Chaperone-mediated gene therapy with recombinant AAV-PPCA in a new mouse model of type I sialidosis.

Bonten, Erik J; Yogalingam, Gouri; Hu, Huimin; Gomero, Elida; van de Vlekkert, Diantha; d'Azzo, Alessandra

Bonten, Erik J; Yogalingam, Gouri; Hu, Huimin; Gomero, Elida; van de Vlekkert, Diantha; d'Azzo, Alessandra.

Afiliación

Bonten EJ; Department of Genetics, St. Jude Children's Research Hospital, Memphis, TN 38105, USA.

Biochim Biophys Acta ; 1832(10): 1784-92, 2013 Oct.

Article en En | MEDLINE | ID: mdl-23770387

Asunto(s)

Dependovirus/genética; Terapia Genética; Chaperonas Moleculares/metabolismo; Mucolipidosis/terapia; Recombinación Genética; Animales; Modelos Animales de Enfermedad; Ratones; Ratones Noqueados

Palabras clave

AAV; BMEF; Chaperone-mediated therapy; ERT; FACE; GS; Gene therapy; IHC; KO; KO/TG; LSD; Lysosomal storage disease; NEU1; PCT; PPCA; Sialidosis; WT; bone marrow extracellular fluid; enzyme replacement therapy; fluorophore-assisted carbohydrate electrophoresis; galactosialidosis; immunohistochemistry; knockout; knockouttransgenic; lysosomal storage disease; pharmacologic-chaperone therapy; protective protein/cathepsin A; scAAV; self-complementary adeno-associated virus; wild-type

Texto completo

Imprimir

XML

PubMed Links

Buscar en Google

Texto completo: 1 Base de datos: MEDLINE Asunto principal: Recombinación Genética / Terapia Genética / Dependovirus / Chaperonas Moleculares / Mucolipidosis Idioma: En Revista: Biochim Biophys Acta Año: 2013 Tipo del documento: Article

Texto completo

Imprimir

XML

PubMed Links

Buscar en Google