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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Bibliografische gegevens
Hoofdauteurs: 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.
Formaat: Artigo
Taal:Inglês
Gepubliceerd in: Mary Ann Liebert, Inc. 2013
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Research Articles
Online toegang: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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