Why we fund gene therapy
For people with CGD stem cell transplantation remains the best option for a cure. More is learnt each year and its success rates continue to rise a little each year. Unfortunately it is not an option for everyone and one of the key objectives of our funding is to develop alternative treatments such as gene therapy.
Gene therapy is a potential new treatment for inherited diseases such as CGD. We rely on our genes functioning correctly, so when a gene is missing or not functioning properly, a genetic disease will result. Gene therapy is a way of putting the correct genetic information back into a cell so that it follows a normal healthy set of instructions. This will mean that CGD affected individuals will be able to fight against infection.
'This is the start of a new branch of medicine for the treatment of inherited disorders such as CGD and the Trust's support has been invaluable in driving this field forward.' Professor Len Seymour, Professor of Gene Therapies at Oxford University and ex-President of the British Society for Gene Therapy.
What is Involved in Gene Therapy?
The key to gene therapy is delivering the right gene to the cells that need it. For CGD the cells that need correcting are called stem cells. These cells are present in bone marrow and blood and are able to generate all the different cell types that are present in blood.
For gene therapy, some bone marrow or blood is taken from the patient and mixed together with the normal healthy gene spliced to a harmless virus (termed vector). The virus carrying the corrective gene then penetrates the patient's stem cells replacing the defective gene with the healthy gene. These corrected cells are then grown up in an incubator. Once sufficient numbers have been obtained the corrected cells are then re-injected into the patient's blood stream. Gradually they are absorbed back into the patient's blood marrow where they carry on their work of manufacturing blood cells - cells that are now capable of fighting infections.
> Find out more about gene therapy for CGD here (PDF)
Key Areas of Research for Gene Therapy
The Trust funds key areas of research that will improve gene therapy procedures for the cure of CGD. These include: -
- Increasing the efficiency of gene therapy so that an optimum amount of the normal healthy gene is delivered to the right cells in a manner that is safe and effective over the long-term. This is dependent on the design of the gene therapy tool or 'vector'.
- Improving the safety of gene therapy vectors.
Major achievements have been made in gene therapy for CGD with help from funding from the CGD Research Trust.
- Establishment of the Gene Therapy Laboratory at the Institute of Child Health
- Development of new techniques for monitoring gene therapy patients
- Funding of clinical reagents to treat CGD patients
- Start of Clinical Trials for CGD with seven patients treated to 2009 demonstrating evidence of therapeutic value.
- Refinements of clinical vectors to improve effectiveness and safety
Read more about Achievements in Gene Therapy for 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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