Treatment Potential for LCA5-Associated Leber Congenital Amaurosis

PURPOSE: To determine the therapeutic window for gene augmentation for Leber congenital amaurosis (LCA) associated with mutations in LCA5. METHODS: Five patients (ages 6–31) with LCA and biallelic LCA5 mutations underwent an ophthalmic examination including optical coherence tomography (SD-OCT), ful...

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Vydáno v:Invest Ophthalmol Vis Sci
Hlavní autoři: Uyhazi, Katherine E., Aravand, Puya, Bell, Brent A., Wei, Zhangyong, Leo, Lanfranco, Serrano, Leona W., Pearson, Denise J., Shpylchak, Ivan, Pham, Jennifer, Vasireddy, Vidyullatha, Bennett, Jean, Aleman, Tomas S.
Médium: Artigo
Jazyk:Inglês
Vydáno: The Association for Research in Vision and Ophthalmology 2020
Témata:
Retina
On-line přístup:https://ncbi.nlm.nih.gov/pmc/articles/PMC7405811/
https://ncbi.nlm.nih.gov/pubmed/32428231
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1167/iovs.61.5.30
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