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Gene Therapy Fully Restores Vision to the All-Cone Nrl(−/−)Gucy2e(−/−) Mouse Model of Leber Congenital Amaurosis-1
Mutations in GUCY2D are the cause of Leber congenital amaurosis type 1 (LCA1). GUCY2D encodes retinal guanylate cyclase-1 (retGC1), a protein expressed exclusively in outer segments of photoreceptors and essential for timely recovery from photoexcitation. Recent clinical data show that, despite a hi...
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Izdano u: | Hum Gene Ther |
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Glavni autori: | , , , , , , , , , , , , |
Format: | Artigo |
Jezik: | Inglês |
Izdano: |
Mary Ann Liebert, Inc.
2015
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Teme: | |
Online pristup: | https://ncbi.nlm.nih.gov/pmc/articles/PMC4575531/ https://ncbi.nlm.nih.gov/pubmed/26247368 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1089/hum.2015.053 |
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