Stuart Ellison

Medicine · Health Sciences

15h-index724citations41works2.42yr avg
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Research Topics

Lysosomal Storage Disorders Research(18), Virus-based gene therapy research(7), Neurogenetic and Muscular Disorders Research(7), Cell Adhesion Molecules Research(4), Signaling Pathways in Disease(4)

Publications41 total

Sustained biochemical correction and improved neurological outcomes at 36-months post hematopoietic stem cell gene therapy for Sanfilippo syndrome
Molecular Genetics and Metabolism·2026Open Access
Long-term HSC gene therapy in mucopolysaccharidosis type IIIB mice corrects disease with no evidence of insertional mutagenesis despite high vector copy numbers
Molecular Genetics and Metabolism·2025
Systemic immune challenge exacerbates neurodegeneration in a model of neurological lysosomal disease
EMBO Molecular Medicine·2024Open Access
Design and validation of a GMP stem cell manufacturing protocol for MPSII hematopoietic stem cell gene therapy
Molecular Therapy — Methods & Clinical Development·2024· 6 citedOpen Access
Systemic inflammation leads to neuronal loss and exacerbates behavioural deficits in a mouse model of MPS IIIA
Molecular Genetics and Metabolism·2024
Clinical outcomes and sustained biochemical engraftment following ex-vivo autologous stem cell gene therapy for mucopolysaccharidosis type IIIA
Molecular Genetics and Metabolism·2024· 3 cited
Optimised protocols to generate high titre lentiviral vectors using a novel transfection agent enabling extended HEK293T culture following transient transfection and suspension culture
Journal of Virological Methods·2024· 8 citedOpen Access
Sustained long-term disease correction in a murine model of MPSII following stem cell gene therapy
Molecular Therapy — Methods & Clinical Development·2023· 7 citedOpen Access
Fusion of Rabies Virus Glycoprotein or gh625 to Iduronate-2-Sulfatase for the Treatment of Mucopolysaccharidosis Type II
Human Gene Therapy·2023· 2 cited
Advances in therapies for neurological lysosomal storage disorders
Journal of Inherited Metabolic Disease·2023· 45 citedOpen Access
Validation of a GMP stem cell gene therapy manufacturing process for mucopolysaccharidosis type II (MPS II) in preparation for an approved phase I/II clinical trial
Molecular Genetics and Metabolism·2023
Identification of novel peptide sequences to fuse to iduronate-2-sulfatase for the treatment of Hunter syndrome
Molecular Genetics and Metabolism·2023
Sustained biochemical engraftment and early clinical outcomes following autologous stem cell gene therapy for mucopolysaccharidosis type IIIA
Molecular Genetics and Metabolism·2023· 2 citedOpen Access
Biochemical Engraftment and Clinical Outcomes Following Ex-Vivo Autologous Stem Cell Gene Therapy for Mucopolysaccharidosis Type IIIA
Blood·2022· 2 cited
Clinical trial update: Ex-vivo autologous hematopoietic stem cell gene therapy in MPS IIIA
Molecular Genetics and Metabolism·2022
Ex-vivo autologous stem cell gene therapy for MPS II (Hunter syndrome)
Molecular Genetics and Metabolism·2022
Enhanced transduction and immunophenotyping demonstrates preclinical safety and efficacy of hematopoietic stem cell gene therapy for mucopolysaccharidosis II using an IDS.ApoEII brain targeted therapy
Molecular Genetics and Metabolism·2022
Ex-vivo autologous stem cell gene therapy clinical trial for mucopolysaccharidosis type IIIA: Update on phase I/II clinical trial
Molecular Genetics and Metabolism·2021· 3 cited
Ex-Vivo Autologous Stem Cell Gene Therapy Clinical Trial for Mucopolysaccharidosis Type IIIA: Trial in Progress - NCT04201405
Blood·2020· 7 cited
Actinomycin D downregulates Sox2 and improves survival in preclinical models of recurrent glioblastoma
Neuro-Oncology·2020· 37 citedOpen Access
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Frequent Co-authors

Brian Bigger(28), Rebecca Holley(15), Simon Jones(12), Robert Wynn(12), Helen Parker(9), Shaun Wood(9), Karen Buckland(9), Claire O’Leary(8), Aiyin Liao(8), Jane Kinsella(7), Claire Booth(7), Heather J. Church(7), Stewart Rust(6), Helena Lee(6), Fiona L. Wilkinson(5), Hélène F.E. Gleitz(5), Jessica Taylor(5), Antonio Trabalza(5), Laura Ford(5), Yuko Ishikawa Learmonth(5)