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10 results on '"Stuart M Ellison"'

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

Author

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

Subjects
Endocrinology, Endocrinology, Diabetes and Metabolism, Genetics, Molecular Biology, Biochemistry
Published
2023
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5. Ex-vivo autologous stem cell gene therapy clinical trial for mucopolysaccharidosis type IIIA: Update on phase I/II clinical trial

Author

Jane Louise Kinsella, Robert Wynn, Adrian J. Thrasher, Heather J. Church, Helena Lee, Laura Ford, Karen Tylee, Claire Booth, Karen F. Buckland, Stuart M. Ellison, Daniel Weisberg, Natalia Izotova, Kathryn L. Brammeier, Atusa Sadegholnejat, Ceri Jones, Simon Jones, Stewart Rust, and Brian W. Bigger

Subjects
business.industry, Endocrinology, Diabetes and Metabolism, Genetic enhancement, Biochemistry, Clinical trial, Endocrinology, Phase i ii, Genetics, Cancer research, Medicine, Stem cell, business, Molecular Biology, Mucopolysaccharidosis Type IIIA, Ex vivo
Published
2021
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6. Interleukin-1 plays a central role in behaviour abnormalities in mucopolysaccharidosis type III (MPS III)

Author

Emmanuel Pinteaux, Brian W. Bigger, Rebecca J. Holley, Stuart M. Ellison, Claire O'Leary, Helen Parker, and Hervé Boutin

Subjects
0301 basic medicine, business.industry, Endocrinology, Diabetes and Metabolism, Interleukin, Mucopolysaccharidosis type III, Biochemistry, 03 medical and health sciences, 030104 developmental biology, Endocrinology, Immunology, Genetics, Medicine, business, Molecular Biology
Published
2018
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7. 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

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

Subjects
0301 basic medicine, Oncology, medicine.medical_specialty, business.industry, Endocrinology, Diabetes and Metabolism, Genetic enhancement, Hematopoietic stem cell, Biochemistry, Viral vector, Clinical trial, 03 medical and health sciences, 030104 developmental biology, Endocrinology, medicine.anatomical_structure, Internal medicine, Genetics, Medicine, Clinical safety, business, Molecular Biology
Published
2018
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8. Neurological correction of mucopolysaccharidosis type IIIB mice by haematopoietic stem cell gene therapy

Author

Patricia I. Dickson, Daniel Fil, N. Senthivel, Alex Langford-Smith, Claire O'Leary, John H McDermott, Rebecca J. Holley, Fiona L. Wilkinson, Stuart M. Ellison, Brian W. Bigger, and S-h Kan

Subjects
Haematopoiesis, Endocrinology, business.industry, Endocrinology, Diabetes and Metabolism, Genetic enhancement, Genetics, Cancer research, Medicine, Stem cell, business, Molecular Biology, Biochemistry, Mucopolysaccharidosis type IIIB
Published
2017
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9. Dose-dependent Neuroprotection of VEGF165 in Huntington's Disease Striatum

Author

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

Subjects
Male, Vascular Endothelial Growth Factor A, Striatum, Animals, Cell Death, Cell Line, Tumor, Cells, Cultured, Corpus Striatum, Disease Models, Animal, Female, Gene Expression Regulation, Genetic Vectors, HEK293 Cells, Humans, Huntington Disease, Lentivirus, Nerve Degeneration, Nerve Tissue Proteins, Neurons, Neuroprotective Agents, Rats, Rats, Sprague-Dawley, Transduction, Genetic, Genetic Therapy, Molecular Biology, Molecular Medicine, Genetics, Drug Discovery3003 Pharmaceutical Science, Pharmacology, Neovascularization, Drug Discovery, Huntingtin Protein, Tumor, Cultured, biology, Anatomy, Astrogliosis, Vascular endothelial growth factor A, Original Article, medicine.symptom, medicine.medical_specialty, Cells, Neuroprotection, NO, Cell Line, Transduction, Huntington's disease, Genetic, Internal medicine, medicine, Animal, medicine.disease, Endocrinology, nervous system, Disease Models, biology.protein, Sprague-Dawley, NeuN
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.

Published
2013

10. Pre-clinical workup of lentiviral mediated stem cell gene therapy for mucopolysaccharidosis type IIIA

Author

Fiona L. Wilkinson, Ana Sergijenko, Rob Wynn, Stuart M. Ellison, Brian W. Bigger, Alex Langford-Smith, and Kia J. Langford-Smith

Subjects
Endocrinology, business.industry, Endocrinology, Diabetes and Metabolism, Genetic enhancement, Genetics, Cancer research, Medicine, Stem cell, business, Molecular Biology, Biochemistry, Mucopolysaccharidosis Type IIIA
Published
2015
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