Matches in SemOpenAlex for { <https://semopenalex.org/work/W4200089724> ?p ?o ?g. }
- W4200089724 abstract "Abstract The solute carrier monocarboxylate transporter 8 (MCT8) transports the thyroid hormones thyroxine and tri-iodothyronine (T3) across cell membranes. MCT8 gene deficiency, termed Allan-Herndon-Dudley syndrome, is an important cause of X-linked intellectual and motor disability. As no treatment of the neurological symptoms is available yet, we tested a gene replacement therapy in Mct8 - and Oatp1c1 -deficient mice as a well-established model of the disease. Here, we report that targeting brain endothelial cells for Mct8 expression by intravenously injecting the vector AAV-BR1- Mct8 increased T3 levels in the brain and ameliorated morphological and functional parameters associated with the disease. Importantly, the therapy resulted in a long-lasting improvement in motor coordination. Thus, the data support the concept that MCT8 mediates the transport of thyroid hormones into the brain and indicate that a readily accessible vascular target can help overcome the consequences of the severe disability associated with MCT8 deficiency." @default.
- W4200089724 created "2021-12-31" @default.
- W4200089724 creator A5009423317 @default.
- W4200089724 creator A5020982137 @default.
- W4200089724 creator A5021958718 @default.
- W4200089724 creator A5034210646 @default.
- W4200089724 creator A5034256496 @default.
- W4200089724 creator A5046680344 @default.
- W4200089724 creator A5049883111 @default.
- W4200089724 creator A5055064257 @default.
- W4200089724 creator A5059374913 @default.
- W4200089724 creator A5075655517 @default.
- W4200089724 creator A5076442919 @default.
- W4200089724 creator A5083058332 @default.
- W4200089724 creator A5085134513 @default.
- W4200089724 date "2021-12-09" @default.
- W4200089724 modified "2023-10-01" @default.
- W4200089724 title "Gene therapy targeting the blood-brain barrier improves neurological symptoms in a model of genetic MCT8 deficiency" @default.
- W4200089724 cites W1483740421 @default.
- W4200089724 cites W150428577 @default.
- W4200089724 cites W1845614074 @default.
- W4200089724 cites W1900556344 @default.
- W4200089724 cites W1965357288 @default.
- W4200089724 cites W1975345061 @default.
- W4200089724 cites W1992268803 @default.
- W4200089724 cites W2003476788 @default.
- W4200089724 cites W2008802603 @default.
- W4200089724 cites W2014894652 @default.
- W4200089724 cites W2017632004 @default.
- W4200089724 cites W2020564162 @default.
- W4200089724 cites W2027397385 @default.
- W4200089724 cites W2046037386 @default.
- W4200089724 cites W2046152275 @default.
- W4200089724 cites W2089423544 @default.
- W4200089724 cites W2106411599 @default.
- W4200089724 cites W2116353330 @default.
- W4200089724 cites W2123696673 @default.
- W4200089724 cites W2276571131 @default.
- W4200089724 cites W2339068621 @default.
- W4200089724 cites W2518440531 @default.
- W4200089724 cites W2615774542 @default.
- W4200089724 cites W2948751912 @default.
- W4200089724 cites W2964563673 @default.
- W4200089724 cites W2999402088 @default.
- W4200089724 cites W3009870860 @default.
- W4200089724 cites W3031744338 @default.
- W4200089724 cites W3104374915 @default.
- W4200089724 cites W3183539586 @default.
- W4200089724 doi "https://doi.org/10.1101/2021.12.05.471343" @default.
- W4200089724 hasPublicationYear "2021" @default.
- W4200089724 type Work @default.
- W4200089724 citedByCount "1" @default.
- W4200089724 countsByYear W42000897242023 @default.
- W4200089724 crossrefType "posted-content" @default.
- W4200089724 hasAuthorship W4200089724A5009423317 @default.
- W4200089724 hasAuthorship W4200089724A5020982137 @default.
- W4200089724 hasAuthorship W4200089724A5021958718 @default.
- W4200089724 hasAuthorship W4200089724A5034210646 @default.
- W4200089724 hasAuthorship W4200089724A5034256496 @default.
- W4200089724 hasAuthorship W4200089724A5046680344 @default.
- W4200089724 hasAuthorship W4200089724A5049883111 @default.
- W4200089724 hasAuthorship W4200089724A5055064257 @default.
- W4200089724 hasAuthorship W4200089724A5059374913 @default.
- W4200089724 hasAuthorship W4200089724A5075655517 @default.
- W4200089724 hasAuthorship W4200089724A5076442919 @default.
- W4200089724 hasAuthorship W4200089724A5083058332 @default.
- W4200089724 hasAuthorship W4200089724A5085134513 @default.
- W4200089724 hasBestOaLocation W42000897241 @default.
- W4200089724 hasConcept C104317684 @default.
- W4200089724 hasConcept C111599444 @default.
- W4200089724 hasConcept C126322002 @default.
- W4200089724 hasConcept C134018914 @default.
- W4200089724 hasConcept C149011108 @default.
- W4200089724 hasConcept C15744967 @default.
- W4200089724 hasConcept C169760540 @default.
- W4200089724 hasConcept C2778402981 @default.
- W4200089724 hasConcept C2779134260 @default.
- W4200089724 hasConcept C2781217356 @default.
- W4200089724 hasConcept C526584372 @default.
- W4200089724 hasConcept C529278444 @default.
- W4200089724 hasConcept C54355233 @default.
- W4200089724 hasConcept C60644358 @default.
- W4200089724 hasConcept C71315377 @default.
- W4200089724 hasConcept C71924100 @default.
- W4200089724 hasConcept C86803240 @default.
- W4200089724 hasConceptScore W4200089724C104317684 @default.
- W4200089724 hasConceptScore W4200089724C111599444 @default.
- W4200089724 hasConceptScore W4200089724C126322002 @default.
- W4200089724 hasConceptScore W4200089724C134018914 @default.
- W4200089724 hasConceptScore W4200089724C149011108 @default.
- W4200089724 hasConceptScore W4200089724C15744967 @default.
- W4200089724 hasConceptScore W4200089724C169760540 @default.
- W4200089724 hasConceptScore W4200089724C2778402981 @default.
- W4200089724 hasConceptScore W4200089724C2779134260 @default.
- W4200089724 hasConceptScore W4200089724C2781217356 @default.
- W4200089724 hasConceptScore W4200089724C526584372 @default.
- W4200089724 hasConceptScore W4200089724C529278444 @default.
- W4200089724 hasConceptScore W4200089724C54355233 @default.
- W4200089724 hasConceptScore W4200089724C60644358 @default.
- W4200089724 hasConceptScore W4200089724C71315377 @default.