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Gene therapy targeting the blood–brain barrier improves neurological symptoms in a model of genetic MCT8 deficiency
A genetic deficiency of the solute carrier monocarboxylate transporter 8 (MCT8), termed Allan–Herndon–Dudley syndrome, is an important cause of X-linked intellectual and motor disability. MCT8 transports thyroid hormones across cell membranes. While thyroid hormone analogues improve peripheral chang...
Autores principales: | Sundaram, Sivaraj M, Arrulo Pereira, Adriana, Müller-Fielitz, Helge, Köpke, Hannes, De Angelis, Meri, Müller, Timo D, Heuer, Heike, Körbelin, Jakob, Krohn, Markus, Mittag, Jens, Nogueiras, Ruben, Prevot, Vincent, Schwaninger, Markus |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
Oxford University Press
2022
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Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9762946/ https://www.ncbi.nlm.nih.gov/pubmed/35929549 http://dx.doi.org/10.1093/brain/awac243 |
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