One of the major problems encountered in the gene therapy trials for treatment of X-CGD is the ‘silencing’ or switching off of expression of the corrective healthy gene over time. Although the patient’s neutrophils may fight infection very efficiently right after receiving gene therapy, this ability declines over months and years.
With funding from the CGD Research Trust, the gene therapy team at the Institute of Child Health, headed by Professor Adrian Thrasher has come up with an innovative way round the problem, working in collaboration with Dr Michael Antoniou of Kings College, London.
They describe the addition of a DNA sequence to the usual gene delivery tool that is highly effective for maintaining the expression of the X-CGD gene in bone marrow cells, and more importantly in differentiated neutrophils, the target cell in CGD. Significantly this sequence stops the gene being switched off and when used in combination with another regulatory sequence it results in an increase in the number of gene corrected neutrophils and a significant revival of the oxidase activity that is able to kill bacteria and fungi.
The breakthrough was presented at the British Society for Gene Therapy’s annual meeting in March by Dr Giorgia Santilli, the research fellow on the project. She says: “This is a significant step forward in the design of future generations of gene therapy tools for treating CGD. We are hopeful that this will make gene therapy more effective for X-CGD.”
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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