| http://purl.uniprot.org/citations/21444805 | http://www.w3.org/1999/02/22-rdf-syntax-ns#type | http://purl.uniprot.org/core/Journal_Citation |
| http://purl.uniprot.org/citations/21444805 | http://www.w3.org/2000/01/rdf-schema#comment | "Patients with Bardet-Biedl syndrome (BBS) experience severe retinal degeneration as a result of impaired photoreceptor transport processes that are not yet fully understood. To date, there is no effective treatment for BBS-associated retinal degeneration, and blindness is imminent by the second decade of life. Here we report the development of an adeno-associated viral (AAV) vector that rescues rhodopsin mislocalization, maintains nearly normal-appearing rod outer segments, and prevents photoreceptor death in the Bbs4-null mouse model. Analysis of the electroretinogram a-wave indicates that rescued rod cells are functionally indistinguishable from wild-type rods. These results demonstrate that gene therapy can prevent retinal degeneration in a mammalian BBS model."xsd:string |
| http://purl.uniprot.org/citations/21444805 | http://purl.org/dc/terms/identifier | "doi:10.1073/pnas.1019222108"xsd:string |
| http://purl.uniprot.org/citations/21444805 | http://purl.uniprot.org/core/author | "Wu S.M."xsd:string |
| http://purl.uniprot.org/citations/21444805 | http://purl.uniprot.org/core/author | "Hauswirth W.W."xsd:string |
| http://purl.uniprot.org/citations/21444805 | http://purl.uniprot.org/core/author | "Simons D.L."xsd:string |
| http://purl.uniprot.org/citations/21444805 | http://purl.uniprot.org/core/author | "Boye S.L."xsd:string |
| http://purl.uniprot.org/citations/21444805 | http://purl.uniprot.org/core/date | "2011"xsd:gYear |
| http://purl.uniprot.org/citations/21444805 | http://purl.uniprot.org/core/name | "Proc Natl Acad Sci U S A"xsd:string |
| http://purl.uniprot.org/citations/21444805 | http://purl.uniprot.org/core/pages | "6276-6281"xsd:string |
| http://purl.uniprot.org/citations/21444805 | http://purl.uniprot.org/core/title | "Gene therapy prevents photoreceptor death and preserves retinal function in a Bardet-Biedl syndrome mouse model."xsd:string |
| http://purl.uniprot.org/citations/21444805 | http://purl.uniprot.org/core/volume | "108"xsd:string |
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| http://purl.uniprot.org/uniprot/P15409#attribution-512C055720AB5F9921F977558B70B41F | http://purl.uniprot.org/core/source | http://purl.uniprot.org/citations/21444805 |
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