AAV-mediated expression of galactose-1-phosphate uridyltransferase corrects defects of galactose metabolism in classic galactosemia patient fibroblasts.

Brophy, Megan L; Stansfield, John C; Ahn, Youngwook; Cheng, Seng H; Murphy, John E; Bell, Robert D

Brophy, Megan L; Stansfield, John C; Ahn, Youngwook; Cheng, Seng H; Murphy, John E; Bell, Robert D.

Afiliación

Brophy ML; Rare Disease Research Unit, Pfizer, Inc., Cambridge, Massachusetts, USA.
Stansfield JC; Early Clinical Development, Pfizer, Inc., Cambridge, Massachusetts, USA.
Ahn Y; Target Sciences, Pfizer, Inc., Cambridge, Massachusetts, USA.
Cheng SH; Rare Disease Research Unit, Pfizer, Inc., Cambridge, Massachusetts, USA.
Murphy JE; Rare Disease Research Unit, Pfizer, Inc., Cambridge, Massachusetts, USA.
Bell RD; Rare Disease Research Unit, Pfizer, Inc., Cambridge, Massachusetts, USA.

J Inherit Metab Dis ; 45(3): 481-492, 2022 05.

Article en En | MEDLINE | ID: mdl-34918784

Asunto(s)

Galactosemias; UTP-Hexosa-1-Fosfato Uridililtransferasa; Animales; Fibroblastos/metabolismo; Galactosa/metabolismo; Galactosemias/genética; Galactosemias/terapia; Humanos; Ratones; Ratones Noqueados; UTP-Hexosa-1-Fosfato Uridililtransferasa/genética; UTP-Hexosa-1-Fosfato Uridililtransferasa/metabolismo

Palabras clave

AAV; CD109; TGFßRII; galactose; galactosemia; gene therapy

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Texto completo: 1 Colección: 01-internacional Banco de datos: MEDLINE Asunto principal: UTP-Hexosa-1-Fosfato Uridililtransferasa / Galactosemias Límite: Animals / Humans Idioma: En Revista: J Inherit Metab Dis Año: 2022 Tipo del documento: Article País de afiliación: Estados Unidos

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