Adeno Associated Virus 9–Based Gene Therapy Delivers a Functional Monocarboxylate Transporter 8, Improving Thyroid Hormone Availability to the Brain of Mct8-Deficient Mice

Background: MCT8 gene mutations produce thyroid hormone (TH) deficiency in the brain, causing severe neuropsychomotor abnormalities not correctable by treatment with TH. This proof-of-concept study examined whether transfer of human MCT8 (hMCT8) cDNA using adeno-associated virus 9 (AAV9) could corre...

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Vydáno v:Thyroid
Hlavní autoři: Iwayama, Hideyuki, Liao, Xiao-Hui, Braun, Lyndsey, Bárez-López, Soledad, Kaspar, Brian, Weiss, Roy E., Dumitrescu, Alexandra M., Guadaño-Ferraz, Ana, Refetoff, Samuel
Médium: Artigo
Jazyk:Inglês
Vydáno: Mary Ann Liebert, Inc. 2016
Témata:
Thyroid Economy: Regulation, Cell Biology, and Thyroid Hormone Metabolism and Action
On-line přístup:https://ncbi.nlm.nih.gov/pmc/articles/PMC5036314/
https://ncbi.nlm.nih.gov/pubmed/27432638
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1089/thy.2016.0060
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