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Previous Volume 358:2282-2284 May 22, 2008 Number 21 Next

	Since this article has no abstract, we have provided an extract of the first 100 words of the full text and any section headings.

	Full Text PDF PDA Full Text Purchase this article Add to Personal Archive Add to Citation Manager Notify a Friend E-mail When Cited E-mail When Letters Appear Related Article by Bainbridge, J. W.B. Related Article by Maguire, A. M. PubMed Citation

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 1869, . . . [Full Text of this Article]

Source Information

This article (10.1056/NEJMe0803081) was published at www.nejm.org on April 27, 2008.

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

This article has been cited by other articles:

• Jacobson, S. G., Cideciyan, A. V., Aleman, T. S., Sumaroka, A., Windsor, E. A. M., Schwartz, S. B., Heon, E., Stone, E. M. (2008). Photoreceptor Layer Topography in Children with Leber Congenital Amaurosis Caused by RPE65 Mutations. IOVS 49: 4573-4577 [Abstract] [Full Text]  
• (2008). Gene Therapy for Congenital Retinal Degeneration. JWatch Neurology 2008: 1-1 [Full Text]