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18 results on '"Ellison, Stuart M"'

1. Systemic inflammation leads to neuronal loss and exacerbates behavioural deficits in a mouse model of MPS IIIA

Author

Mandolfo, Oriana, primary, Parker, Helen, additional, Aguado, Èlia, additional, Learmonth, Yuko Ishikawa, additional, Liao, Aiyin, additional, O'Leary, Claire, additional, Ellison, Stuart M., additional, Taylor, Jessica, additional, Wood, Shaun R., additional, Holley, Rebecca, additional, Boutin, Herve, additional, and Bigger, Brian, additional

Published
2024
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2. Clinical outcomes and sustained biochemical engraftment following ex-vivo autologous stem cell gene therapy for mucopolysaccharidosis type IIIA

Author

Jones, Simon, primary, Kinsella, Jane, additional, Holley, Rebecca J., additional, Potter, Jane, additional, Booth, Claire, additional, Buckland, Karen, additional, Rust, Stewart, additional, Bromley, Rebecca, additional, Church, Heather J., additional, Lee, Helena, additional, Ford, Laura, additional, Learmonth, Yuko Ishikawa, additional, Searle, Rachel, additional, Ellison, Stuart M., additional, Wynn, Robert, additional, and Bigger, Brian, additional

Published
2024
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4. Sustained biochemical engraftment and early clinical outcomes following autologous stem cell gene therapy for mucopolysaccharidosis type IIIA

Author

Jones, Simon, primary, Kinsella, Jane, additional, Holley, Rebecca, additional, Potter, Jane, additional, Booth, Claire, additional, Buckland, Karen, additional, Rust, Stewart, additional, Church, Heather J., additional, Brammeier, Kathryn L., additional, Lee, Helena, additional, Ford, Laura, additional, Ellison, Stuart M., additional, Bigger, Brian, additional, and Wynn, Robert, additional

Published
2023
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9. Pre-clinical Safety and Efficacy of Lentiviral Vector-Mediated Ex Vivo Stem Cell Gene Therapy for the Treatment of Mucopolysaccharidosis IIIA

Author

Ellison, Stuart M., primary, Liao, Aiyin, additional, Wood, Shaun, additional, Taylor, Jessica, additional, Youshani, Amir Saam, additional, Rowlston, Sam, additional, Parker, Helen, additional, Armant, Myriam, additional, Biffi, Alessandra, additional, Chan, Lucas, additional, Farzaneh, Farzin, additional, Wynn, Rob, additional, Jones, Simon A., additional, Heal, Paul, additional, Gaspar, H. Bobby, additional, and Bigger, Brian W., additional

Published
2019
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10. Pre-clinical safety and efficacy evaluation of GMP lentiviral vector in preparation for a clinical trial of hematopoietic stem cell gene therapy in MPS IIIA

Author

Ellison, Stuart M., primary, Wood, Shaun, additional, Sergijenko, Ana, additional, Armant, Myriam, additional, Chan, Lucas, additional, Farzaneh, Farzin, additional, Jones, Simon, additional, Wynn, Rob, additional, Gaspar, Bobby, additional, Biffi, Alessandra, additional, and Bigger, Brian, additional

Published
2018
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11. Macrophage enzyme and reduced inflammation drive brain correction of mucopolysaccharidosis IIIB by stem cell gene therapy

Author

Holley, Rebecca J, primary, Ellison, Stuart M, additional, Fil, Daniel, additional, O’Leary, Claire, additional, McDermott, John, additional, Senthivel, Nishanthi, additional, Langford-Smith, Alexander W W, additional, Wilkinson, Fiona L, additional, D’Souza, Zelpha, additional, Parker, Helen, additional, Liao, Aiyin, additional, Rowlston, Samuel, additional, Gleitz, Hélène F E, additional, Kan, Shih-Hsin, additional, Dickson, Patricia I, additional, and Bigger, Brian W, additional

Published
2017
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12. 364. Neurological Correction of Mucopolysaccharidosis IIIB Mice by Haematopoietic Stem Cell Gene Therapy

Author

Ellison, Stuart M., primary, Holley, Rebecca, additional, Fil, Daniel, additional, Dermott, John Mc, additional, Senthivel, Nisha, additional, Langford-Smith, Alex, additional, Wilkinson, Fiona, additional, Jones, Simon, additional, Wynn, Rob, additional, Kan, Shih-hsin, additional, Dickson, Patti, additional, and Bigger, Brian, additional

Published
2016
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14. Vesicular Stomatitis Virus Glycoprotein– and Venezuelan Equine Encephalitis Virus-Derived Glycoprotein–Pseudotyped Lentivirus Vectors Differentially Transduce Corneal Endothelium, Trabecular Meshwork, and Human Photoreceptors

Author

Lipinski, Daniel M., primary, Barnard, Alun R., additional, Issa, Peter Charbel, additional, Singh, Mandeep S., additional, De Silva, Samantha R., additional, Trabalza, Antonio, additional, Eleftheriadou, Ioanna, additional, Ellison, Stuart M., additional, Mazarakis, Nicholas D., additional, and MacLaren, Robert E., additional

Published
2014
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16. Dose-dependent Neuroprotection of VEGF165 in Huntington's Disease Striatum.

Author

Ellison, Stuart M, Trabalza, Antonio, Tisato, Veronica, Pazarentzos, Evangelos, Lee, Shirley, Papadaki, Vasiliki, Goniotaki, Despoina, Morgan, Sarah, Mirzaei, Nazanin, and Mazarakis, Nicholas D

Subjects
*HUNTINGTON disease, *NEURODEGENERATION, *POLYGLUTAMINE, *NEUROBLASTOMA, *NEOVASCULARIZATION, *VASCULAR endothelial growth factors
Abstract

Huntington's disease (HD) is a devastating neurodegenerative disorder caused by abnormal polyglutamine expansion in the huntingtin protein (Exp-Htt). Currently, there are no effective treatments for HD. We used bidirectional lentiviral transfer vectors to generate in vitro and in vivo models of HD and to test the therapeutic potential of vascular endothelial growth factor 165 (VEGF165). Lentiviral-mediated expression of Exp-Htt caused cell death and aggregate formation in human neuroblastoma SH-SY5Y and rat primary striatal cultures. Lentiviral-mediated VEGF165 expression was found to be neuroprotective in both of these models. Unilateral stereotaxic vector delivery of Exp-Htt vector in adult rat striatum led to progressive inclusion formation and striatal neuron loss at 10 weeks post-transduction. Coinjection of a lower dose VEGF165 significantly attenuated DARPP-32+ neuronal loss, enhanced NeuN staining and reduced Exp-Htt aggregation. A tenfold higher dose VEGF165 led to overt neuronal toxicity marked by tissue damage, neovascularization, extensive astrogliosis, vascular leakage, chronic inflammation and distal neuronal loss. No overt behavioral phenotype was observed in these animals. Expression of VEGF165 at this higher dose in the brain of wild-type rats led to early mortality with global neuronal loss. This report raises important safety concerns about unregulated VEGF165 CNS applications.Molecular Therapy (2013); 21 10, 1862-1875. doi:10.1038/mt.2013.132 [ABSTRACT FROM AUTHOR]

Published
2013
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17. Sustained biochemical engraftment and early clinical outcomes following ex-vivo autologous stem cell gene therapy for mucopolysaccharidosis type IIIA.

Author

Jones, Simon, Kinsella, Jane, Holley, Rebecca, Potter, Jane, Booth, Claire, Buckland, Karen, Rust, Stewart, Church, Heather J., Brammeier, Kathryn L., Lee, Helena, Ford, Laura, Ellison, Stuart M., Bigger, Brian, and Wynn, Robert

Subjects
*STEM cell treatment, *TREATMENT effectiveness, *MUCOPOLYSACCHARIDOSIS, *NEUROPEPTIDE Y
Published
2023
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18. Pre-clinical Safety and Efficacy of Lentiviral Vector-Mediated Ex Vivo Stem Cell Gene Therapy for the Treatment of Mucopolysaccharidosis IIIA.

Author

Ellison SM, Liao A, Wood S, Taylor J, Youshani AS, Rowlston S, Parker H, Armant M, Biffi A, Chan L, Farzaneh F, Wynn R, Jones SA, Heal P, Gaspar HB, and Bigger BW

Abstract

Hematopoietic stem cell gene therapy is a promising therapeutic strategy for the treatment of neurological disorders, since transplanted gene-corrected cells can traffic to the brain, bypassing the blood-brain barrier, to deliver therapeutic protein to the CNS. We have developed this approach for the treatment of Mucopolysaccharidosis type IIIA (MPSIIIA), a devastating lysosomal storage disease that causes progressive cognitive decline, leading to death in early adulthood. In a previous pre-clinical proof-of-concept study, we demonstrated neurological correction of MPSIIIA utilizing hematopoietic stem cell gene therapy via a lentiviral vector encoding the SGSH gene. Prior to moving to clinical trial, we have undertaken further studies to evaluate the efficiency of gene transfer into human cells and also safety studies of biodistribution and genotoxicity. Here, we have optimized hCD34 + cell transduction with clinical grade SGSH vector to provide improved pharmacodynamics and cell viability and validated effective scale-up and cryopreservation to generate an investigational medicinal product. Utilizing a humanized NSG mouse model, we demonstrate effective engraftment and biodistribution, with no vector shedding or transmission to germline cells. SGSH vector genotoxicity assessment demonstrated low transformation potential, comparable to other lentiviral vectors in the clinic. This data establishes pre-clinical safety and efficacy of HSCGT for MPSIIIA.

Published
2019
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