Leber Congenital Amaurosis Due to GUCY2D Mutations: Longitudinal Analysis of Retinal Structure and Visual Function

Gene augmentation therapy is being planned for GUCY2D-associated Leber congenital amaurosis (LCA). To increase our understanding of the natural history of GUCY2D-LCA, patients were evaluated twice with an interval of 4 to 7 years between visits using safety and efficacy outcome measures previously d...

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Vydáno v:Int J Mol Sci
Hlavní autoři: Jacobson, Samuel G., Cideciyan, Artur V., Sumaroka, Alexander, Roman, Alejandro J., Wu, Vivian, Swider, Malgorzata, Sheplock, Rebecca, Krishnan, Arun K., Garafalo, Alexandra V.
Médium: Artigo
Jazyk:Inglês
Vydáno: MDPI 2021
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On-line přístup:https://ncbi.nlm.nih.gov/pmc/articles/PMC7922686/
https://ncbi.nlm.nih.gov/pubmed/33670772
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.3390/ijms22042031
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