A recent study in the US has resulted in the development of viral vector which is designed to deliver a gene into the eyes of people born with an inherited, progressive form of blindness.
Researchers at Ohio State University Medical Center and Nationwide Children"s Hospital have developed the vector, which is part of a clinical trial investigating the use of gene therapy to cure choroideremia at the University of Oxford in England.
The disease affects an estimated 100,000 people worldwide and is hoped the new therapy will help to provide relief for patients, many of whom are male.
The vector was designed by Dr Matthew During, professor of molecular virology, immunology and medical genetics and of neuroscience and neurological surgery at Ohio State University, in collaboration with Robert MacLaren, professor of ophthalmology at the University of Oxford.
Dr During explained: "I and my colleagues are excited about contributing to this significant medical breakthrough. We have worked for many years to engineer and optimize viruses to safely deliver genes to humans, and the eye is an ideal target in many ways."
He described the clinical vector manufacturing facility at Nationwide Children"s Hospital as "outstanding" and said the clinical vector will for the first time offers patients the possibility of an effective therapy.
The expert said the trial represents the first attempt to treat the disease and also the first time that gene therapy has been directed towards the photoreceptor cells of the human retina.
Experts now believe it holds great promise for the treatment of other genetic causes of blindness such as retinitis pigmentosa.
Mr MacLaren summed up the feelings of the researchers: "This disease has been recognized as an incurable form of blindness for over a hundred years. I cannot describe the excitement in thinking that we have designed a genetic treatment that could potentially stop it in its tracks with one single injection."
by Adrian Galbreth