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Published at www.nejm.org April 27, 2008 (10.1056/NEJMe0803081)

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In this issue of the Journal, two groups of investigators — Bainbridge et al.¹ and Maguire et al.² — describe the first results of separate clinical trials investigating the short-term safety and preliminary efficacy of gene therapy for Leber's congenital amaurosis. Both groups present short-term data (12 months and 5 months, respectively) on three patients with Leber's congenital amaurosis in each study; the patients were enrolled in trials of recombinant adeno-associated viral delivery of the human retinal pigment epithelium-specific 65 kDa protein gene (RPE65), which was administered as a subretinal injection during vitrectomy.

Originally described by Leber in . . . [Full Text of this Article]

Source Information

From the Massachusetts Eye and Ear Infirmary, Harvard Medical School, Boston.

This article (10.1056/NEJMe0803081) was published at www.nejm.org on April 27, 2008. It will appear in the May 22 issue of the Journal.

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