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Article

Long-Term Effects of Retinal Gene Therapy in Childhood Blindness

4 May 2015

Abstract excerpt

Current ocular imaging techniques provide extraordinarily high-resolution visualization of the retinal layers, amounting to what has been called in vivo microscopy. Combine this with a battery of sophisticated retinal-function tests, and the retina becomes unusually accessible in the evaluation of trials of retinal gene therapy for the treatment of conditions such as Leber’s congenital amaurosis.Leber’s...

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PMID: 25938534
DOI: 10.1056/nejme1503419

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