List of works - Brian Bigger

A non-myeloablative chimeric mouse model accurately defines microglia and macrophage contribution in glioma

scientific article published on 20 April 2018

A novel adeno-associated virus capsid with enhanced neurotropism corrects a lysosomal transmembrane enzyme deficiency.

scientific article published in July 2018

Actigraphic investigation of circadian rhythm functioning and activity levels in children with mucopolysaccharidosis type III (Sanfilippo syndrome)

scientific article

An araC-controlled bacterial cre expression system to produce DNA minicircle vectors for nuclear and mitochondrial gene therapy.

scientific article published on 13 April 2001

An investigation of the middle and late behavioural phenotypes of Mucopolysaccharidosis Type-III.

scientific article

Analytical strategies for characterization of oxysterol lipidomes: liver X receptor ligands in plasma.

scientific article published on 27 July 2012

Anatomical changes and pathophysiology of the brain in mucopolysaccharidosis disorders

scientific article published on 10 August 2018

Assessment of sleep in children with mucopolysaccharidosis type III.

scientific article

Biomarker responses correlate with antibody status in mucopolysaccharidosis type I patients on long-term enzyme replacement therapy

scientific article published on 29 October 2014

Brain-targeted stem cell gene therapy corrects mucopolysaccharidosis type II via multiple mechanisms.

scientific article published in July 2018

Busulfan Conditioning Enhances Engraftment of Hematopoietic Donor-derived Cells in the Brain Compared With Irradiation ⬇️

scientific article published on February 19, 2013

Central and haematopoietic interleukin-1 both contribute to ischaemic brain injury in mice

scientific article

Characterisation of the T cell and dendritic cell repertoire in a murine model of mucopolysaccharidosis I (MPS I)

article

Chronic norovirus infection in pediatric hematopoietic stem cell transplant recipients: a cause of prolonged intestinal failure requiring intensive nutritional support

scientific article published on 19 April 2011

Circadian rhythm and suprachiasmatic nucleus alterations in the mouse model of mucopolysaccharidosis IIIB.

scientific article published on 4 February 2010

Comparison of HIV- and EIAV-based vectors on their efficiency in transducing murine and human hematopoietic repopulating cells

scientific article published on September 2005

Corrigendum to "Oncogenesis Following Delivery of a Nonprimate Lentiviral Gene Therapy Vector to Fetal and Neonatal Mice".

scientific article published on 3 November 2005

Delivering Hematopoietic Stem Cell Gene Therapy Treatments for Neurological Lysosomal Diseases

scientific article published on 23 August 2018

Development of a self-assembling nuclear targeting vector system based on the tetracycline repressor protein.

scientific article published on 7 November 2003

Enzyme replacement therapy prior to haematopoietic stem cell transplantation in Mucopolysaccharidosis Type I: 10 year combined experience of 2 centres

scientific article published on 27 January 2016

Evaluation of heparin cofactor II-thrombin complex as a biomarker on blood spots from mucopolysaccharidosis I, IIIA and IIIB mice

scientific article published on 23 October 2009

Female mucopolysaccharidosis IIIA mice exhibit hyperactivity and a reduced sense of danger in the open field test

scientific article

Gene therapy of cystic fibrosis; review of completed clinical studies

scientific article published on 01 October 2001

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

scientific article

Haematopoietic stem cell gene therapy with IL-1Ra rescues cognitive loss in mucopolysaccharidosis IIIA

scientific article published on 14 February 2020

Hematopoietic Stem Cell and Gene Therapy Corrects Primary Neuropathology and Behavior in Mucopolysaccharidosis IIIA Mice ⬇️

scientific article published on May 1, 2012

Hematopoietic stem cell transplantation improves the high incidence of neutralizing allo-antibodies observed in Hurler's syndrome after pharmacological enzyme replacement therapy.

scientific article

Heparan sulfate inhibits hematopoietic stem and progenitor cell migration and engraftment in mucopolysaccharidosis I.

scientific article

Hepatic stem cells: from inside and outside the liver?

scientific article published on February 2004

High content screening of patient-derived cell lines highlights the potential of non-standard chemotherapeutic agents for the treatment of glioblastoma

scientific article published on 2 March 2018

High dose genistein in Sanfilippo syndrome: A randomised controlled trial

scientific article published in 2021

Identification of age-dependent motor and neuropsychological behavioural abnormalities in a mouse model of Mucopolysaccharidosis Type II

scientific article

Identification of unusual oxysterols and bile acids with 7-oxo or 3β,5α,6β-trihydroxy functions in human plasma by charge-tagging mass spectrometry with multistage fragmentation.

scientific article published on 6 April 2018

Immune activation or immunomodulation in the brains of MPS IIIB mice? Commentary on "Innate and adaptive immune activation in the brain of MPS IIIB mouse model".

scientific article published in February 2010

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

scientific article published on 01 April 2009

In vivo T-cell depletion using alemtuzumab in family and unrelated donor transplantation for pediatric non-malignant disease achieves engraftment with low incidence of graft vs. host disease

scientific article published on 26 December 2014

Is it congenital or acquired von Willebrands disease?

scientific article published on 7 November 2014

Long-term transgene expression by administration of a lentivirus-based vector to the fetal circulation of immuno-competent mice.

scientific article published in August 2003

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

scientific article published on 27 November 2017

Metabolism of Non-Enzymatically Derived Oxysterols: Clues from sterol metabolic disorders

scientific article published on 19 April 2019

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

scientific article published on 08 May 2013

Mucopolysaccharidosis type I, unique structure of accumulated heparan sulfate and increased N-sulfotransferase activity in mice lacking α-l-iduronidase.

scientific article

Murine leukemia following irradiation conditioning for transplantation of lentivirally-modified hematopoietic stem cells

scientific article published in April 2007

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

scientific article

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

scientific article

Non-myeloablative busulfan chimeric mouse models are less pro-inflammatory than head-shielded irradiation for studying immune cell interactions in brain tumours

scientific article published on 05 February 2019

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

scientific article

Parental social support, coping strategies, resilience factors, stress, anxiety and depression levels in parents of children with MPS III (Sanfilippo syndrome) or children with intellectual disabilities (ID).

scientific article published on 15 November 2012

Permanent phenotypic correction of hemophilia B in immunocompetent mice by prenatal gene therapy.

scientific article published in July 2004

Plasma and urinary levels of dermatan sulfate and heparan sulfate derived disaccharides after long-term enzyme replacement therapy (ERT) in MPS I: correlation with the timing of ERT and with total urinary excretion of glycosaminoglycans.

scientific article published on 19 September 2012

Post-transplant laronidase augmentation for children with Hurler syndrome: biochemical outcomes

scientific article published on 01 October 2019

Pre-clinical Safety and Efficacy of Lentiviral Vector-Mediated Stem Cell Gene Therapy for the Treatment of Mucopolysaccharidosis IIIA

article

Quantitative charge-tags for sterol and oxysterol analysis

scientific article published on 15 December 2014

RecET driven chromosomal gene targeting to generate a RecA deficient Escherichia coli strain for Cre mediated production of minicircle DNA.

scientific article

Recommendations on clinical trial design for treatment of Mucopolysaccharidosis Type III.

scientific article

Signal one and two blockade are both critical for non-myeloablative murine HSCT across a major histocompatibility complex barrier

scientific article

Sleep disordered breathing in mucopolysaccharidosis I: a multivariate analysis of patient, therapeutic and metabolic correlators modifying long term clinical outcome.

scientific article published on 10 April 2015

Sterols and oxysterols in plasma from Smith-Lemli-Opitz syndrome patients

scientific article

Strategies for the Induction of Immune Tolerance to Enzyme Replacement Therapy in Mucopolysaccharidosis Type I

scientific article published on 02 March 2019

Substrate accumulation and extracellular matrix remodelling promote persistent upper airway disease in mucopolysaccharidosis patients on enzyme replacement therapy

scientific article published in PLoS ONE

Successful allogeneic bone marrow transplant for Niemann-Pick disease type C2 is likely to be associated with a severe 'graft versus substrate' effect.

scientific article published in December 2010

The bone marrow functionally contributes to liver fibrosis

scientific article published in May 2006

The fetal mouse is a sensitive genotoxicity model that exposes lentiviral-associated mutagenesis resulting in liver oncogenesis

scientific article published on 13 November 2012

The impact of the immune system on the safety and efficiency of enzyme replacement therapy in lysosomal storage disorders

scientific article published on 16 February 2016

The role of antibodies in enzyme treatments and therapeutic strategies

scientific article published on 24 January 2015

The role of innate immunity in mucopolysaccharide diseases

scientific article published on 13 December 2018

Trial and error: how the unclonable human mitochondrial genome was cloned in yeast.

scientific article published on 9 July 2011

List of works by Brian Bigger

A non-myeloablative chimeric mouse model accurately defines microglia and macrophage contribution in glioma

A novel adeno-associated virus capsid with enhanced neurotropism corrects a lysosomal transmembrane enzyme deficiency.

Actigraphic investigation of circadian rhythm functioning and activity levels in children with mucopolysaccharidosis type III (Sanfilippo syndrome)

An araC-controlled bacterial cre expression system to produce DNA minicircle vectors for nuclear and mitochondrial gene therapy.

An investigation of the middle and late behavioural phenotypes of Mucopolysaccharidosis Type-III.

Analytical strategies for characterization of oxysterol lipidomes: liver X receptor ligands in plasma.

Anatomical changes and pathophysiology of the brain in mucopolysaccharidosis disorders

Assessment of sleep in children with mucopolysaccharidosis type III.

Biomarker responses correlate with antibody status in mucopolysaccharidosis type I patients on long-term enzyme replacement therapy

Brain-targeted stem cell gene therapy corrects mucopolysaccharidosis type II via multiple mechanisms.

Busulfan Conditioning Enhances Engraftment of Hematopoietic Donor-derived Cells in the Brain Compared With Irradiation ⬇️

Central and haematopoietic interleukin-1 both contribute to ischaemic brain injury in mice

Characterisation of the T cell and dendritic cell repertoire in a murine model of mucopolysaccharidosis I (MPS I)

Chronic norovirus infection in pediatric hematopoietic stem cell transplant recipients: a cause of prolonged intestinal failure requiring intensive nutritional support

Circadian rhythm and suprachiasmatic nucleus alterations in the mouse model of mucopolysaccharidosis IIIB.

Comparison of HIV- and EIAV-based vectors on their efficiency in transducing murine and human hematopoietic repopulating cells

Corrigendum to "Oncogenesis Following Delivery of a Nonprimate Lentiviral Gene Therapy Vector to Fetal and Neonatal Mice".

Delivering Hematopoietic Stem Cell Gene Therapy Treatments for Neurological Lysosomal Diseases

Development of a self-assembling nuclear targeting vector system based on the tetracycline repressor protein.

Enzyme replacement therapy prior to haematopoietic stem cell transplantation in Mucopolysaccharidosis Type I: 10 year combined experience of 2 centres

Evaluation of heparin cofactor II-thrombin complex as a biomarker on blood spots from mucopolysaccharidosis I, IIIA and IIIB mice

Female mucopolysaccharidosis IIIA mice exhibit hyperactivity and a reduced sense of danger in the open field test

Gene therapy of cystic fibrosis; review of completed clinical studies

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

Haematopoietic stem cell gene therapy with IL-1Ra rescues cognitive loss in mucopolysaccharidosis IIIA

Hematopoietic Stem Cell and Gene Therapy Corrects Primary Neuropathology and Behavior in Mucopolysaccharidosis IIIA Mice ⬇️

Hematopoietic stem cell transplantation improves the high incidence of neutralizing allo-antibodies observed in Hurler's syndrome after pharmacological enzyme replacement therapy.

Heparan sulfate inhibits hematopoietic stem and progenitor cell migration and engraftment in mucopolysaccharidosis I.

Hepatic stem cells: from inside and outside the liver?

High content screening of patient-derived cell lines highlights the potential of non-standard chemotherapeutic agents for the treatment of glioblastoma

High dose genistein in Sanfilippo syndrome: A randomised controlled trial

Identification of age-dependent motor and neuropsychological behavioural abnormalities in a mouse model of Mucopolysaccharidosis Type II

Identification of unusual oxysterols and bile acids with 7-oxo or 3β,5α,6β-trihydroxy functions in human plasma by charge-tagging mass spectrometry with multistage fragmentation.

Immune activation or immunomodulation in the brains of MPS IIIB mice? Commentary on "Innate and adaptive immune activation in the brain of MPS IIIB mouse model".

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

In vivo T-cell depletion using alemtuzumab in family and unrelated donor transplantation for pediatric non-malignant disease achieves engraftment with low incidence of graft vs. host disease

Is it congenital or acquired von Willebrands disease?

Long-term transgene expression by administration of a lentivirus-based vector to the fetal circulation of immuno-competent mice.

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

Metabolism of Non-Enzymatically Derived Oxysterols: Clues from sterol metabolic disorders

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

Mucopolysaccharidosis type I, unique structure of accumulated heparan sulfate and increased N-sulfotransferase activity in mice lacking α-l-iduronidase.

Murine leukemia following irradiation conditioning for transplantation of lentivirally-modified hematopoietic stem cells

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

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

Non-myeloablative busulfan chimeric mouse models are less pro-inflammatory than head-shielded irradiation for studying immune cell interactions in brain tumours

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

Parental social support, coping strategies, resilience factors, stress, anxiety and depression levels in parents of children with MPS III (Sanfilippo syndrome) or children with intellectual disabilities (ID).

Permanent phenotypic correction of hemophilia B in immunocompetent mice by prenatal gene therapy.

Plasma and urinary levels of dermatan sulfate and heparan sulfate derived disaccharides after long-term enzyme replacement therapy (ERT) in MPS I: correlation with the timing of ERT and with total urinary excretion of glycosaminoglycans.

Post-transplant laronidase augmentation for children with Hurler syndrome: biochemical outcomes

Pre-clinical Safety and Efficacy of Lentiviral Vector-Mediated Stem Cell Gene Therapy for the Treatment of Mucopolysaccharidosis IIIA

Quantitative charge-tags for sterol and oxysterol analysis

RecET driven chromosomal gene targeting to generate a RecA deficient Escherichia coli strain for Cre mediated production of minicircle DNA.

Recommendations on clinical trial design for treatment of Mucopolysaccharidosis Type III.

Signal one and two blockade are both critical for non-myeloablative murine HSCT across a major histocompatibility complex barrier

Sleep disordered breathing in mucopolysaccharidosis I: a multivariate analysis of patient, therapeutic and metabolic correlators modifying long term clinical outcome.

Sterols and oxysterols in plasma from Smith-Lemli-Opitz syndrome patients

Strategies for the Induction of Immune Tolerance to Enzyme Replacement Therapy in Mucopolysaccharidosis Type I

Substrate accumulation and extracellular matrix remodelling promote persistent upper airway disease in mucopolysaccharidosis patients on enzyme replacement therapy

Successful allogeneic bone marrow transplant for Niemann-Pick disease type C2 is likely to be associated with a severe 'graft versus substrate' effect.

The bone marrow functionally contributes to liver fibrosis

The fetal mouse is a sensitive genotoxicity model that exposes lentiviral-associated mutagenesis resulting in liver oncogenesis

The impact of the immune system on the safety and efficiency of enzyme replacement therapy in lysosomal storage disorders

The role of antibodies in enzyme treatments and therapeutic strategies

The role of innate immunity in mucopolysaccharide diseases

Trial and error: how the unclonable human mitochondrial genome was cloned in yeast.