Long-term safety and tolerability of ProSavin, a lentiviral vector-based gene therapy for Parkinson's disease: a dose escalation, open-label, phase 1/2 trial
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Published in The Lancet (British edition) (29.03.2014)
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Neuroinflammation in the normal-appearing white matter (NAWM) of the multiple sclerosis brain causes abnormalities at the nodes of Ranvier
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Published in PLoS biology (01.12.2020)
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Persistent elevation of intrathecal pro-inflammatory cytokines leads to multiple sclerosis-like cortical demyelination and neurodegeneration
James, Rachel E, Schalks, Renee, Browne, Eleanor, Eleftheriadou, Ioanna, Munoz, Carmen Picon, Mazarakis, Nicholas D, Reynolds, Richard
Published in Acta neuropathologica communications (12.05.2020)
Published in Acta neuropathologica communications (12.05.2020)
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Familial amyotrophic lateral sclerosis is associated with a mutation in D-amino acid oxidase
Mitchell, John, Paul, Praveen, Chen, Han-Jou, Morris, Alex, Payling, Miles, Falchi, Mario, Habgood, James, Panoutsou, Stefania, Winkler, Sabine, Tisato, Veronica, Hajitou, Amin, Smith, Bradley, Vance, Caroline, Shaw, Christopher, Mazarakis, Nicholas D, de Belleroche, Jacqueline
Published in Proceedings of the National Academy of Sciences - PNAS (20.04.2010)
Published in Proceedings of the National Academy of Sciences - PNAS (20.04.2010)
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Cyclin-dependent-like kinase 5 is required for pain signaling in human sensory neurons and mouse models
La Montanara, Paolo, Hervera, Arnau, Baltussen, Lucas L, Hutson, Thomas H, Palmisano, Ilaria, De Virgiliis, Francesco, Kong, Guiping, Chadwick, Jessica, Gao, Yunan, Bartus, Katalin, Majid, Qasim A, Gorgoraptis, Nikos, Wong, Kingsley, Downs, Jenny, Pizzorusso, Tommaso, Ultanir, Sila K, Leonard, Helen, Yu, Hongwei, Millar, David S, Istvan, Nagy, Mazarakis, Nicholas D, Di Giovanni, Simone
Published in Science translational medicine (08.07.2020)
Published in Science translational medicine (08.07.2020)
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Silencing mutant SOD1 using RNAi protects against neurodegeneration and extends survival in an ALS model
Ralph, G Scott, Azzouz, Mimoun, Radcliffe, Pippa A, Day, Denise M, Carthy, Janine M, Leroux, Marie A, Lee, Debbie C P, Wong, Liang-Fong, Bilsland, Lynsey G, Greensmith, Linda, Kingsman, Susan M, Mitrophanous, Kyriacos A, Mazarakis, Nicholas D
Published in Nature medicine (01.04.2005)
Published in Nature medicine (01.04.2005)
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Dopamine gene therapy for Parkinson's disease in a nonhuman primate without associated dyskinesia
Jarraya, Béchir, Boulet, Sabrina, Ralph, G Scott, Jan, Caroline, Bonvento, Gilles, Azzouz, Mimoun, Miskin, James E, Shin, Masahiro, Delzescaux, Thierry, Drouot, Xavier, Hérard, Anne-Sophie, Day, Denise M, Brouillet, Emmanuel, Kingsman, Susan M, Hantraye, Philippe, Mitrophanous, Kyriacos A, Mazarakis, Nicholas D, Palfi, Stéphane
Published in Science translational medicine (14.10.2009)
Published in Science translational medicine (14.10.2009)
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Lentivector-mediated SMN replacement in a mouse model of spinal muscular atrophy
Azzouz, Mimoun, Le, Thanh, Ralph, G Scott, Walmsley, Lucy, Monani, Umrao R, Lee, Debbie C P, Wilkes, Fraser, Mitrophanous, Kyriacos A, Kingsman, Susan M, Burghes, Arthur H M, Mazarakis, Nicholas D
Published in The Journal of clinical investigation (01.12.2004)
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Enhanced Central Nervous System Transduction with Lentiviral Vectors Pseudotyped with RVG/HIV-1gp41 Chimeric Envelope Glycoproteins
Trabalza, Antonio, Eleftheriadou, Ioanna, Sgourou, Argyro, Liao, Ting-Yi, Patsali, Petros, Lee, Heyne, Mazarakis, Nicholas D
Published in Journal of Virology (01.03.2014)
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Lentivirus-mediated gene transfer to the central nervous system: therapeutic and research applications
Wong, Liang-Fong, Goodhead, Lucy, Prat, Christine, Mitrophanous, Kyriacos A, Kingsman, Susan M, Mazarakis, Nicholas D
Published in Human gene therapy (01.01.2006)
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A heterotypic bystander effect for tumor cell killing after adeno-associated virus/phage–mediated, vascular-targeted suicide gene transfer
Trepel, Martin, Stoneham, Charlotte A, Eleftherohorinou, Hariklia, Mazarakis, Nicholas D, Pasqualini, Renata, Arap, Wadih, Hajitou, Amin
Published in Molecular cancer therapeutics (01.08.2009)
Published in Molecular cancer therapeutics (01.08.2009)
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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
Azzouz, Mimoun, Martin-Rendon, Enca, Barber, Robert D, Mitrophanous, Kyriacos A, Carter, Emma E, Rohll, Jonathan B, Kingsman, Susan M, Kingsman, Alan J, Mazarakis, Nicholas D
Published in The Journal of neuroscience (01.12.2002)
Published in The Journal of neuroscience (01.12.2002)
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Lentiviral vector expressing retinoic acid receptor β2 promotes recovery of function after corticospinal tract injury in the adult rat spinal cord
Yip, Ping K., Wong, Liang-Fong, Pattinson, Damian, Battaglia, Anna, Grist, John, Bradbury, Elizabeth J., Maden, Malcolm, McMahon, Stephen B., Mazarakis, Nicholas D.
Published in Human molecular genetics (01.11.2006)
Published in Human molecular genetics (01.11.2006)
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Lentivector-mediated delivery of GDNF protects complex motor functions relevant to human Parkinsonism in a rat lesion model
Dowd, Eilís, Monville, Christelle, Torres, Eduardo M., Wong, Liang-Fong, Azzouz, Mimoun, Mazarakis, Nicholas D., Dunnett, Stephen B.
Published in The European journal of neuroscience (01.11.2005)
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Neuron-specific activation of necroptosis signaling in multiple sclerosis cortical grey matter
Picon, Carmen, Jayaraman, Anusha, James, Rachel, Beck, Catriona, Gallego, Patricia, Witte, Maarten E., van Horssen, Jack, Mazarakis, Nicholas D., Reynolds, Richard
Published in Acta neuropathologica (01.04.2021)
Published in Acta neuropathologica (01.04.2021)
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PPARγ-coactivator-1α gene transfer reduces neuronal loss and amyloid-β generation by reducing β-secretase in an Alzheimer’s disease model
Katsouri, Loukia, Lim, Yau M., Blondrath, Katrin, Eleftheriadou, Ioanna, Lombardero, Laura, Birch, Amy M., Mirzaei, Nazanin, Irvine, Elaine E., Mazarakis, Nicholas D., Sastre, Magdalena
Published in Proceedings of the National Academy of Sciences - PNAS (25.10.2016)
Published in Proceedings of the National Academy of Sciences - PNAS (25.10.2016)
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Transduction Patterns of Pseudotyped Lentiviral Vectors in the Nervous System
Wong, Liang-Fong, Azzouz, Mimoun, Walmsley, Lucy E, Askham, Zoe, Wilkes, Fraser J, Mitrophanous, Kyriacos A, Kingsman, Susan M, Mazarakis, Nicholas D
Published in Molecular therapy (01.01.2004)
Published in Molecular therapy (01.01.2004)
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