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.
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.
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”.
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.
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.
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.
Page last modified on 01 sep 11 11:04