The eyes have it.

摘要

In this month's Molecular Therapy, Simonelli et al. describe the 18-month follow-up data for the first three patients with Leber's congenital blindness treated with adeno-associated viral (AAV) vector gene transfer of the RPE65 gene. Together with the authors' description of their complete study in the Lancet and the initial report of the first three patients in the New England Journal of Medicine, these data represent one of gene therapy's most impressive clinical accomplishments to date. Few who have seen the Web videos of a blind child being restored to sight can fail to be impressed by the power of gene therapy or moved by its benefits (http://www.nature.com/ mt/journal/vaopcurrent/extref/mt2009277x3. avi). Although it may be a challenge to extend these accomplishments to other genetic causes of blindness, the fundamental properties of the eye in terms of its accessibility to local gene delivery and the extraordinary benefits to quality of life of even a limited restoration of sight will ensure that extensive efforts will be made to use gene therapy in a much broader range of retinal diseases.