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Correspondence
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Volume 361:725-727 August 13, 2009 Number 7
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Vision 1 Year after Gene Therapy for Leber's Congenital Amaurosis

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To the Editor: Leber's congenital amaurosis, a common cause of blindness in infants and children,1 recently became the first human genetic retinal disease to show improved vision in response to treatment. Patients with mutations in the gene encoding retinal pigment epithelium–specific 65-kD protein (RPE65) had gains in vision within weeks after subretinal injection of a vector containing the gene in one eye.2,3,4,5 At 1-year follow-up after gene therapy, the three young adult patients in our trial4,5 remained without serious adverse events.

A noteworthy observation in one patient at 1 year after treatment prompted further studies. For the first . . . [Full Text of this Article]



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