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Gene Therapy Improves Vision In First-Of-A-Kind Clinical Study
| Author: Tony Cappasso |
| Article Date: 5/10/2008 |
Physicians in Pennsylvania have for the first time used gene therapy to restore some vision in children with a congenital vision problem called Leber congenital amaurosis or LCA. The team reports their findings in an online version of the New England Journal of Medicine.
The researchers, from the U.S. and Italy performed surgery at the Children’s Hospital of Philadelphia to insert a gene that was carried on an adenovirus vector. The virus carried a normal version of the gene called RPE65 that is mutated in some forms of LCA, a group of vision-robbing diseases that usually strike in childhood. The three patients, aged 19, 26 and 26, were treated in the Children’s Hospital.
All three patients could detect motion directly in front of their eyes before the therapy. After treatment, all three were able to read some lines from a vision chart.
The patients enrolled in the study to date were identified at the Department of Ophthalmology at the Second University of Naples, an institution with long-standing experience in collecting and studying patients with inherited retinal diseases, under the supervision of Francesca Simonelli, M.D.
Testing continued over a period of six months following the gene therapy vector administration. One patient was better able to navigate an obstacle course compared to before the injection. The patients also had less nystagmus, an involuntary movement of the eyes that is common in LCA. In the patient who experienced better vision even in the uninjected eye, the researchers suggest that the reduced nystagmus benefited both eyes.
The researchers, from the U.S. and Italy performed surgery at the Children’s Hospital of Philadelphia to insert a gene that was carried on an adenovirus vector. The virus carried a normal version of the gene called RPE65 that is mutated in some forms of LCA, a group of vision-robbing diseases that usually strike in childhood. The three patients, aged 19, 26 and 26, were treated in the Children’s Hospital.
All three patients could detect motion directly in front of their eyes before the therapy. After treatment, all three were able to read some lines from a vision chart.
The patients enrolled in the study to date were identified at the Department of Ophthalmology at the Second University of Naples, an institution with long-standing experience in collecting and studying patients with inherited retinal diseases, under the supervision of Francesca Simonelli, M.D.
Testing continued over a period of six months following the gene therapy vector administration. One patient was better able to navigate an obstacle course compared to before the injection. The patients also had less nystagmus, an involuntary movement of the eyes that is common in LCA. In the patient who experienced better vision even in the uninjected eye, the researchers suggest that the reduced nystagmus benefited both eyes.