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.
© 2001-2005 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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