Reduction of lysosome abundance and GAG accumulation after odiparcil treatment in MPS I and MPS VI models.

Tuyaa-Boustugue, Pascale; Jantzen, Ingrid; Zhang, Haoyue; Young, Sarah P; Broqua, Pierre; Tallandier, Mireille; Entchev, Eugeni

Tuyaa-Boustugue, Pascale; Jantzen, Ingrid; Zhang, Haoyue; Young, Sarah P; Broqua, Pierre; Tallandier, Mireille; Entchev, Eugeni.

Afiliação

Tuyaa-Boustugue P; Inventiva Pharma, 50 Rue de Dijon, Daix 21121, France.
Jantzen I; Inventiva Pharma, 50 Rue de Dijon, Daix 21121, France.
Zhang H; Duke University Health System Biochemical Genetics Lab, Durham, NC, USA.
Young SP; Duke University Health System Biochemical Genetics Lab, Durham, NC, USA.
Broqua P; Division of Medical Genetics, Department of Pediatrics, Duke School of Medicine, Durham, NC, USA.
Tallandier M; Inventiva Pharma, 50 Rue de Dijon, Daix 21121, France.
Entchev E; Inventiva Pharma, 50 Rue de Dijon, Daix 21121, France.

Mol Genet Metab Rep ; 37: 101011, 2023 Dec.

Article em En | MEDLINE | ID: mdl-38053941

Palavras-chave

GAG composition; Lysosome; MPS I therapy; MPS VI therapy; Mucopolysaccharidosis type I; Mucopolysaccharidosis type VI; Odiparcil

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Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Idioma: En Ano de publicação: 2023 Tipo de documento: Article

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Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Idioma: En Ano de publicação: 2023 Tipo de documento: Article