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...

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Detalles Bibliográficos
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
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Oxford University Press 2022
Materias:
Original Article
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

Internet

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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