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See Latest ArticlesAge-dependent effects of RPE65 gene therapy for Leber's congenital amaurosis: a phase 1 dose-escalation trial
The Lancet, 11/09/09
Maguire AM et al. – The safety, extent, and stability of improvement in vision in all patients support the use of AAV–mediated gene therapy for treatment of inherited retinal diseases, with early intervention resulting in the best potential gain.
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