本帖最后由 chinatiger 于 2012-2-12 22:46 编辑
这个消息是2011年十月的，以前有人发过吗？
基因治疗失明患者，包括无脉络膜症和RP，在英国牛津大学和Moorfields 眼科医院进行，后者也是ACT胚胎干细胞治疗stargardt's病和干性黄斑变性的欧洲合作伙伴
breakthrough in gene therapy for blindness
A clinical trial to treat genetic blindness caused by a disease known as choroideraemia（无脉络膜症） has started in Oxford as part of a nationwide NHS study, which includes the National Institute for Health Research (NIHR) Biomedical Research Centre (BRC) for Ophthalmology, based at Moorfields Eye Hospital and the UCL Institute of Ophthalmology.
Patients in the trial will have a virus injected into their eye that has been genetically engineered to carry a missing gene. The virus has been modified to enable it to infect the light sensitive cells known as photoreceptors that line the back of the eye. This is the first time these cells have been targeted using gene therapy and it paves the way for the treatment of other genetic causes of blindness such as retinitis pigmentosa.
The trial is being led by Professor Robert MacLaren, professor of ophthalmology at the University of Oxford and at Moorfields Eye Hospital by Mr Andrew Webster, a consultant and senior lecturer at the UCL Institute of Ophthalmology. The clinical trial builds on previous gene therapy research innovations developed through UCL and Moorfields and performed in collaboration with Professor Miguel Seabra at Imperial College London.
“This disease has been recognised around the world as an incurable form of blindness for over a hundred years,” says Professor MacLaren. “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.”
The 12 patients enrolled to the trial from across all four centres will undergo gene surgery treatment to one eye, and researchers expect it to take a further 24 months to know whether or not the degeneration has been stopped completely by the gene therapy treatment.
The clinical trial is funded by the Department of Health and the Wellcome Trust, through the Health Innovation Challenge Fund. Early development was also supported by Moorfields Eye Charity and the Lanvern Foundation. The trial is sponsored by the University of Oxford and includes four NHS centres: Oxford Radcliffe Hospitals NHS Trust, Moorfields Eye Hospital NHS Foundation Trust, Manchester Royal Eye Hospital and Southampton University Hospitals NHS Trust. |
