Matches in SemOpenAlex for { <https://semopenalex.org/work/W4378839062> ?p ?o ?g. }
Showing items 1 to 78 of
78
with 100 items per page.
- W4378839062 abstract "Retinitis pigmentosa affects 1 in 4,000 people worldwide. Mutations in over twenty genes cause autosomal dominant retinitis pigmentosa. 30-40% of autosomal dominant retinitis pigmentosa are due to mutations rhodopsin with more than 100 known mutations being identified. Due to this allelic and causal diversity, allele-independent approaches are an attractive option. Here, we demonstrate an allele-independent CRISPR/Cas9 approach; this contrasts with many current allele-specific rhodopsin approaches. A homology-independent transgene is co-delivered with RSV-spCas9 packaged in an AAV5 vector for gene replacement via insertion in the Rho I307N mouse model of autosomal dominant retinitis pigmentosa. First, we establish the safety of this system, in C57BL/6J mice, demonstrating no loss of retinal thickness or function. We further show that outer nuclear layer thickness, electrical response, and rhodopsin expression in heterozygous Rho I307N were significantly preserved six months after treatment. This retention results from a 5.7% transgene integration and 88% indel rate in treated animals." @default.
- W4378839062 created "2023-06-01" @default.
- W4378839062 creator A5045630481 @default.
- W4378839062 creator A5091715701 @default.
- W4378839062 date "2023-05-17" @default.
- W4378839062 modified "2023-09-30" @default.
- W4378839062 title "Rhodopsin Replacement in a I307N Mouse Model of ADRP by the Homology Independent Transgene Insertion Method" @default.
- W4378839062 cites W144597972 @default.
- W4378839062 cites W1988378555 @default.
- W4378839062 cites W2008147758 @default.
- W4378839062 cites W2052586386 @default.
- W4378839062 cites W2055198588 @default.
- W4378839062 cites W2061145166 @default.
- W4378839062 cites W2073995081 @default.
- W4378839062 cites W2077508733 @default.
- W4378839062 cites W2113669792 @default.
- W4378839062 cites W2136272785 @default.
- W4378839062 cites W2140092254 @default.
- W4378839062 cites W2141296965 @default.
- W4378839062 cites W2267596434 @default.
- W4378839062 cites W2371599855 @default.
- W4378839062 cites W2410674135 @default.
- W4378839062 cites W2411180332 @default.
- W4378839062 cites W2519167750 @default.
- W4378839062 cites W2550079706 @default.
- W4378839062 cites W2595964111 @default.
- W4378839062 cites W2788789448 @default.
- W4378839062 cites W2793309516 @default.
- W4378839062 cites W2800711204 @default.
- W4378839062 cites W2888444358 @default.
- W4378839062 cites W2902652258 @default.
- W4378839062 cites W3001729408 @default.
- W4378839062 doi "https://doi.org/10.14293/s2199-1006.1.sor-.ppbjpmo.v2" @default.
- W4378839062 hasPublicationYear "2023" @default.
- W4378839062 type Work @default.
- W4378839062 citedByCount "0" @default.
- W4378839062 crossrefType "posted-content" @default.
- W4378839062 hasAuthorship W4378839062A5045630481 @default.
- W4378839062 hasAuthorship W4378839062A5091715701 @default.
- W4378839062 hasBestOaLocation W43788390621 @default.
- W4378839062 hasConcept C102230213 @default.
- W4378839062 hasConcept C104317684 @default.
- W4378839062 hasConcept C118083812 @default.
- W4378839062 hasConcept C180754005 @default.
- W4378839062 hasConcept C202033177 @default.
- W4378839062 hasConcept C2780827179 @default.
- W4378839062 hasConcept C2781114197 @default.
- W4378839062 hasConcept C501734568 @default.
- W4378839062 hasConcept C54355233 @default.
- W4378839062 hasConcept C55493867 @default.
- W4378839062 hasConcept C86803240 @default.
- W4378839062 hasConceptScore W4378839062C102230213 @default.
- W4378839062 hasConceptScore W4378839062C104317684 @default.
- W4378839062 hasConceptScore W4378839062C118083812 @default.
- W4378839062 hasConceptScore W4378839062C180754005 @default.
- W4378839062 hasConceptScore W4378839062C202033177 @default.
- W4378839062 hasConceptScore W4378839062C2780827179 @default.
- W4378839062 hasConceptScore W4378839062C2781114197 @default.
- W4378839062 hasConceptScore W4378839062C501734568 @default.
- W4378839062 hasConceptScore W4378839062C54355233 @default.
- W4378839062 hasConceptScore W4378839062C55493867 @default.
- W4378839062 hasConceptScore W4378839062C86803240 @default.
- W4378839062 hasLocation W43788390621 @default.
- W4378839062 hasOpenAccess W4378839062 @default.
- W4378839062 hasPrimaryLocation W43788390621 @default.
- W4378839062 hasRelatedWork W143481608 @default.
- W4378839062 hasRelatedWork W1971452416 @default.
- W4378839062 hasRelatedWork W2025181221 @default.
- W4378839062 hasRelatedWork W2026654731 @default.
- W4378839062 hasRelatedWork W2057739827 @default.
- W4378839062 hasRelatedWork W2162650178 @default.
- W4378839062 hasRelatedWork W2223919059 @default.
- W4378839062 hasRelatedWork W2408941577 @default.
- W4378839062 hasRelatedWork W2412378213 @default.
- W4378839062 hasRelatedWork W3035627949 @default.
- W4378839062 isParatext "false" @default.
- W4378839062 isRetracted "false" @default.
- W4378839062 workType "article" @default.