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

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

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.

Afiliação

Sundaram SM; Institute for Experimental and Clinical Pharmacology and Toxicology, Center of Brain, Behavior and Metabolism, University of Lübeck, 23562 Lübeck, Germany.
Arrulo Pereira A; Institute for Experimental and Clinical Pharmacology and Toxicology, Center of Brain, Behavior and Metabolism, University of Lübeck, 23562 Lübeck, Germany.
Müller-Fielitz H; Institute for Experimental and Clinical Pharmacology and Toxicology, Center of Brain, Behavior and Metabolism, University of Lübeck, 23562 Lübeck, Germany.
Köpke H; Institute for Experimental and Clinical Pharmacology and Toxicology, Center of Brain, Behavior and Metabolism, University of Lübeck, 23562 Lübeck, Germany.
De Angelis M; Institute for Diabetes and Obesity, Helmholtz Zentrum Munich, Munich, and German Center for Diabetes Research (DZD), 85764 Neuherberg, Germany.
Müller TD; Institute of Experimental Genetics, Helmholtz Zentrum Munich, German Research Center for Environmental Health (GmbH), 85764 Neuherberg, Germany.
Heuer H; Institute for Diabetes and Obesity, Helmholtz Zentrum Munich, Munich, and German Center for Diabetes Research (DZD), 85764 Neuherberg, Germany.
Körbelin J; Department of Endocrinology, Diabetes and Metabolism, University Hospital Essen, University Duisburg-Essen, 45147 Essen, Germany.
Krohn M; Institute for Experimental and Clinical Pharmacology and Toxicology, Center of Brain, Behavior and Metabolism, University of Lübeck, 23562 Lübeck, Germany.
Mittag J; Department of Oncology, Hematology and Bone Marrow Transplantation, UKE Hamburg-Eppendorf, 20246 Hamburg, Germany.
Nogueiras R; Institute for Experimental and Clinical Pharmacology and Toxicology, Center of Brain, Behavior and Metabolism, University of Lübeck, 23562 Lübeck, Germany.
Prevot V; Institute for Endocrinology and Diabetes, Center of Brain, Behavior and Metabolism, University of Lübeck, 23562 Lübeck, Germany.
Schwaninger M; Department of Physiology, CIMUS, University of Santiago de Compostela-Instituto de Investigación Sanitaria, 15782 Santiago de Compostela, Spain.

Brain ; 145(12): 4264-4274, 2022 12 19.

Article em En | MEDLINE | ID: mdl-35929549

Assuntos

Pessoas com Deficiência; Deficiência Intelectual Ligada ao Cromossomo X; Transtornos Motores; Simportadores; Camundongos; Animais; Humanos; Barreira Hematoencefálica/metabolismo; Deficiência Intelectual Ligada ao Cromossomo X/genética; Deficiência Intelectual Ligada ao Cromossomo X/metabolismo; Hipotonia Muscular/genética; Atrofia Muscular; Células Endoteliais/metabolismo; Transportadores de Ácidos Monocarboxílicos/genética; Transportadores de Ácidos Monocarboxílicos/metabolismo; Hormônios Tireóideos/metabolismo; Terapia Genética; Simportadores/genética; Simportadores/metabolismo

Palavras-chave

cretinism; hypothyroidism; myelination; thyroxine; tri-iodothyronine

Texto completo

Imprimir

XML

PubMed Links

Buscar no Google

Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Assunto principal: Pessoas com Deficiência / Simportadores / Deficiência Intelectual Ligada ao Cromossomo X / Transtornos Motores Tipo de estudo: Diagnostic_studies Limite: Animals / Humans Idioma: En Revista: Brain Ano de publicação: 2022 Tipo de documento: Article País de afiliação: Alemanha

Texto completo

Imprimir

XML

PubMed Links

Buscar no Google