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Gene Therapy for Choroideremia Using an Adeno-Associated Viral (AAV) Vector

Choroideremia is an outer retinal degeneration with a characteristic clinical appearance that was first described in the nineteenth century. The disorder begins with reduction of night vision and gradually progresses to blindness by middle age. The appearance of the fundus in sufferers is recognizab...

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Pubblicato in:Cold Spring Harb Perspect Med
Autori principali: Barnard, Alun R., Groppe, Markus, MacLaren, Robert E.
Natura: Artigo
Lingua:Inglês
Pubblicazione: Cold Spring Harbor Laboratory Press 2015
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Accesso online:https://ncbi.nlm.nih.gov/pmc/articles/PMC4355255/
https://ncbi.nlm.nih.gov/pubmed/25359548
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1101/cshperspect.a017293
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