| http://purl.uniprot.org/citations/25855802 | http://www.w3.org/1999/02/22-rdf-syntax-ns#type | http://purl.uniprot.org/core/Journal_Citation |
| http://purl.uniprot.org/citations/25855802 | http://www.w3.org/2000/01/rdf-schema#comment | "The CNGA3(-/-)/Nrl(-/-) mouse is a cone-dominant model with Cnga3 channel deficiency, which partially mimics the all cone foveal structure of human achromatopsia 2 with CNGA3 mutations. Although subretinal (SR) AAV vector administration can transfect retinal cells efficiently, the injection-induced retinal detachment can cause retinal damage, particularly when SR vector bleb includes the fovea. We therefore explored whether cone function-structure could be rescued in CNGA3(-/-)/Nrl(-/-) mice by intravitreal (IVit) delivery of tyrosine to phenylalanine (Y-F) capsid mutant AAV8. We find that AAV-mediated CNGA3 expression can restore cone function and rescue structure following IVit delivery of AAV8 (Y447, 733F) vector. Rescue was assessed by restoration of the cone-mediated electroretinogram (ERG), optomotor responses, and cone opsin immunohistochemistry. Demonstration of gene therapy in a cone-dominant mouse model by IVit delivery provides a potential alternative vector delivery mode for safely transducing foveal cones in achromatopsia patients and in other human retinal diseases affecting foveal function."xsd:string |
| http://purl.uniprot.org/citations/25855802 | http://purl.org/dc/terms/identifier | "doi:10.1093/hmg/ddv114"xsd:string |
| http://purl.uniprot.org/citations/25855802 | http://purl.uniprot.org/core/author | "Dai X."xsd:string |
| http://purl.uniprot.org/citations/25855802 | http://purl.uniprot.org/core/author | "Li J."xsd:string |
| http://purl.uniprot.org/citations/25855802 | http://purl.uniprot.org/core/author | "Liu X."xsd:string |
| http://purl.uniprot.org/citations/25855802 | http://purl.uniprot.org/core/author | "Tao Y."xsd:string |
| http://purl.uniprot.org/citations/25855802 | http://purl.uniprot.org/core/author | "Zhang Y."xsd:string |
| http://purl.uniprot.org/citations/25855802 | http://purl.uniprot.org/core/author | "Zhang Z."xsd:string |
| http://purl.uniprot.org/citations/25855802 | http://purl.uniprot.org/core/author | "Shi W."xsd:string |
| http://purl.uniprot.org/citations/25855802 | http://purl.uniprot.org/core/author | "Zhu P."xsd:string |
| http://purl.uniprot.org/citations/25855802 | http://purl.uniprot.org/core/author | "Zhao C."xsd:string |
| http://purl.uniprot.org/citations/25855802 | http://purl.uniprot.org/core/author | "Qu J."xsd:string |
| http://purl.uniprot.org/citations/25855802 | http://purl.uniprot.org/core/author | "Du W."xsd:string |
| http://purl.uniprot.org/citations/25855802 | http://purl.uniprot.org/core/author | "Hauswirth W.W."xsd:string |
| http://purl.uniprot.org/citations/25855802 | http://purl.uniprot.org/core/author | "Biel M."xsd:string |
| http://purl.uniprot.org/citations/25855802 | http://purl.uniprot.org/core/author | "Ding X.Q."xsd:string |
| http://purl.uniprot.org/citations/25855802 | http://purl.uniprot.org/core/author | "Michalakis S."xsd:string |
| http://purl.uniprot.org/citations/25855802 | http://purl.uniprot.org/core/author | "Chiodo V.A."xsd:string |
| http://purl.uniprot.org/citations/25855802 | http://purl.uniprot.org/core/author | "Pang J.J."xsd:string |
| http://purl.uniprot.org/citations/25855802 | http://purl.uniprot.org/core/author | "Deng W.T."xsd:string |
| http://purl.uniprot.org/citations/25855802 | http://purl.uniprot.org/core/date | "2015"xsd:gYear |
| http://purl.uniprot.org/citations/25855802 | http://purl.uniprot.org/core/name | "Hum Mol Genet"xsd:string |
| http://purl.uniprot.org/citations/25855802 | http://purl.uniprot.org/core/pages | "3699-3707"xsd:string |
| http://purl.uniprot.org/citations/25855802 | http://purl.uniprot.org/core/title | "Vitreal delivery of AAV vectored Cnga3 restores cone function in CNGA3-/-/Nrl-/- mice, an all-cone model of CNGA3 achromatopsia."xsd:string |