May 1, 2007

gene therapy trial for childhood blindness

Scientists react to announcement of the start of a new trial to use gene therapy to treat blindness in children.

Andrew George, Professor of Molecular Immunology, Imperial College London, said:

“A lot of painstaking work has been done to get everything ready for this trial, including research to understand the biology of the condition, choosing the vector and working on the surgery techniques. The eye is good for gene therapy because it is a simple organ and it is easy to see what is going on. There is hope that once gene therapy is developed in the eye, scientists could move on to more complex organs.”

Alastair Kent, Director of the Genetic Interest Group (The Genetic Interest Group (a national alliance of organisations that support children, families and individuals affected by genetic disorders), said:

“Our organisation represents more than a hundred charities supporting conditions caused by single gene disorders; so to hear of such quick progress in a gene therapy treatment is fantastic. We hope this success will lead to more funding of gene therapy research into rare conditions that currently have no cure or treatment.”

Leonard Seymour, Professor of Genetic Therapies and President of the British Society for Gene Therapy University of Oxford and Radcliffe Infirmary, said:

“The retina is a really good place for gene therapy because it can be accessed by direct injection – overcoming the problem of gene delivery. When gene delivery is efficient, the whole power of gene therapy is unleashed. The retina is also good because it is relatively immune-privileged, meaning that the vector (in this case a virus) should not be neutralised immediately upon administration. These early trials are really aiming to slow down deterioration of vision, and its only by performing clinical studies such as these that we can identify what we need to do to correct the problem altogether.”