Article

Gene therapy targeting the blood-brain barrier improves neurological symptoms in a model of genetic MCT8 deficiency

2021-12-09

Abstract excerpt

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...

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Literature Corpus work: 1b3254b5-191f-5ad2-8cd4-a93091b18b21
DOI: 10.1101/2021.12.05.471343

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