Dr Brian Bigger PhD

Photograph of Brian Bigger


Stem Cell & Neurotherapies Laboratory
Faculty of Medical and Human Sciences
3.721 Stopford Building
University of Manchester
Manchester M13 9PT, UK


Group leader Stem Cell & Neurotherapies Laboratory


Neurodegenerative metabolic diseases mainly affect children and often lack effective treatments.  Some, such as the lysosomal storage disease, Mucopolysaccharidosis (MPS) I, can already be partly treated by allogeneic transplantation of bone marrow or cord blood derived stem cells from a healthy donor, but there is often a high associated risk of morbidity and mortality when using mismatched transplants.

Dr Bigger’s Stem Cell & Neurotherapies laboratory uses a multidisciplinary approach to investigate stem cell and gene therapies for neurological diseases. It consists of two programmes


MPS lysosomal disease group

  1. Comparative neuropathology and mechanisms of neurodegeneration in models of MPS disease
  2. Clinical development of novel stem cell and gene therapies in neurodegenerative lysosomal diseases
  3. Biomarker development
  4. The therapeutic use of haematopoietic stem cell transplantation in neurodegenerative disease
  5. The use of substrate reducing agents to ameliorate mucopolysaccharide diseases.

The Brain Tumour group

  1. Brain tumour stem cell biology and their interaction with Microglia in vivo
  2. Novel therapies for brain tumours using high throughput drug screening in cancer stem cell lines derived from primary gliomas

We are primarily a research laboratory but have close clinical links, with both the MPS and metabolic disease patients and neurosurgical patients.


Lentiviral transduced eGFP positive GL-261 glioma tumour cells

Image: Lentiviral transduced eGFP positive GL-261 glioma tumour cells implanted orthotopically into the brains of C57BL/6 mice, demonstrating the infiltrative component of microglia in the brain tumour microenvironment. Green: eGFP expressing GL261 cells, Red: Iba-1 microglia/macrophages, Blue: DAPI nuclear stain



2014 - Videolinks

Methodological Knowledge

2013 - Videolinks - Rare Disease Report

2012 - Videolinks


Main Areas of Expertise

  • Lysosomal disease model characterisation and treatment

  • Stem cell biology and treatment of primary brain tumours

  • Haematopoetic stem cell gene therapy and tolerance induction

  • Haematopoietic cell migration and interactions within bone marrow and neurological niches

  • Clinical translation of gene and cell therapies



The laboratory is funded by a programme grant and subsidiary grants from the UK Society for Mucopolysaccharide (MPS) diseases to Dr Bigger

We gratefully acknowledge past and present funders including: The Norah Al Balla foundation, Great Ormond Street Research Chraity, Action Medical Research, Jonah's Just Begun, The Lady Shauna Gosling Trust, The Sanfilippo Children's Research Foundation (Canada), The Ollie G Ball,The National MPS Society (US), The Irish MPS society,  The Children's Bone Marrow Trust, BBSRC, The Manchester Biomedical Research Centre, Department of Health, CMFT NHS trust endowments, The Association for Glycogen Storage Disorders, The Brain Tumour Charity, The GEM appeal, Lois Gosling,

as well as contributions from the MPS societies of Austria, Canada, Germany, Ireland, Japan, Netherlands, Spain, Sweden, Switzerland, UK and USA.


Dr Bigger was awarded a Bachelors degree from the University of Bath in Applied Biology. His PhD was conducted in the Gene Therapy Research Group, Imperial College, London, where he worked with Professor Charles Coutelle on developing a gene delivery vehicle for mitochondrial gene therapy. On completion of his PhD in 2000, Dr Bigger joined Dr Mike Themis, Imperial College, London to work on a Wellcome Trust collaborative project with Cancer Research UK, investigating gene delivery to stem cells for liver diseases. In 2004 he joined Dr Suzanne Watt’s group in Oxford University and the National Blood Service as a Senior Research Scientist to work on mechanisms of stem cell homing. In 2006 Dr Bigger set up the Stem Cell & Neurotherapies laboratory at the University of Manchester and the Royal Manchester Children's hospital.

Collaborators and affiliated staff

Group Members and Affiliated Staff

  • Dr Simon Jones - Consultant, Head of Willink Biochemical Genetics Unit
  • Dr Rob Wynn - Consultant, Head of Bone Marrow Transplantation Unit
  • Dr Becki Holley - Senior Postdoctoral Associate
  • Dr Claire O'Leary - Postdoctoral Associate
  • Dr Stuart Ellison -Postdoctoral Associate
  • Mr Kenny Yu - Speciality Registrar Neurosurgery/PhD student
  • Mr Ricky Pal -Speciality Registrar ENT/MD student
  • Miss Ai Yin Liao - Research Technician/part time PhD
  • Miss Helen Parker -PhD Student
  • Mr Saam Yoshani - PhD Student
  • Miss Helene Gleitz - PhD Student




  • Dr Cathy Merry, University of Manchester. Heparan sulphate and cellular interactions in neurodegenerative diseases
  • Dr Maria Canal, University of Manchester. Circadian profiling in MPS disease
  • Dr James Fildes, University of Manchester. Immunology in lysosomal disease
  • Professsor Nancy Rothwell, University of Manchester. Microglial interactions with brain tumours
  • Mr Ian Kamaly and Mr Omar Pathmanaban - Neurosurgery, Salford Royal NHS Foundation Trust. Brain tumour treatment development
  • Imagen Biotech Ltd


  • Professor Adrian Thrasher and Professor Bobby Gaspar, UCL. Stem cell gene therapy for lysosomal diseases
  • Professor Frits Wijburg, AMC Amsterdam. Biomarker development for lysosomal diseases
  • Professor Alexey Pshezhetsky, Sainte Justine Children's Hospital, Montreal, Canada. Gene therapy for MPSIIIC


Job Opportunities

We offer laboratory placements for motivated high quality project students on Manchester University Medical undergraduate or postgraduate MSc and MRes courses and will consider self-funded internships for excellent non-clinical or clinical candidates wishing to gain laboratory experience in the field.

Funded posts are advertised on the University jobsite and national websites such as www.jobs.ac.uk



  • Bigger, B. W., Saif, M., & Linthorst, G. E. (2015). The role of antibodies in enzyme treatments and therapeutic strategies.Best practice & research. Clinical endocrinology & metabolism, 29(2). DOI:10.1016/j.beem.2015.01.006. Publication link: 176d75cc-86c4-4f85-b218-f4e4ce5da7d4 | PubMed:25987172
  • Crick, P. J., William Bentley, T., Abdel-Khalik, J., Matthews, I., Clayton, P. T., Morris, A. A., ... Griffiths, W. J. (2015). Quantitative charge-tags for sterol and oxysterol analysis.Clinical Chemistry, 61(2). DOI:10.1373/clinchem.2014.231332. Publication link: fdef16b7-23fc-49c5-9230-45555cd43d52 | PubMed:25512642
  • Langereis, E. J., van Vlies, N., Church, H. J., Geskus, R. B., Hollak, C. E. M., Jones, S. A., ... Bigger, B. W. (2015). Biomarker responses correlate with antibody status in mucopolysaccharidosis type I patients on long-term enzyme replacement therapy.Molecular genetics and metabolism, 114(2). DOI:10.1016/j.ymgme.2014.10.012. Publication link: 92b19a40-877b-4275-80f7-34db002294d9 | PubMed:25467058
  • Martins, C., Hůlková, H., Dridi, L., Dormoy-Raclet, V., Grigoryeva, L., Choi, Y., ... Pshezhetsky, A. V. (2015). Neuroinflammation, mitochondrial defects and neurodegeneration in mucopolysaccharidosis III type C mouse model.Brain : a journal of neurology, 138(Pt 2). DOI:10.1093/brain/awu355. Publication link: b764ec04-6cbb-47cc-86fb-80042b0a5507 | PubMed:25567323
  • Mumford, R. A., Mahon, L. V., Jones, S., Bigger, B., Canal, M., & Hare, D. J. (2015). Actigraphic investigation of circadian rhythm functioning and activity levels in children with mucopolysaccharidosis type III (Sanfilippo syndrome).Journal of neurodevelopmental disorders, 7(1). DOI:10.1186/s11689-015-9126-5. Publication link: b8ee5efa-15b7-4708-a332-4e6b84c78670 | PubMed:26388955
  • Pal, A. R., Langereis, E. J., Saif, M. A., Mercer, J., Church, H. J., Tylee, K. L., ... Bigger, B. W. (2015). Sleep disordered breathing in mucopolysaccharidosis I: a multivariate analysis of patient, therapeutic and metabolic correlators modifying long term clinical outcome.Orphanet Journal of Rare Diseases, 10. DOI:10.1186/s13023-015-0255-4. Publication link: ca03692f-0aed-4f50-8899-9c9657b55416 | PubMed:25887468
  • Saif, M. A., Borrill, R., Bigger, B. W., Lee, H., Logan, A., Poulton, K., ... Wynn, R. F. (2015). 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.Pediatric transplantation, 19(2). DOI:10.1111/petr.12416. Publication link: dd2d26f4-9f8b-45f9-9f8b-aa5f5545604c | PubMed:25546609
  • Saif, M. A., Thachil, J., Brown, R., Bigger, B. W., Wynn, R. F., Nash, M., & Hay, C. R. (2015). Is it congenital or acquired von Willebrands disease?. Haemophilia, 21(1), e113-e115. . Publication link: 0bf159a2-3c59-424f-89cb-25b28e07e181


  • Archer, L. D., Langford-Smith, K. J., Bigger, B. W., & Fildes, J. E. (2014). Mucopolysaccharide diseases: A complex interplay between neuroinflammation, microglial activation and adaptive immunity. Journal of Inherited Metabolic Disease, 37(1), 1-12. DOI:10.1007/s10545-013-9613-3. Publication link: a135c897-edbe-469c-91bc-e78ed04473c2 | PubMed:23653226
  • Bigger, B. W., & Wynn, R. F. (2014). Novel approaches and mechanisms in hematopoietic stem cell gene therapy. Discovery Medicine, 17(94). . Publication link: 667bc815-5605-4310-afba-e7f921e2cff8 | PubMed:24759625
  • Cross, E. M., Grant, S., Jones, S., Bigger, B. W., Wraith, J. E., Mahon, L. V., ... Hare, D. J. (2014). An investigation of the middle and late behavioural phenotypes of Mucopolysaccharidosis Type-III.Journal of neurodevelopmental disorders, 6(1). DOI:10.1186/1866-1955-6-46. Publication link: 682a7b46-5e09-43f4-9293-45e9705e4163 | PubMed:25657821
  • Mahon, L. V., Lomax, M., Grant, S., Cross, E., Hare, D. J., Wraith, J. E., ... Canal, M. (2014). Assessment of sleep in children with mucopolysaccharidosis type III. PLoS ONE, 9(2), [e84128]. DOI:10.1371/journal.pone.0084128. Publication link: 72dc1e05-2873-44e8-b40f-5c2dc478391c | PubMed:24504123
  • Watson, H. A., Holley, R. J., Langford-Smith, K. J., Wilkinson, F. L., van Kuppevelt, T. H., Wynn, R. F., ... Bigger, B. W. (2014). Heparan sulfate inhibits hematopoietic stem and progenitor cell migration and engraftment in mucopolysaccharidosis I.The Journal of biological chemistry, 289(52), 36194-36203. DOI:10.1074/jbc.M114.599944. Publication link: 5fd144bd-31f6-4ba8-8baf-bcfd02222025 | PubMed:25359774


  • Archer, L. D., Langford-Smith, K. J., Critchley, W. R., Bigger, B. W., & Fildes, J. E. (2013). Characterisation of the T cell and dendritic cell repertoire in a murine model of mucopolysaccharidosis i (MPS I). Journal of Inherited Metabolic Disease, 36(2), 257-262. DOI:10.1007/s10545-012-9508-8. Publication link: d177ba88-01b6-48c8-a6dd-9197f6b7b536 | PubMed:22773246
  • De Ru, M. H., Van Der Tol, L., Van Vlies, N., Bigger, B. W., Hollak, C. E. M., Ijlst, L., ... Wijburg, F. A. (2013). 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. Journal of Inherited Metabolic Disease, 36(2), 247-255. DOI:10.1007/s10545-012-9538-2. Publication link: 8dbcf60b-b9a0-4de5-a21c-778be86c9a97 | PubMed:22991166
  • Denes, A., Wilkinson, F., Bigger, B., Chu, M., Rothwell, N. J., & Allan, S. M. (2013). Central and haematopoietic interleukin-1 both contribute to ischaemic brain injury in mice. DMM Disease Models and Mechanisms, 6(4), 1043-1048. DOI:10.1242/dmm.011601. Publication link: fe5c07a7-90cf-46bc-ad29-861bbfd72452 | PubMed:23519030
  • Grant, S., Cross, E., Wraith, J. E., Jones, S., Mahon, L., Lomax, M., ... Hare, D. (2013). 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). Journal of Inherited Metabolic Disease, 36(2), 281-291. DOI:10.1007/s10545-012-9558-y. Publication link: 4149f1d8-a1d7-42b9-b882-c31fbf8d46cb | PubMed:23151683
  • Griffiths, W. J., Crick, P. J., Wang, Y., Ogundare, M., Tuschl, K., Morris, A. A., ... Wang, Y. (2013). Analytical strategies for characterization of oxysterol lipidomes: Liver X receptor ligands in plasma. Free Radical Biology and Medicine, 59, 69-84. DOI:10.1016/j.freeradbiomed.2012.07.027. Publication link: 1468e36a-78f8-4951-9b6e-cf5e268c3670 | PubMed:22846477
  • Langford-Smith, K. J., Sandiford, Z., Langford-Smith, A., Wilkinson, F. L., Jones, S. A., Wraith, J. E., ... Bigger, B. W. (2013). Signal One and Two Blockade Are Both Critical for Non-Myeloablative Murine HSCT across a Major Histocompatibility Complex Barrier. PLoS ONE, 8(10), [e77632]. DOI:10.1371/journal.pone.0077632. Publication link: f6f7f231-7170-4a49-88dc-04fcea35ed15 | PubMed:24147041
  • Nowrouzi, A., Cheung, W. T., Li, T., Zhang, X., Arens, A., Paruzynski, A., ... Themis, M. (2013). The fetal mouse is a sensitive genotoxicity model that exposes lentiviral-associated mutagenesis resulting in liver oncogenesis. Molecular Therapy, 21(2), 324-337. DOI:10.1038/mt.2012.224. Publication link: 2c2ce6f6-df3d-4531-9417-11bfc2f1817d
  • Sergijenko, A., Langford-Smith, A., Liao, A. Y., Pickford, C. E., McDermott, J., Nowinski, G., ... Bigger, B. W. (2013). Myeloid/Microglial driven autologous hematopoietic stem cell gene therapy corrects a neuronopathic lysosomal disease. Molecular Therapy, 21(10), 1938-1949. DOI:10.1038/mt.2013.141. Publication link: 92d80ae2-89e6-48eb-a53c-d742191871c8 | PubMed:23748415
  • Wilkinson, F. L., Sergijenko, A., Langford-Smith, K. J., Malinowska, M., Wynn, R. F., & Bigger, B. W. (2013). Busulfan conditioning enhances engraftment of hematopoietic donor-derived cells in the brain compared with irradiation. Molecular Therapy, 21(4), 868-876. DOI:10.1038/mt.2013.29. Publication link: 556b6ab0-cb43-402a-b223-bda498964c52 | PubMed:23423338


  • Langford-Smith, A., Wilkinson, F. L., Langford-Smith, K. J., Holley, R. J., Sergijenko, A., Howe, S. J., ... Bigger, B. W. (2012). Hematopoietic stem cell and gene therapy corrects primary neuropathology and behavior in mucopolysaccharidosis IIIA mice. Molecular Therapy, 20(8), 1610-1621. DOI:10.1038/mt.2012.82. Publication link: ea8ca6d7-545f-4a88-bb23-d9d423626ee8 | PubMed:22547151
  • Saif, M., Bigger, B., Brookes, K., Mercer, J., Tylee, K. L., Church, H. J., ... Wynn, R. F. (2012). Hematopoietic stem cell transplantation improves the high incidence of neutralizing allo-antibodies observed in Hurler's syndrome after pharmacological enzyme replacement therapy. Haematologica, 97(9), 1320-1328. DOI: 10.3324/haematol.2011.058644. Publication link: 5876bd70-cfcc-4fd9-97d4-7c7f3fce0902 | PubMed:22371174
  • Wilkinson, F. L., Holley, R. J., Langford-Smith, K. J., Badrinath, S., Liao, A., Langford-Smith, A., ... Bigger, B. W. (2012). Neuropathology in mouse models of mucopolysaccharidosis type I, IIIA and IIIB. P L o S One, 7(4), [e35787]. DOI:10.1371/journal.pone.0035787. Publication link: 464c42c3-0684-4a80-8ba7-aca234835a54 | PubMed:22558223


  • Bigger, B. W., Liao, A. Y., Sergijenko, A., & Coutelle, C. (2011). Trial and error: How the unclonable human mitochondrial genome was cloned in yeast. Pharmaceutical Research, 28(11), 2863-2870. DOI:10.1007/s11095-011-0527-1. Publication link: 88ff1b94-f9ad-4c6b-b892-d96f6f323e8a | PubMed:21739320
  • Holley, R. J., Deligny, A., Wei, W., Watson, H. A., Niñonuevo, M. R., Dagälv, A., ... Merry, C. L. R. (2011). Mucopolysaccharidosis type I, unique structure of accumulated heparan sulfate and increased N-sulfotransferase activity in mice lacking α-L-iduronidase. Journal of Biological Chemistry, 286(43), 37515-37524. DOI:10.1074/jbc.M111.287474. Publication link: 74b3b39c-8648-418b-b6e6-0530710d4d88 | PubMed:21873421
  • Langford-Smith, A., Langford-Smith, K. J., Jones, S. A., Wynn, R. F., Wraith, J. E., Wilkinson, F. L., & Bigger, B. W. (2011). Female mucopolysaccharidosis IIIA mice exhibit hyperactivity and a reduced sense of danger in the open field test. PLoS ONE, 6(10), [e25717]. DOI:10.1371/journal.pone.0025717. Publication link: 4b390327-0f38-4f31-abdd-36b4b5c2aa45 | PubMed:22028789
  • Langford-Smith, A., Malinowska, M., Langford-Smith, K. J., Wegrzyn, G., Jones, S., Wynn, R., ... Bigger, B. W. (2011). Hyperactive behaviour in the mouse model of mucopolysaccharidosis iiib in the open field and home cage environments. Genes, Brain and Behavior, 10(6), 673-682. DOI:10.1111/j.1601-183X.2011.00706.x. Publication link: 25925fb9-da82-4ae7-a98f-643c255fd8b6 | PubMed:21635693
  • Langford-Smith, K. J., Mercer, J., Petty, J., Tylee, K., Church, H., Roberts, J., ... Bigger, B. W. (2011). Heparin cofactor II-thrombin complex and dermatan sulphate:chondroitin sulphate ratio are biomarkers of short- and long-term treatment effects in mucopolysaccharide diseases. Journal of Inherited Metabolic Disease, 34(2), 499-508. DOI:10.1007/s10545-010-9254-8. Publication link: 12161aa8-eb92-46ab-b60e-61a4742f490d | PubMed:21170681
  • Saif, M. A., Bonney, D. K., Bigger, B., Forsythe, L., Williams, N., Page, J., ... Wynn, R. F. (2011). Chronic norovirus infection in pediatric hematopoietic stem cell transplant recipients: A cause of prolonged intestinal failure requiring intensive nutritional support. Pediatric transplantation, 15(5), 505-509. DOI:10.1111/j.1399-3046.2011.01500.x. Publication link: 1e6b1b9f-e20b-4281-8df6-262485846d44 | PubMed:21504523


  • Archer, L. D., Langford, K., Bigger, B., & Fildes, J. E. (2010). 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". Journal of Neuroscience Research, 88(2), 233. DOI:10.1002/jnr.22211. Publication link: ae238047-c8ee-4137-abf4-5f6686d119ab | PubMed:19746428
  • Bonney, D. K., O'Meara, A., Shabani, A., Imrie, J., Bigger, B. W., Jones, S., ... Wynn, R. F. (2010). Successful allogeneic bone marrow transplant for Niemann-Pick disease type C2 is likely to be associated with a severe 'graft versus substrate' effect. Journal of Inherited Metabolic Disease, 33(3), S171-S173. DOI:10.1007/s10545-010-9060-3. Publication link: bda11104-d3b1-40d2-8242-05d1c07b9714 | PubMed:20393800
  • Canal, M. M., Wilkinson, F. L., Cooper, J. D., Ed Wraith, J., Wynn, R., & Bigger, B. W. (2010). Circadian rhythm and suprachiasmatic nucleus alterations in the mouse model of mucopolysaccharidosis IIIB. Behavioural Brain Research, 209(2), 212-220. DOI:10.1016/j.bbr.2010.01.045. Publication link: 0dcac905-5f13-4819-a67e-330816cdb431 | PubMed:20138090
  • Langford-Smith, K., Arasaradnam, M., Wraith, J. E., Wynn, R., & Bigger, B. W. (2010). Evaluation of heparin cofactor II-thrombin complex as a biomarker on blood spots from mucopolysaccharidosis I, IIIA and IIIB mice. Molecular Genetics and Metabolism, 99(3), 269-274. DOI:10.1016/j.ymgme.2009.10.175. Publication link: 9c76f0b2-034f-439b-9d3c-d284b5f8a7b7 | PubMed:19926322
  • Malinowska, M., Wilkinson, F. L., Langford-Smith, K. J., Langford-Smith, A., Brown, J. R., Crawford, B. E., ... Bigger, B. W. (2010). Genistein improves neuropathology and corrects behaviour in a mouse model of neurodegenerative metabolic disease. PLoS ONE, 5(12), [e14192]. DOI:10.1371/journal.pone.0014192. Publication link: 69b65744-9a91-4fea-ac68-0930103f73f7 | PubMed:21152017


  • Malinowska, M., Wilkinson, F. L., Bennett, W., Langford-Smith, K. J., O'Leary, H. A., Jakobkiewicz-Banecka, J., ... Bigger, B. W. (2009). Genistein reduces lysosomal storage in peripheral tissues of mucopolysaccharide IIIB mice. Molecular Genetics and Metabolism, 98(3), 235-242. DOI:10.1016/j.ymgme.2009.06.013. Publication link: 8c866e3e-2282-48e0-8f83-70d19f42f8fb | PubMed:19632871
  • Wynn, R. F., Stubbs, M., Ozyilmaz, N., Wraith, J. E., & Bigger, B. (2009). Cellular therapy of lysosomal storage disorders: Current status and future prospects. Current Pediatric Reviews, 5(3), 147-159. DOI:10.2174/157339609789007187. Publication link: f191f8a3-5f52-45af-baef-566a9333e314
  • Wynn, R. F., Wraith, J. E., Mercer, J., O'Meara, A., Tylee, K., Thornley, M., ... Bigger, B. W. (2009). Improved Metabolic Correction in Patients with Lysosomal Storage Disease Treated with Hematopoietic Stem Cell Transplant Compared with Enzyme Replacement Therapy. Journal of Pediatrics, 154(4), 609-611. DOI:10.1016/j.jpeds.2008.11.005. Publication link: b3b9d7b3-4a2c-4aa3-974c-9b7b0c0cb6a5 | PubMed:19324223


  • Katrangi, E., D'Souza, G., Boddapati, S. V., Kulawiec, M., Singh, K. K., Bigger, B., & Weissig, V. (2007). Xenogenic transfer of isolated murine mitochondria into human ρ0 cells can improve respiratory function. Rejuvenation Research, 10(4), 561-570. DOI:10.1089/rej.2007.0575. Publication link: 2f1c4887-c04d-4831-8a1c-dbb464cc0afb | PubMed:18069915
  • Siapati, E. K., Bigger, B. W., Kashofer, K., Themis, M., Thrasher, A. J., & Bonnet, D. (2007). Murine leukemia following irradiation conditioning for transplantation of lentivirally-modified hematopoietic stem cells. European Journal of Haematology, 78(4), 303-313. DOI:10.1111/j.1600-0609.2006.00813.x. Publication link: a5a83afa-10ed-4ac2-a911-a448c52f4b25 | PubMed:17378892


  • Bigger, B., Siapati, E. K., Mistry, A., Waddington, S. N., Nivsarkar, M. S., Jacobs, L., ... Themis, M. (2006). Permanent partial phenotypic correction and tolerance in a mouse model of hemophilia B by stem cell gene delivery of human factor IX. Gene Therapy, 13(2), 117-126. DOI:10.1038/sj.gt.3302638. Publication link: 74401453-8d19-400e-94b3-831dba63e83c | PubMed:16163377
  • Russo, F. P., Alison, M. R., Bigger, B. W., Amofah, E., Florou, A., Amin, F., ... Forbes, S. J. (2006). The Bone Marrow Functionally Contributes to Liver Fibrosis. Gastroenterology, 130(6), 1807-1821. DOI:10.1053/j.gastro.2006.01.036. Publication link: 984b82e2-50c2-4fe0-bf49-2a3e15c2a8dd | PubMed:16697743
  • Tolmachov, O., Palaszewski, I., Bigger, B., & Coutelle, C. (2006). RecET driven chromosomal gene targeting to generate a RecA deficient Escherichia coli strain for Cre mediated production of minicircle DNA. BMC Biotechnology, 6, [17]. DOI:10.1186/1472-6750-6-17. Publication link: 2de692fe-bb22-441f-8f32-d271fde98fb7


  • Siapati, E. K., Bigger, B. W., Miskin, J., Chipchase, D., Parsley, K. L., Mitrophanous, K., ... Bonnet, D. (2005). Comparison of HIV- and EIAV-based vectors on their efficiency in transducing murine and human hematopoietic repopulating cells. Molecular Therapy, 12(3), 537-546. DOI:10.1016/j.ymthe.2005.01.022. Publication link: cc4a6772-931d-4d95-8742-a91af86fd3d9
  • Themis, M., Waddington, S. N., Schmidt, M., von Kalle, C., Wang, Y., Al-Allaf, F., ... Coutelle, C. (2005). Oncogenesis following delivery of a nonprimate lentiviral gene therapy vector to fetal and neonatal mice. Molecular Therapy, 12(4), 763-771. DOI:10.1016/j.ymthe.2005.07.358. Publication link: 16270ed3-c860-41c2-b5e6-4fafecd49b2f
  • Bigger, B. W., Tolmachov, O., Harbottle, R., Coutelle, C., & Schleef, M. (Ed.) (2005). Minimised, CpG-depleted and methylated DNA vectors: Towards perfection in non-viral gene therapy. In DNA-Pharmaceuticals: Formulation and delivery in gene therapy, DNA vaccination and immunotherapy. Cambridge, UK: Wiley. . Publication link: 0458a37d-acc2-41e1-bce6-b2ce3e634429


  • Alison, M. R., Vig, P., Russo, F., Bigger, B. W., Amofah, E., Themis, M., & Forbes, S. (2004). Hepatic stem cells: From inside and outside the liver?. Cell Proliferation, 37(1), 1-21. DOI:10.1111/j.1365-2184.2004.00297.x. Publication link: 514b84fb-55b6-422a-9d68-8015214b5997 | PubMed:14871234
  • Bigger, B., Gregory, L. G., Waddington, S. N., Holder, M. V., Mitrophanous, K. A., Buckley, S. M. K., ... Themis, M. (2004). Highly efficient EIAV-mediated in utero gene transfer and expression in the major muscle groups affected by Duchenne muscular dystrophy. Gene Therapy, 11(14), 1117-1125. DOI:10.1038/sj.gt.3302268. Publication link: 7d84cc3a-a1f5-43d9-a7a8-6e0cfa6d0486 | PubMed:15141156
  • Vaysse, L., Harbottle, R., Bigger, B., Bergau, A., Tolmachov, O., & Coutelle, C. (2004). Development of a Self-assembling Nuclear Targeting Vector System Based on the Tetracycline Repressor Protein. Journal of Biological Chemistry, 279(7), 5555-5564. DOI:10.1074/jbc.M311894200. Publication link: 4d0973a8-348a-47e8-b8c6-ba381586eca9 | PubMed:14607832
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  • Bigger, B., Lightowlers, R. N., Taylor, R. W., Turnbull, D., & Holt, I. J. (Ed.) (2003). Gene Therapy for Mitochondrial DNA Disorders. In Genetics of Mitochondrial Diseases. Oxford: Oxford University Press. . Publication link: 3d8437a3-0a81-42c6-91d3-751cd15254f7



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