Brian Bigger

Medicine · Health Sciences

41h-index5.2kcitations206works2.22yr avg
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Research Topics

Lysosomal Storage Disorders Research(107), Virus-based gene therapy research(28), Trypanosoma species research and implications(27), CRISPR and Genetic Engineering(21), Cellular transport and secretion(20)

Publications205 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
Ex-vivo modification of autologous CD34+ HSPCs using a CD11b-directed lentiviral vector encoding ApoEII-tagged human IDS leads to supraphysiological enzyme activity and biochemical correction of neuronopathic MPS II patients
Molecular Genetics and Metabolism·2026Open Access
Ex-vivo modification of autologous CD34+ HSPCs using a CD11B-directed lentiviral vector encoding APOEII-tagged human IDS leads to supraphysiological enzyme activity and early biochemical correction of neuronopathic mucopolysaccharidosis type II patients
Blood·2025Open Access
Collagen fibril formation at the plasma membrane occurs independently from collagen secretion
Wellcome Open Research·2025Open Access
Designing better gene therapies for lysosomal storage disorders using engineered enzymes
Molecular Therapy·2025Open Access
Induced Pluripotent Stem Cells for the Treatment of Lysosomal Storage Disorders
Journal of Inherited Metabolic Disease·2025Open Access
Neuraminidase 1 secondary deficiency contributes to CNS pathology in neurological mucopolysaccharidoses via brain protein hypersialylation
Journal of Clinical Investigation·2025· 1 citedOpen 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
An evidence-based individual treatment trial with adalimumab in a patient with Morquio syndrome
Molecular Genetics and Metabolism·2025
Development of a novel tool for individual treatment trials in mucopolysaccharidosis
Journal of Inherited Metabolic Disease·2024· 3 citedOpen Access
Lentiviral Vector-Mediated <i>Ex Vivo</i> Hematopoietic Stem Cell Gene Therapy for Mucopolysaccharidosis IVA Murine Model
Human Gene Therapy·2024· 11 citedOpen Access
14 A precision medicine tool for high utilization and quality of individual treatment trials with immunomodulatory drugs in mucopolysaccharidosis
2024Open Access
Generation of a novel immunodeficient mouse model of Mucopolysaccharidosis type IIIA to test human stem cell-based therapies
Molecular Genetics and Metabolism·2024· 3 citedOpen Access
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
Collagen fibril formation at the plasma membrane occurs independently from collagen secretion
2024· 2 citedOpen Access
Secondary deficiency of neuraminidase 1 contributes to CNS pathology in neurological mucopolysaccharidoses via hypersialylation of brain glycoproteins
2024· 1 citedOpen Access
Improving newborn screening test performance for metachromatic leukodystrophy: Recommendation from a pre-pilot study that identified a late-infantile case for treatment
Molecular Genetics and Metabolism·2024· 12 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
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Frequent Co-authors

Simon Jones(17), Robert Wynn(16), Fiona L. Wilkinson(12), Kia Langford‐Smith(12), Rebecca Holley(9), J. E. Wraith(9), Helen Parker(9), Alex Langford-Smith(7), Karen Tylee(7), Heather J. Church(7), Michael Themis(6), Charles Coutelle(6), Jean Mercer(6), Claire O’Leary(6), Arunabha Ghosh(6), Simon N. Waddington(5), Aiyin Liao(5), Catherine L.R. Merry(5), Stuart Ellison(5), Hélène F.E. Gleitz(4)