COMPARATIVE GENE THERAPY TRIALS FOR X-CGD AT THREE EUROPEAN CENTRES Professor Adrian Thrasher, Dr Manuel Grez and Professor Hossle. Institute for Child Health; Georg-Speyer-Haus, Frankfurt, Germany and the University Children's Hospital, Zurich, Switzerland. Two year award: £68,194.
This European collaborative grant was awarded to cover the costs of producing clinical grade vector to treat X-CGD. This improved vector was used to treat seven patients into the clinical trials for CGD in London, Frankfurt and Zurich.
PROTEIN TRANSFER AS A NEW THERAPEUTIC APPROACH IN X-LINKED CGD Professor Morel, Lab Enzymologie, Grenoble, France. £30,083 over one year. Ended in 2007.
An alternative therapeutic treatment for CGD is by the delivery of therapeutic proteins. In this new approach the protein that is missing in CGD will be put back into cells rather than the gene. This study established proof of principle showing that using special delivery agents called proteoliposomes Gp91phox, the protein affected in X-linked CGD, can be transferred into cells. Once taken up by the cells the articificial Gp91phox showed similar activities to those associated with its natural counterpart. The work demonstrated good 'proof of concept' and that protein therapy could be another option for the treatment of CGD in the future. This therapy would help 'top up' their immune systems over a short period of time to fight off infections.
ANALYSIS OF IMMUNE RECONSTITUTION AND FUNCTION IN CLINICAL TRIALS OF GENE THERAPY FOR CGD Professor Adrian Thrasher, Professor Bobby Gaspar and Professor Christine Kinnon
The Institute of Child Health, London
Two Year Award: £110,258, ended in 2005.
Gene therapy clinical trials for patients with X-linked CGD and other primary immuno-deficiency diseases are taking place at the Institute of Child Health. This project developed methods to measure how many copies of corrected genes are introduced into patients' cells, exactly where, at the gene level the corrective gene integrated and in what cell types. The project also funded facilities for the storage and cataloguing of patients' samples so that measurement and monitoring of corrected gene levels can be done over long periods of time for people undergoing gene therapy for a number of genetic disorders. This important project helped safer gene therapy procedures to be developed and helped improve the design of new vectors for the treatment of CGD,
THERAPEUTIC STRATEGIES FOR THE TREATMENT OF CGD Professor Adrian Thrasher and Professor Christine Kinnon
The Institute of Child Health, London
Three-Year Award: £190,291 Ended in 2004.
This was the first CGD strategic programme grant awarded and was given to secure core funding to develop and implement gene therapy strategies for the treatment of CGD. This project resulted in the design of vectors for use in clinical trials for gene therapy for CGD.
INCREASING THE CHANCES OF SUCCESS FOR CGD GENE THERAPY Dr Manuel Grez Institute for Medical Research, Frankfurt, Germany
Three and a half Year Award: £182,961. Ended in 2005.
In CGD the gene-corrected cells do not have any selective advantage over the non-corrected cells present in the bone marrow. This collaborative project with Institute of Child Health in London, set up methods to increase the number of cells carrying the corrective gene gp91phox and increase their survival once transplanted back into patients. The group has close ties with the research teams in the UK, who felt that success in this project represented an exciting step forward for this condition and many others.
TARGETING THERAPEUTIC GENE TRANSFER TO SPECIFIC CELL TYPES
Professors J.P. Hossle and Professor R. Seger.
University Children's Hospital, Zurich
Two Year Award: £84,401 Ended in 2003.
This important study helped refine gene therapy protocols for X-linked CGD so that the corrective gene, gp91phox, would only be expressed in specific types of blood cells. Two important gene sequences that are capable of regulating the expression of this corrective therapeutic gene were identified. The progress made in this study was used to develop better vectors through the work at ICH and in Frankfurt.
LEARNING MORE ABOUT THE p47 PHOX GENE
Dr Colin Casimir.
Imperial College at the Hammersmith Hospital, London
Three-Year Award: £121,615 Ended in 2000.
The p47phox protein is missing in about one quarter to one third of all CGD patients and is the most common cause of autosomal recessive CGD (the type that affects boys and girls equally). This project investigated the gene that codes for p47phox, in particular, how the gene is regulated, so that it is highly active in phagocytes but not other types of cells. The work identified two regions of DNA that are important for p47phox gene expression. This knowledge about the regulation of the p47phox gene was used to design better and more effective gene therapy vectors with the potential for treating CGD.
Currently funded projects - read more...
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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