The Trust supports a number of inter-related collaborative projects based in London, Frankfurt, Zurich and the USA.
IMPROVING GENE THERAPY FOR CGD: VECTOR DEVELOPMENT, TESTING AND CLINICAL APPLICATION.
JEANS FOR GENES 10TH ANNIVERSARY AWARD
£680,000 over 3 years, CGD Research Trust grant (from 2004 appeal proceeds)
This is a joint collaborative award between the Gene Therapy Group at the Institute of Child Health and the Institute of Biomedical Research in Frankfurt.
Gene transfer into blood stem cells may provide a new way to cure many inherited diseases of the blood and immune system including CGD. Gene replacement therapy, in which a new working gene is introduced into stem cells, is particularly important for patients for whom conventional bone marrow transplantation is not possible. Recently, clinical trials for the treatment of CGD patients by gene therapy have been started with four patients treated to date. Although the results of the study are very promising, it is already known that there are improvements that can be made that will make it both more effective and safer. Therefore, the aim of this proposal is to test newly designed vectors that will take the new genes into the stem cells. Because the aim is to introduce these vectors into clinical studies (and hopefully develop gene therapy as a regular treatment), the vectors need to be evaluated very carefully. This programme of work incorporates the design and optimisation of gene therapy technology for CGD with the continuing refinement of clinical trials. By doing this more effective tools for the treatment of CGD by gene therapy will be generated. This project will contribute to the development of gene therapy procedures for many genetic disorders.
TESTING IMPROVED GENE THERAPY VECTORS FOR TREATMENT OF INFECTION AND CHRONIC INFLAMMATION IN CGD
Professor Mary Dinauer
Herman B Wells Centre for Paediatric Research at the Indiana University School of Medicine, USA. $20,000 donation from the Chronic Granulomatous Disorder Association, USA.
This project will test how well the new gene delivery tools for CGD, designed by our researchers at Great Ormond Street and in Frankfurt, will work in animal models. These second generation gene transfer tools are designed for improved safety and for more natural regulation of the X-CGD protein production in white blood cells. Following gene transfer into the blood stem cells of CGD mice, the group will determine the effectiveness of the new tools in improving resistance to fight infections and inflammation caused by the fungi and bacteria such as Aspergillus, Burkholderia cepacia, and Staphylococcus aureus that are threatening to people with CGD. The studies are an essential prerequisite for future clinical trials and will add real value to our gene therapy research programme.
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.
Three-Year Award: £106,000
This European collaborative grant was awarded to cover the costs of producing clinical grade vector to treat X-CGD. This improved vector is now being used to treat patients recruited 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. £39,000 over one year.
The aim of this project is to provide an alternative therapeutic treatment for CGD by delivery of therapeutic proteins. They will replace the protein that is missing in CGD, rather than the gene using delivery agents called proteoliposomes. It is hoped this work will work out how to deliver the gp91 phox protein for the treatment of X-linked CGD.
Previously 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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