QUINN, ANTHONY,XIA, ZHINAN,LEAVITT, MARKLEY, C.,RUTKOWSKI, JOSEPH, VICTOR
申请号：
CL2014000927
公开号：
CL2014000927A1
申请日:
2014.04.11
申请国别(地区):
CL
年份:
2014
代理人:
摘要：
The present invention provides compositions comprising an isolated mixture of recombinant human NaGlu proteins in which a substantial amount of the NaGlu proteins in the mixture has increased levels of phosphorylated mannose that confer the proteins to be efficiently internalized into human cells. The present invention also provides methods of producing such mixture of NaGlu proteins, vectors used in transgenesis and expression, host cells harboring such vectors, and methods of isolating and purifying the mixture of NaGlu proteins. The invention further provides methods of treating NaGlu associated diseases.una composición que comprende una mezcla aislada de N-acetil-a-D-glucosaminidasa (rhNaGlu) humana recombinante donde dicha mezcla comprende de rhNaGlu que contiene una o más estructuras de glucano que comprenden manosa-6-fosfato (M6P), y donde la rhNaGlu contiene al menos 1 mol de M6P por mol de proteína rhNaGlu. Uso de rhNaGlu que sirve para la preparación de un medicamento útil para tratar a un individuo que sufre de deficiencia de NaGlu
