Patient iPSC-derived neural stem cells exhibit phenotypes in concordance with the clinical severity of mucopolysaccharidosis I.

Swaroop, Manju; Brooks, Matthew J; Gieser, Linn; Swaroop, Anand; Zheng, Wei

Swaroop, Manju; Brooks, Matthew J; Gieser, Linn; Swaroop, Anand; Zheng, Wei.

Afiliação

Swaroop M; National Therapeutics for Rare and Neglected Diseases, National Center for Advancing Translational Sciences, National Institutes of Health, Rockville, MD, USA.
Brooks MJ; Neurobiology-Neurodegeneration and Repair Laboratory, National Eye Institute, National Institutes of Health, Bethesda, MD, USA.
Gieser L; Neurobiology-Neurodegeneration and Repair Laboratory, National Eye Institute, National Institutes of Health, Bethesda, MD, USA.
Swaroop A; Neurobiology-Neurodegeneration and Repair Laboratory, National Eye Institute, National Institutes of Health, Bethesda, MD, USA.
Zheng W; National Therapeutics for Rare and Neglected Diseases, National Center for Advancing Translational Sciences, National Institutes of Health, Rockville, MD, USA.

Hum Mol Genet ; 27(20): 3612-3626, 2018 10 15.

Article em En | MEDLINE | ID: mdl-30052969

Assuntos

Iduronidase/genética; Mucopolissacaridose I/enzimologia; Células-Tronco Neurais; Fenótipo; Linhagem Celular; Criança; Pré-Escolar; Perfilação da Expressão Gênica; Glicosaminoglicanos/metabolismo; Humanos; Iduronidase/metabolismo; Células-Tronco Pluripotentes Induzidas; Lisossomos; Masculino; Mucopolissacaridose I/genética; Mucopolissacaridose I/metabolismo; Mutação

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Texto completo: 1 Base de dados: MEDLINE Assunto principal: Fenótipo / Mucopolissacaridose I / Células-Tronco Neurais / Iduronidase Tipo de estudo: Prognostic_studies Limite: Child / Child, preschool / Humans / Male Idioma: En Ano de publicação: 2018 Tipo de documento: Article

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