AAV-Mediated Gene Therapy in the Guanylate Cyclase (RetGC1/RetGC2) Double Knockout Mouse Model of Leber Congenital Amaurosis

Mutations in GUCY2D are associated with recessive Leber congenital amaurosis-1 (LCA1). GUCY2D encodes photoreceptor-specific, retinal guanylate cyclase-1 (RetGC1). Reports of retinal degeneration in LCA1 are conflicting; some describe no obvious degeneration and others report loss of both rods and c...

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Autori principali: Boye, Sanford L., Peshenko, Igor V., Huang, Wei Chieh, Min, Seok Hong, McDoom, Issam, Kay, Christine N., Liu, Xuan, Dyka, Frank M., Foster, Thomas C., Umino, Yumiko, Karan, Sukanya, Jacobson, Samuel G., Baehr, Wolfgang, Dizhoor, Alexander, Hauswirth, William W., Boye, Shannon E.
Natura: Artigo
Lingua:Inglês
Pubblicazione: Mary Ann Liebert, Inc. 2013
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Research Articles
Accesso online:https://ncbi.nlm.nih.gov/pmc/articles/PMC3581260/
https://ncbi.nlm.nih.gov/pubmed/23210611
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1089/hum.2012.193
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