REVIEWS ON CGD
Chronic Granulomatous Disease; fundamental stages in our understanding of CGD. Med Immunol. 5: 4; 2006.
Tracy Assari. This article is available from http://www.medimmunol.com/content/5/1/4
GENE THERAPY
Correction of X-linked Chronic Granulomatous Disease by gene therapy, augmented by insertional activation of MDS/EVI1, PRDM16 or SETBP1. Nature Medicine 12:401-409, 2006.
MG Ott, M Schmidt, K Schwarzwaelder, S Stein, U Siler, U Koehl, H Glimm, K Kühlcke, A Schilz, H Kunkel, S Naundorf, A Brinkmann, A Deichmann, M Fischer, C Ball, I Pilz, C Dunbar, Y Du, NA Jenkins, NG Copeland, U Lüthi, M Hassan, AJ Thrasher, D Hoelzer, C von Kalle, R Seger, M Grez.
Successful reconstitution of immune function in ADA-SCID by stem cell gene therapy following cessation of PEG-ADA and use of mild preconditioning. Mol Ther. 14(4):505-13, 2006.
HB Gaspar, E Bjorkegren, K Parsley, K Gilmour, D King, J Sinclair, S Adams, LD Fairbanks, J Gaspar, L Henderson, EG Davies, KC Veys, AJ Thrasher.
Risks of gene therapy should be weighed against lack of alternatives for many diseases
BMJ;330:79, Letter, 2005.
RE MacLaren, RR Ali, AJ Thrasher.
Available at http://www.bmj.com/cgi/content/full/330/7494/791-a
Failure of SCID-X1 gene therapy in older patients. Blood, 105:4255-4257, 2005.
AJ Thrasher, S Hacein-Bey-Abina, HB Gaspar, S Blanche, EG Davies, K Parsley, K Gilmour, D King, S Howe, J Sinclair, C Hue, F Carlier, C von Kalle, BG de Saint, F Le Deist, A Fischer, M Cavazzana-Calvo.
Gene therapy for lympho-hematopoietic disorders. Current Hematology Reports 4:305-309, 2005.
AJ Thrasher, and HB Gaspar.
Gene therapy in primary immunodeficiency. Expert Opin Biol Ther. 5:1175-82, 2005.
AJ Thrasher and HB Gaspar.
Successful gene therapy of SCID-X1 using a pseudotyped gammaretroviral vector. Lancet, 364(9452):2181-7, 2004.
Gaspar HB, Parsley K, Howe S, King D, Gilmour KC, Sinclair J, White H, Smith J, Barington T, Jacobsen M, Christensen HO, Brouns G, AlGhonaim A, Schmidt M, Von Kalle c, Ali, RR, Kinnon C, Thrasher AJ.
Enhancer-deleted retroviral vectors restore high levels of superoxide generation in a mouse model of CGD. J Gene Med, 6:6, 603-615, 2004.
Schwickerath O, Brouns, G,Thrasher, A, Kinnon C, Roes, J, Casimir, C.
Enhancer-Deleted Retroviral Vectors Can Restore High Levels of Superoxide Generation in a Mouse Model of CGD. J Gene Med. 6(6):603-15, 2004.
Schwickerath O, Brouns G, Thrasher, AJ, Kinnon C, Roes J, Casimir CM.
Differentiation-Dependent Up-Regulation of p47phox Gene Transcription is Associated with Changes in PU.1 Phosphorylation and Increased Binding Affinity. Biochem. Biophys. Res.Commun. 305, 193-202, 2003.
Marden CA, Cunninghame Graham, DS, Casimir CM.
A Functional ISRE is Required for Myeloid Transcription of the p47phox gene. Biochim. et Biophys. Acta 1630 117-122. 2003.
Marden, CM, Cunninghame Graham D, Thrasher AJ, Casimir, C.M.
High-level transduction and gene expression in haematopoietic repopulating cells using a human immunodeficiency virus type-1 based lentiviral vector containing an internal spleen focus forming virus promoter. Human Gene Therapy 13:803-813, 2003.
Demaison C, Parsley K, Brouns G, Scherr M, Battmer K, Kinnon C, Grez M,Thrasher AJ.
Mobilisation and transduction of CD34+ peripheral blood stem cells in patients with X-linked chronic granulomatous disease. J Hematotherapy & Stem Cell Research: 11: 683-694, 2002.
Ott MG, H Merget-Millitzer, OG Ottmann, H Martin, N Brüggenolte, H Bialek, R Seger, JP Hossle, D.Hoelzer, M Grez.
Gene Therapy of Hematopoietic Stem Cells: Strategies for Improvement. News Physiol Sci 17: 87-92, 2002.
Hossle JP, Seger RA, Steinhoff D.
A defined window for efficient gene marking of severe combined immunodeficient repopulating cells using a gibbon ape leukaemia virus-pseudotyped retroviral vector. Human Gene Therapy 11:91-100, 2000.
Demaison C, Brouns G, Blundell MP, Goldman JP, Levinsky RJ, Grez M, Kinnon C, Thrasher AJ.
Quality of repopulation in NOD/SCID mice engrafted with expanded cord blood CD34+ cells. Blood 94: 3269-3270, 1999.
Blundell M, Lazzari L, Demaison C, Brouns G, Goldman J, Gaspar HB, Kinnon C, Sirchia G, and Thrasher AJ.
Functional Analysis of the p47phox gene promoter. PhD Thesis, University of London (1999).
Marden CM.
ORAL PRESENTATIONS AT INTERNATIONAL CONFERENCES
- American Society of Gene Therapy, 9th Annual Meeting, Baltimore, June 2006
M. Grez. ‘Clinical results from a Phase I gene therapy trial for CGD’.
AJ. Thrasher. ‘Clinical Therapy for Immunodeficiency’. - International Society for Cellular Therapy, Berlin, May 2006
M Grez. ‘Gene Therapy of CGD’. - Public Education Day, British Society for Gene Therapy, 3rd Annual Conference, London, March 2006
AJ Thrasher. ‘Gene therapy for immune deficiencies’. - British Society for Gene Therapy, London, March 2006
M Grez. ‘Correction of X-linked CGD by Gene Therapy’. - European Society for Clinical Investigation, Prague, February 2006
M Grez. ‘Improving gene therapy for CGD: vector development, testing and clinical application’. - EU CONSERT Meeting, Leukerbad, January 2006
M Grez. ‘Retroviral gene therapy for CGD’. - EUROCONFERENCE Cellular and Gene Therapy, Paris, December 2005
M Grez. ‘Gene Therapy of CGD’. - European Society for Gene Therapy, XIII Annual Conference, Prague, November 2005
M Grez. ‘Gene Therapy of CGD’.
AJ Thrasher. ‘Gene Therapy of Immunodeficiencies’ and ‘Safety and Regulatory Issues’ - Cell-and Gene-Therapy Symposium, Budapest, September 2005
M Grez. ‘Gene Therapy of CGD’. - Japanese Society of Gene Therapy, Tokyo, July 2005
M. Grez. ‘Reconstitution of phagocytic killing in X-CGD patients after gene therapy’. - German Society of Gene Therapy, Würzburg, July 2005
M Grez. ‘Update on the X-CGD gene therapy phase I clinical study’. - American Society of Gene Therapy, 8th Annual Meeting, St. Louis, USA, June 2005
M Grez. ‘Correction of CGD by gene therapy’.
AJ Thrasher. ‘Clinical trials for immunodeficiency’. - State of the Art Conference on Gene Therapy, Royal Society of Medicine, London, April 2005
AJ Thrasher. ‘Gene therapy for immunodeficiency’. - Life Science Congress, Austrian Societies for Biochemistry, Gene Technology, Bio Technology and Gene Therapy, Vienna, Austria, September 2005
AJ Thrasher. ‘Successful Gene Therapy for Inherited Immunodeficiency’. - XIth Meeting of the European Society for Immunodeficiencies, Versailles, France, 2004
M. Grez. ‘Gene Therapy for X-linked Chronic Granulomatous Disease’.
A. Thrasher. ‘Update on Gene Therapy Trials’ - The 12th Annual Congress of the European Society of Gene Therapy, Tampere, Finland, 2004
M. Grez ‘Gene Therapy for X-linked Chronic Granulomatous Disease’. - American Society of Hematology Annual Meeting, San Diego, CA USA, 2004
M. Grez. ‘Gene Therapy for X-linked Chronic Granulomatous Disease’. - Danish Society for Clinical Immunology, Copenhagen, June 2004
HB Gaspar. ‘Gene Therapy for Primary Immunodeficiencies’ - National Institute of Health, Bethesda, February 2004
A. Thrasher. ‘Clinical Gene Therapy for SCID-XI’ - American Society of Blood and Marrow Transplantation tandem meeting, Orlando, February 2004
A. Thrasher. ‘Gene Therapy for X-SCID – the UK Experience’ - British Society for Gene Therapy, Keble College, Oxford, March 2004
A. Thrasher. ‘SCID Clinical Trials and Beyond – lessons to be learned’ - University of Glasgow, May 2004
A. Thrasher. ‘Successful Gene Therapy for SCID’ - Infection and Immunity in Children, Keble College Oxford, June 2004
A. Thrasher. ‘New Insights into Primary Immunodeficiency’ - British Society of Immunology, Harrogate, December 2004
A. Thrasher. ‘Gene Therapy for CGD’ - 5th Annual Meeting of the American Society of Gene Therapy, Boston, June 2002
M. Grez. 'Selective expansion of retrovirally transduced haematopoietic cells', - 6th Annual Meeting of the European Haematology Association, Frankfurt, June 2001
M. Grez. ‘Ex vivo expansion of haematopoietic stem cells' - 9th Meeting of the European Society of Gene Therapy, Antalya, November 2001
M. Grez. 'An ex vivo expansion system for retrovirally transduced haematopoietic
stem cells'
INFLAMMATION IN CGD
NADPH oxidase is not required for spontaneous and Staphylococcus aureus-induced apoptosis of monocytes. Annals of Hematology 83(4):206-11, 2004.
Von Bernuth H, Kulka C, Roesler J, Gahr M, Rosen-Wolff A.
Diminished production of anti-inflammatory mediators during neutrophil apoptosis and macrophage phagocytosis in CGD. Journal of Leukocyte Biology: 73, 591-599, 2003. Brown JR, Goldblatt D, Buddle J, Morton L, Thrasher AJ.
Association between p47phox pseudogenes and inflammatory bowel disease. Blood 101(8), 3337, 2003.
M. Harbord, A. Hankin, S. Bloom, H. Mitchison.
Ym1 is a neutrophil granule protein that crystallizes in p47phox-deficient mice. J Biol Chem. 277(7), 5468-75, 2002.
Harbord M, Novelli M, Canas B, Power D, Davis C, Godovac-Zimmermann J, Roes J, Segal AW.
Cantharidin blisters: a technique for investigating leukocyte trafficking and cytokine production at sites of inflammation in humans. J Immunol Methods. 257(1-2), 213-20, 2001.
Day RM, Harbord M, Forbes A, Segal AW.
FIGHTING FUNGAL INFECTIONS
Virulence comparisons of Aspergillus nidulans mutants are confounded by the inflammatory response of p47phox-/- mice. Infection and Immunity 73(8): 5204-7, 2005.
Bignell E, Negrete-Urtasun S, Calcagno AM, Arst HN, Rogers T, Haynes K.
The Aspergillus pH-responsive transcription factor PacC regulates virulence. Mol Microbiol. 55(4):1072-84, 2005.
Bignell E, Negrete-Urtasun S, Calcagno AM, Haynes K, Arst HN Jr, Rogers T.
ORAL PRESENTATIONS
- Blood and Fungi Meeting, November 2005.
E Bignell. Virulence comparisons of Aspergillus nidulans mutants are confounded by the inflammatory response of p47phox-/- mice.
POSTER PRESENTATIONS
- 2nd Advances against Aspergillosis, Athens, Greece, February 2006.
Suzy Turnbull. Transcript profiling of the murine immune response to Aspergillus Fumigatus.
UNDERSTANDING THE ENZYME AFFECTED IN CGD
Single-cell optical imaging of the phagocyte NADPH oxidase. Antioxidants and Redox Signaling, Antioxid Redox Signal. 1509-22, 2006.
H-J van Manen, R van Bruggen, D Roos, C Otto.
Raman microscopy of phagocytosis: shedding light on macrophage foam cell formation. Proc. SPIE Vol. 6093, 60930I-1, 2006.
H-J van Manen, AA van Apeldoorn, D Roos, C Otto.
Single-cell Raman and fluorescence microscopy reveal the association of lipid bodies with phagosomes in leukocytes. Proc Natl Acad Sci USA 102(29):10159-64, 2005.
Van Manen HJ, Kraan YM, Roos D, Otto C.
Continuous translocation of Rac2 and the NADPH oxidase component p67(phox) during phagocytosis. J Biol Chem. 279(10):9097-102, 2004.
Van Bruggen R, Anthony E, Fernandez-Borja M, Roos D.
Intracellular chemical imaging of heme-containing enzymes involved in innate immunity using resonance Raman microscopy. J.Phys. Chem B 108, 18762-18771, 2004.
van Manen HJ, Kraan YM, Roos D, Otto C.
Resonance Raman imaging of the NADPH oxidase subunit cytochrome b558 in single neutrophilic granulocytes. J.Am. Chem. Soc. 125: 12112-12113, 2003.
Van Manen H-J, Uzunbajakava Nvan Bruggen R, Roos D, Otto C.
Non-reasonant confocal Raman imaging of DNA and protein distribution in apoptotic cells. Biophy. J. 84: 3968-3981, 2003.
Uzunbajakava N, Lenferink A, Kraan Y, Volokhina E, Vrensen G, Greve J, Otto C.
ORAL PRESENTATIONS
- Focus on Microscopy 2004. Philadelphia. USA, 2004
D Roos and C Otto. Confocal Raman imaging of cytochrome b558 in single cells. - European Conference of the Spectroscopy of Biomolecules (ECSBM) Aschaffenburg Germany, September 2005.
C Otto. Single cell Raman and fluorescence microscopy of leukocytes. - Spec2006: Shedding light on Disease; Optical Diagnosis for the New Millenium, Heidelberg, Germany, May 2006.
C Otto. Lipid Droplets: a new player in the molecular mechanism of the immune response in neutrophilic granulocytes. - Photonics West – Biomedical Optics, January 2006, San Jose, CA, USA.
H-J van Manen, D Roos, V Subramaniam, C Otto. Raman microscopy of phagocytosis: shedding light on innate immunity. - Annual Dutch Meeting on Cellular and Molecular Biophysics, October 2006, Lunteren, The Netherlands.
H-J van Manen, D Roos, V Subramaniam, C Otto. Raman imaging reveals a role for lipid droplets in the innate immune response.
CGD REGISTRIES
ORAL PRESENTATIONS
- ESID Conference, Versailles, France, October 2004.
D Roos and T Kuijpers. European CGD Registry. - CGD RT Consultant's Conference, London, November 2002.
L Jones. CGD in the UK and Ireland. - British Thoracic Society Meeting, London, December 2002
L Jones. Respiratory Disease in CGD. - Royal College of Paediatrics and Child Health, York, April 2003
L Jones. CGD in the UK and Ireland: Epidemiological and Clinical Features.
POSTER PRESENTATIONS
- European Society of Immunodeficiencies, Weimar, October 2002.
CGD in the UK and Ireland: A report from the Registry. L Jones. - European Society of Paediatric Gastroenterology, Hepatology and Nutrition, Prague, June 2003.
Liver Abscesses in CGD. PJ McGrogan.
IMPORTANT NOTE :
The information contained on this website is intended only as a guideline, not as a substitute for medical advice. Always consult your doctor if you or your child has any CGD symptoms or concerns.
© 2001-2007 The Chronic Granulomatous Disorder (CGD) Research Trust
Registered Charity No. 1003425 email:cgd@cgdrt.co.uk
The CGD Research Trust is a member of the Association of Medical Research Charities (AMRC), the Genetic Interest Group (GiG) and an associate member of the International Patient Organisation of Primary Immunodeficiencies (IPOPI)
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