Expression of cDNA-encoded human acid alpha-glucosidase in milk of transgenic mice.
Biochim Biophys Acta
; 1308(2): 93-6, 1996 Aug 14.
Article
em En
| MEDLINE
| ID: mdl-8764823
ABSTRACT
Enzyme replacement therapy is at present the option of choice for treatment of lysosomal storage diseases. To explore the feasibility of lysosomal enzyme production in milk of transgenic animals, the human acid alpha-glucosidase cDNA was placed under control of the alpha S1-casein promoter and expressed in mice. The milk contained recombinant enzyme at a concentration up to 1.5 micrograms/ml. Enzyme purified from milk of transgenic mice was internalized via the mannose 6-phosphate receptor and corrected enzyme deficiency in fibroblasts from patients. We conclude that transgenically produced human acid alpha-glucosidase meets the criteria for therapeutic application.
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Base de dados:
MEDLINE
Assunto principal:
Proteínas Recombinantes
/
Doença de Depósito de Glicogênio Tipo II
/
Glucana 1,4-alfa-Glucosidase
/
Leite
Limite:
Animals
/
Humans
Idioma:
En
Ano de publicação:
1996
Tipo de documento:
Article