[HTML][HTML] Anatomical changes and pathophysiology of the brain in mucopolysaccharidosis disorders

BW Bigger, DJ Begley, D Virgintino… - Molecular genetics and …, 2018 - Elsevier
Mucopolysaccharidosis (MPS) disorders are caused by deficiencies in lysosomal enzymes,
leading to impaired glycosaminoglycan (GAG) degradation. The resulting GAG accumulation …

Mucopolysaccharide diseases: a complex interplay between neuroinflammation, microglial activation and adaptive immunity

LD Archer, KJ Langford-Smith, BW Bigger… - Journal of inherited …, 2014 - Springer
Brian W. Bigger … Archer LD, Langford-Smith KJ, Critchley WR, Bigger BW, Fildes JE (2013)
Characterisation of the T cell and dendritic cell repertoire in a murine model of …

The bone marrow functionally contributes to liver fibrosis

FP Russo, MR Alison, BW Bigger, E Amofah, A Florou… - Gastroenterology, 2006 - Elsevier
Background & Aims Bone marrow (BM) cells may transdifferentiate into or fuse with organ
parenchymal cells. BM therapy shows promise in murine models of cirrhosis, and clinical trials …

The role of innate immunity in mucopolysaccharide diseases

H Parker, BW Bigger - Journal of neurochemistry, 2019 - Wiley Online Library
Mucopolysaccharidoses are lysosomal storage disorders characterised by accumulation of
abnormal pathological glycosaminoglycans, cellular dysfunction and widespread …

[HTML][HTML] Oncogenesis following delivery of a nonprimate lentiviral gene therapy vector to fetal and neonatal mice

…, SMK Buckley, N Dighe, AT Ruthe, A Mistry, B Bigger… - Molecular Therapy, 2005 - cell.com
Gene therapy by use of integrating vectors carrying therapeutic transgene sequences offers
the potential for a permanent cure of genetic diseases by stable vector insertion into the …

[HTML][HTML] Neuropathology in mouse models of mucopolysaccharidosis type I, IIIA and IIIB

…, JE Wraith, RF Wynn, CLR Merry, BW Bigger - PloS one, 2012 - journals.plos.org
Mucopolysaccharide diseases (MPS) are caused by deficiency of glycosaminoglycan (GAG)
degrading enzymes, leading to GAG accumulation. Neurodegenerative MPS diseases …

[HTML][HTML] An araC-controlled bacterialcre expression system to produce DNA minicircle vectors for nuclear and mitochondrial gene therapy

BW Bigger, O Tolmachov, JM Collombet… - Journal of Biological …, 2001 - ASBMB
The presence of CpG motifs and their associated sequences in bacterial DNA causes an
immunotoxic response following the delivery of these plasmid vectors into mammalian hosts. …

[HTML][HTML] Genistein improves neuropathology and corrects behaviour in a mouse model of neurodegenerative metabolic disease

…, RF Wynn, JE Wraith, G Wegrzyn, BW Bigger - PloS one, 2010 - journals.plos.org
Background Neurodegenerative metabolic disorders such as mucopolysaccharidosis IIIB (MPSIIIB
or Sanfilippo disease) accumulate undegraded substrates in the brain and are often …

Improved metabolic correction in patients with lysosomal storage disease treated with hematopoietic stem cell transplant compared with enzyme replacement therapy

…, K Tylee, M Thornley, HJ Church, BW Bigger - The Journal of …, 2009 - Elsevier
We compared substrate reduction in patients with lysosomal storage disorder treated with
hematopoietic stem cell transplant and found that it was significantly reduced compared with …

Neuroinflammation, mitochondrial defects and neurodegeneration in mucopolysaccharidosis III type C mouse model

…, Z Hájková, M Tesařová, H Hansíková, BW Bigger… - Brain, 2015 - academic.oup.com
Severe progressive neurological paediatric disease mucopolysaccharidosis III type C is
caused by mutations in the HGSNAT gene leading to deficiency of acetyl-CoA: α-glucosaminide …