Chaperone-mediated gene therapy with recombinant AAV-PPCA in a new mouse model of type I sialidosis.
Biochim Biophys Acta
; 1832(10): 1784-92, 2013 Oct.
Article
en En
| MEDLINE
| ID: mdl-23770387
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:
1
Base de datos:
MEDLINE
Asunto principal:
Recombinación Genética
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Terapia Genética
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Dependovirus
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Chaperonas Moleculares
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Mucolipidosis
Idioma:
En
Revista:
Biochim Biophys Acta
Año:
2013
Tipo del documento:
Article