Restoring Sight By Gene Therapy

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Gregory M. Acland (Nature Genetics, May issue) report on the first successful use of gene therapy in restoring the sight of dogs blinded by an inherited retinal degenerative disease thus offering hope for humans with a similar condition. The defect was in RPE65, which is a gene associated with the retinal pigment epithelium (RPE). The gene therapy experiments employed functional RPE65 genes and were delivered using a viral vector injected into the subretinal space of dogs that had the defective RPE65 gene. Within six weeks, the treated eyes were producing the correct form of RPE65 protein, and by three months vision was restored to the treated eyes.


Trends In Biotechnology, July 2001, Vol. 19, No.7