-CoV-2 replication.bioRxiv2022
35313342SPG15 protein deficits are at the crossroads between lysosomal abnormalities, altered lipid metabolism and synaptic dysfunction.Hum Mol Genet2022
34904932Circumventing the packaging limit of AAV-mediated gene replacement therapy for neurological disorders.Expert Opin Biol Ther2022
33314575Directly converted astrocytes retain the ageing features of the donor fibroblasts and elucidate the astrocytic contribution to human CNS health and disease.Aging Cell2021
33792056Proteostatic imbalance and protein spreading in amyotrophic lateral sclerosis.EMBO J2021
34376242SRSF1-dependent inhibition of C9ORF72-repeat RNA nuclear export: genome-wide mechanisms for neuroprotection in amyotrophic lateral sclerosis.Mol Neurodegener2021
34156740Reply to: Gene therapy to cure haemophilia: Is robust scientific inquiry the missing factor?Haemophilia2021
32994995AAV9-mediated AIRE gene delivery clears circulating antibodies and tissue T-cell infiltration in a mouse model of autoimmune polyglandular syndrome type-1.Clin Transl Immunology2020
31915823Adaptor protein complex 4 deficiency: a paradigm of childhood-onset hereditary spastic paraplegia caused by defective protein trafficking.Hum Mol Genet2020
31444303The hybrid AAVP tool gets an upgrade.Proc Natl Acad Sci U S A2019
29552580Plastin 3 Promotes Motor Neuron Axonal Growth and Extends Survival in a Mouse Model of Spinal Muscular Atrophy.Mol Ther Methods Clin Dev2018
30002654Meta-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.Front Immunol2018
30195799Translating SOD1 Gene Silencing toward the Clinic: A Highly Efficacious, Off-Target-free, and Biomarker-Supported Strategy for fALS.Mol Ther Nucleic Acids2018
28840561Gene Therapy in the Nervous System: Failures and Successes.Adv Exp Med Biol2017
28158451C9ORF72 hexanucleotide repeat exerts toxicity in a stable, inducible motor neuronal cell model, which is rescued by partial depletion of Pten.Hum Mol Genet2017
29116194Site Specific Modification of Adeno-Associated Virus Enables Both Fluorescent Imaging of Viral Particles and Characterization of the Capsid Interactome.Sci Rep2017
28677678SRSF1-dependent nuclear export inhibition of C9ORF72 repeat transcripts prevents neurodegeneration and associated motor deficits.Nat Commun2017
28550099Viral delivery of <i>C9orf72</i> hexanucleotide repeat expansions in mice leads to repeat-length-dependent neuropathology and behavioural deficits.Dis Model Mech2017
28714954C9orf72 expansion disrupts ATM-mediated chromosomal break repair.Nat Neurosci2017
26921650Deficiency in the mRNA export mediator Gle1 impairs Schwann cell development in the zebrafish embryo.Neuroscience2016
27699224Systemic restoration of UBA1 ameliorates disease in spinal muscular atrophy.JCI Insight2016
26942208AAV9-mediated central nervous system-targeted gene delivery via cisterna magna route in mice.Mol Ther Methods Clin Dev2016
26700614Development of Nonviral Vectors Targeting the Brain as a Therapeutic Approach For Parkinson's Disease and Other Brain Disorders.Mol Ther2016
25369768PTEN depletion decreases disease severity and modestly prolongs survival in a mouse model of spinal muscular atrophy.Mol Ther2015
26446293Corrigendum: Comparison of stimulus-evoked cerebral hemodynamics in the awake mouse and under a novel anesthetic regime.Sci Rep2015
26189707LRP-1-mediated intracellular antibody delivery to the Central Nervous System.Sci Rep2015
26218081Comparison of stimulus-evoked cerebral hemodynamics in the awake mouse and under a novel anesthetic regime.Sci Rep2015
25959569Current developments in gene therapy for amyotrophic lateral sclerosis.Expert Opin Biol Ther2015
25978041The AIRE -230Y Polymorphism Affects AIRE Transcriptional Activity: Potential Influence on AIRE Function in the Thymus.PLoS One2015
24577094PTEN regulates AMPA receptor-mediated cell viability in iPS-derived motor neurons.Cell Death Dis2014
25268710Early detection of motor dysfunction in the SOD1G93A mouse model of Amyotrophic Lateral Sclerosis (ALS) using home cage running wheels.PLoS One2014
24845847Gene therapy: a promising approach to treating spinal muscular atrophy.Hum Gene Ther2014
23732987Viral delivery of antioxidant genes as a therapeutic strategy in experimental models of amyotrophic lateral sclerosis.Mol Ther2013
23023772Encapsulation of biomacromolecules within polymersomes by electroporation.Angew Chem Int Ed Engl2012
21228060Phosphatase and tensin homologue/protein kinase B pathway linked to motor neuron survival in human superoxide dismutase 1-related amyotrophic lateral sclerosis.Brain2011
21876739Optimised and rapid pre-clinical screening in the SOD1(G93A) transgenic mouse model of amyotrophic lateral sclerosis (ALS).PLoS One2011
20525971PTEN depletion rescues axonal growth defect and improves survival in SMN-deficient motor neurons.Hum Mol Genet2010
20538619Systemic delivery of scAAV9 expressing SMN prolongs survival in a model of spinal muscular atrophy.Sci Transl Med2010
19660657Gene therapy for neurodegenerative diseases based on lentiviral vectors.Prog Brain Res2009
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Collaborators

Pamela J Shaw
Sheffield Institute for Translational Neuroscience, University of Sheffield
Co-authored papers 24
Guillaume M Hautbergue
Sheffield Institute for Translational Neuroscience (SITraN), University of Sheffield
Co-authored papers 12
Laura Ferraiuolo
Sheffield Institute for Translational Neuroscience (SITraN), University of Sheffield
Co-authored papers 11
Lydia M Castelli
Sheffield Institute for Translational Neuroscience (SITraN), University of Sheffield
Co-authored papers 7
Jonathan D Cooper
Sheffield Institute for Translational Neuroscience (SITraN), University of Sheffield
Co-authored papers 5
Cleide Dos Santos Souza
Sheffield Institute for Translational Neuroscience (SITraN), University of Sheffield
Co-authored papers 3
Mark O Collins
University of Sheffield
Co-authored papers 3
Nevan J Krogan (CM4AI)
University of California San Francisco
Co-authored papers 2
Jan Rehwinkel
MRC Weatherall Institute of Molecular Medicine, University of Oxford
Co-authored papers 2
Jack Ng Marshall
Sheffield Institute for Translational Neuroscience, University of Sheffield
Co-authored papers 1
Sai Zhang
Center for Genomics and Personalized Medicine, Stanford University School of Medicine
Co-authored papers 1
Elizabeth D Buttermore
Boston Children's Hospital
Co-authored papers 1
Calum Harvey
Sheffield Institute for Translational Neuroscience, University of Sheffield
Co-authored papers 1
Andrew Peden
University of Sheffield
Co-authored papers 1
Michael P Snyder
Stanford University School of Medicine
Co-authored papers 1
David E Gordon
Emory University
Co-authored papers 1
Calum Harvey
Sheffield Institute for Translational Neuroscience, University of Sheffield
Co-authored papers 1
John Franklin
Sheffield Institute for Translational Neuroscience, University of Sheffield
Co-authored papers 1
Mustafa Sahin
Harvard Medical School
Co-authored papers 1
Sai Zhang
Stanford University School of Medicine
Co-authored papers 1
Emily Graves
Sheffield Institute for Translational Neuroscience, University of Sheffield
Co-authored papers 1
Andrew A Peden
University of Sheffield
Co-authored papers 1
Valerie Odon
Co-authored papers 1
Tobias Moll
Sheffield Institute for Translational Neuroscience, University of Sheffield
Co-authored papers 1
Emily Graves
Sheffield Institute for Translational Neuroscience, University of Sheffield
Co-authored papers 1
Valerie Odon
University of Oxford
Co-authored papers 1
Andrea Martinuzzi
Scientific Institute, IRCCS Eugenio Medea, Conegliano-Pieve di Soligo Research Centre
Co-authored papers 1
John Franklin
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Release 1.2