FRINK Linked Data Fragments server

Matches in SemOpenAlex for { <https://semopenalex.org/work/W3022921364> ?p ?o ?g. }

• W3022921364 abstract "ABSTRACT Retinal degeneration is a common clinical feature of ciliopathies, a group of genetic diseases linked to ciliary dysfunction, and gene therapy is an attractive treatment option to prevent vision loss. Although the efficacy of retinal gene therapy is well established by multiple proof-of-concept preclinical studies, its long-term effect, particularly when treatments are given at advanced disease stages, is controversial. Incomplete treatment and intrinsic variability of gene delivery methods may contribute to the variable outcomes. Here, we used a genetic rescue approach to “optimally” treat retinal degeneration at various disease stages and examined the long-term efficacy of gene therapy in a mouse model of ciliopathy. We used a Bardet-Biedl syndrome type 17 (BBS17) mouse model, in which the gene-trap that suppresses Bbs17 (also known as Lztfl1 ) expression can be removed by tamoxifen administration, restoring normal gene expression systemically. Our data indicate that therapeutic effects of retinal gene therapy decrease gradually as treatments are given at later stages. These results suggest the presence of limited time window for successful gene therapy in certain retinal degenerations. Our study also implies that the long-term efficacy of retinal gene therapy may depend on not only the timing of treatment but also other factors such as the function of mutated genes and residual activities of mutant alleles." @default.
• W3022921364 created "2020-05-13" @default.
• W3022921364 creator A5019405283 @default.
• W3022921364 creator A5039831882 @default.
• W3022921364 creator A5081449876 @default.
• W3022921364 date "2020-05-06" @default.
• W3022921364 modified "2023-10-16" @default.
• W3022921364 title "Limited time window for retinal gene therapy in a preclinical model of ciliopathy" @default.
• W3022921364 cites W1621414929 @default.
• W3022921364 cites W1776335365 @default.
• W3022921364 cites W1864140687 @default.
• W3022921364 cites W1915214953 @default.
• W3022921364 cites W1954666599 @default.
• W3022921364 cites W1968904141 @default.
• W3022921364 cites W1968937480 @default.
• W3022921364 cites W1973721465 @default.
• W3022921364 cites W1974113099 @default.
• W3022921364 cites W1976421227 @default.
• W3022921364 cites W1976846514 @default.
• W3022921364 cites W1979921735 @default.
• W3022921364 cites W1980100245 @default.
• W3022921364 cites W1980603153 @default.
• W3022921364 cites W1983623527 @default.
• W3022921364 cites W1990832044 @default.
• W3022921364 cites W1991090223 @default.
• W3022921364 cites W1997956353 @default.
• W3022921364 cites W2001488073 @default.
• W3022921364 cites W2001603084 @default.
• W3022921364 cites W2012796647 @default.
• W3022921364 cites W2013588839 @default.
• W3022921364 cites W2016211106 @default.
• W3022921364 cites W2018490222 @default.
• W3022921364 cites W2023339971 @default.
• W3022921364 cites W2050040558 @default.
• W3022921364 cites W2052513354 @default.
• W3022921364 cites W2054158396 @default.
• W3022921364 cites W2070394325 @default.
• W3022921364 cites W2076521138 @default.
• W3022921364 cites W2081273157 @default.
• W3022921364 cites W2086498377 @default.
• W3022921364 cites W2086790354 @default.
• W3022921364 cites W2094326537 @default.
• W3022921364 cites W2094799320 @default.
• W3022921364 cites W2095486148 @default.
• W3022921364 cites W2096190491 @default.
• W3022921364 cites W2098642477 @default.
• W3022921364 cites W2100648176 @default.
• W3022921364 cites W2107277218 @default.
• W3022921364 cites W2112171220 @default.
• W3022921364 cites W2112348219 @default.
• W3022921364 cites W2132812842 @default.
• W3022921364 cites W2134841035 @default.
• W3022921364 cites W2137222303 @default.
• W3022921364 cites W2138374168 @default.
• W3022921364 cites W2139312688 @default.
• W3022921364 cites W2141575938 @default.
• W3022921364 cites W2151809142 @default.
• W3022921364 cites W2162290849 @default.
• W3022921364 cites W2165703612 @default.
• W3022921364 cites W2167706986 @default.
• W3022921364 cites W2170087992 @default.
• W3022921364 cites W2473832992 @default.
• W3022921364 cites W2595964111 @default.
• W3022921364 cites W2608671477 @default.
• W3022921364 cites W2611565615 @default.
• W3022921364 cites W2620518508 @default.
• W3022921364 cites W265142245 @default.
• W3022921364 cites W2766072916 @default.
• W3022921364 cites W2767601133 @default.
• W3022921364 cites W2793994644 @default.
• W3022921364 cites W2809293496 @default.
• W3022921364 cites W2971157227 @default.
• W3022921364 cites W2971463685 @default.
• W3022921364 cites W2987060209 @default.
• W3022921364 cites W3022842541 @default.
• W3022921364 doi "https://doi.org/10.1101/2020.05.05.072793" @default.
• W3022921364 hasPublicationYear "2020" @default.
• W3022921364 type Work @default.
• W3022921364 sameAs 3022921364 @default.
• W3022921364 citedByCount "0" @default.
• W3022921364 crossrefType "posted-content" @default.
• W3022921364 hasAuthorship W3022921364A5019405283 @default.
• W3022921364 hasAuthorship W3022921364A5039831882 @default.
• W3022921364 hasAuthorship W3022921364A5081449876 @default.
• W3022921364 hasBestOaLocation W30229213641 @default.
• W3022921364 hasConcept C104317684 @default.
• W3022921364 hasConcept C111599444 @default.
• W3022921364 hasConcept C118487528 @default.
• W3022921364 hasConcept C127716648 @default.
• W3022921364 hasConcept C169760540 @default.
• W3022921364 hasConcept C2777093970 @default.
• W3022921364 hasConcept C2780561973 @default.
• W3022921364 hasConcept C2780827179 @default.
• W3022921364 hasConcept C2781040256 @default.
• W3022921364 hasConcept C2910395002 @default.
• W3022921364 hasConcept C54355233 @default.
• W3022921364 hasConcept C60644358 @default.
• W3022921364 hasConcept C71924100 @default.
• W3022921364 hasConcept C86803240 @default.
• W3022921364 hasConceptScore W3022921364C104317684 @default.

• next