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• Centre for Gene Therapy of Childhood Disease
• Wolfson Centre for Gene Therapy of Childhood Disease

Wolfson Centre for Gene Therapy of Childhood Disease

This new Centre brings together a number of groups from across Great Ormond Street Hospital and UCL Institute of Child Health who are all working on different aspects of gene therapy in re-furbished laboratories on the ground floor of the main ICH building. 

Aims

The aims of our work are to develop the techniques for gene therapy of a wide range of disorders that affect children.

These include genetic disorders of the immune system, cancer, eye diseases and cystic fibrosis.

Successes

We have already been successful in developing gene therapy techniques for two severe combined  immunodeficiency disorders (SCID), X-linked SCID and adenosine deaminase SCID.

This has enabled us to go from the “bench” to the “bedside”. 

Recently

We have recently initiated clinical trials for these disorders, which have so far been successful in treating 14 patients. See summary of recent media coverage following publication of two papers in Science Translational Medicine on 24th August 2011.

Currently

We are now working on improving these treatments, using new improved, safer vectors and protocols, and on developing similar treatments for other immunodeficiency disorders.

Applications of this technology can be extended to a wide range of disorders and work is continuing on these fronts. 

Future

It is envisaged that the centre will enable further developments in this exciting new technology which will translate into cures for a wide range of diseases and many more patients will be treated in the future.