ANALYSIS OF IMMUNE RECONSTITUTION AND FUNCTION IN CLINICAL TRIALS OF GENE
THERAPY FOR CGD
Professor Adrian Thrasher and Professor Christine Kinnon.
The Institute of Child Health, London
Two Year Award: £110,258
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 is to study how many copies of the corrected gene have been
introduced into patients and exactly where, at the gene level and in what
cell types, the corrected gene has been placed. This work will indicate
if there are preferential sites for the insertion of the new gene and will
provide valuable information on what types of cells in the body are important
for restoring the ability of CGD patients to fight infection. The project
will also fund 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. This important project will allow safer gene
therapy procedures to be developed and will help improve the design of
new vectors for the insertion of the corrective gene into cells.
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.
INCREASING THE CHANCES OF SUCCESS FOR CGD GENE THERAPY- DEVELOPING
STRATEGIES FOR THE SELECTIVE EXPANSION OF TRANSDUCED CGD CELLS
Dr Manuel Grez
Institute for Medical Research, Frankfurt, Germany
Three and a half Year Award: £182,961. Ended in January 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 ICH, set up methods to increase the number of cells carrying
the corrective gene gp91phox and increase their survival once transplanted
back into patients.
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. The progress made in this study is now
being 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. It is hoped to use this knowledge about the regulation of the
p47phox gene to design better and more effective gene therapy vectors with
the potential for treating CGD.
Currently funded projects - read more...
Publications from CGD RT funded research - 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.
© 2001-2005 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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